Dr. Fred Nyberg, PhD

  • Dean of The Faculty of Pharmacy, Uppsala University
  • Professor in Biological Research on Drug Dependence
  • Swedish Medical Research Council at the Department Pharmaceutical Biosciences

Dr. Fred Nyberg has over 300 publications in internationally recognized industry and research journals, and his recent honors include the reward Sigillum of the Ateneo at University of Bologna in 2005, as well as appointments as Karl Johan Öbrink Lecturer at BMC in 2007, and Visiting Professor at Hoshi University, Tokyo (Japan) in 2009.

Dr. Fred Nyberg is currently the Dean for the Faculty of Pharmacy and a Full Professor at the Division of Biological Research on Drug Dependence, Department of Pharmaceutical Biosciences, Uppsala University. Dr. Nyberg received his PhD in 1981 on a thesis directed to studies on the polymorphism of the pituitary hormone prolactin, and subsequently  performed postdoc studies on opioid peptides in relation to chronic pain and opioid addiction.

Dr. Nyberg was appointed as researcher at the Swedish Medical Research Council in 1985, and between 1988-1992 he was employed as Professor in Pharmacology at the Department of Pharmacology, Faculty of Pharmacy, Uppsala University. In 1993 he was appointed by the Swedish Government as Professor for Biological Research on Drug Dependence at the Swedish Medical Research Council, then between 2002-2007 he was employed by the Swedish Government as Head of Research Issues at the National Drug Policy Coordinator. Since 2008 he has joined the Advisory Board at the ANDT unit at the Social Ministry dealing with questions related to drugs and drug addiction. Dr. Nyberg was Chairman for the Department of Pharmaceutical Biosciences between 1999-2006, and in 2006 he was appointed as Dean at the Faculty of Pharmacy at Uppsala University.

Dr. Nyberg’s field of expertise is neuropharmacology with particular emphasis on neuroendocrine and peptidergic mechanisms in chronic pain and drug dependence. He has over 350 publications in internationally-recognized industry and research journals, and his recent honors include the reward Sigillum of the Ateneo at University of Bologna in 2005, as well as appointments as Karl Johan Öbrink Lecturer at BMC in 2007, and Visiting Professor at Hoshi University, Tokyo (Japan) in 2009.

Recent Publications

Nehlin C, Nyberg F, Oster C.The Patient’s Perspective on the Link Between ADHD and Substance Use: A Qualitative Interview Study. J Atten Disord. 2014 Oct 30. pii: 1087054714554618. [Epub ahead of print] PMID: 25359762.

Heddini U, Bohm-Starke N, Grönbladh A, Nyberg F, Nilsson KW, Johannesson U. Serotonin receptor gene (5HT-2A) polymorphism is associated with provoked vestibulodynia and comorbid symptoms of pain. J Sex Med. 2014 Dec;11(12):3064-71. doi: 10.1111/jsm.12685. Epub 2014 Sep 1. PMID: 25174699.

Nyberg F. Structural plasticity of the brain to psychostimulant use. Neuropharmacology. 2014 Dec;87:115-24. doi: 10.1016/j.neuropharm.2014.07.004. Epub 2014 Jul 11. PMID: 25018041.

Carlsson-Jonsson A, Gao T, Hao JX, Fransson R, Sandström A, Nyberg F, Wiesenfeld-Hallin Z, Xu XJ. N-terminal truncations of substance P 1-7 amide affect its action on spinal cord injury-induced mechanical allodynia in rats. Eur J Pharmacol. 2014 Sep 5;738:319-25. doi: 10.1016/j.ejphar.2014.05.060. Epub 2014 Jun 13. PMID: 24933646.

Grönbladh A, Johansson J, Nyberg F, Hallberg M. Administration of growth hormone and nandrolone decanoate alters mRNA expression of the GABAB receptor subunits as well as of the GH receptor, IGF-1, and IGF-2 in rat brain. Growth Horm IGF Res. 2014 Apr-Jun;24(2-3):60-6. doi: 10.1016/j.ghir.2014.01.002. Epub 2014 Jan 16. PMID: 24480470.

Pope HG Jr, Wood RI, Rogol A, Nyberg F, Bowers L, Bhasin S. Adverse health consequences of performance-enhancing drugs: an Endocrine Society scientific statement. Endocr Rev. 2014 Jun;35(3):341-75. doi: 10.1210/er.2013-1058. Epub 2013 Dec 17. Review. PMID: 24423981.

Ali MA, Kazzam E, Amir N, Nyberg F, Adem A. Effects of dehydration and blockade of angiotensin II AT1 receptor on stress hormones and anti-oxidants in the one-humped camel. BMC Vet Res. 2013 Nov 19;9:232. doi: 10.1186/1746-6148-9-232. PMID: 24252635.

Hallberg M, Nyberg F. [Still unclear whether steroids induces addiction on its own]. Lakartidningen. 2013 Sep 25-Oct 8;110(39-40):1736-9. Review. Swedish. PMID: 24245429.

Righard L, Carlsson-Jonsson A, Nyberg F. Enhanced levels of immunoreactive β-casomorphin-8 in milk of breastfeeding women with mastitis. Peptides. 2014 Jan;51:54-8. doi: 10.1016/j.peptides.2013.10.027. Epub 2013 Nov 1. PMID: 24189037.

Watanabe H, Fitting S, Hussain MZ, Kononenko O, Iatsyshyna A, Yoshitake T, Kehr J, Alkass K, Druid H, Wadensten H, Andren PE, Nylander I, Wedell DH, Krishtal O, Hauser KF, Nyberg F, Karpyak VM, Yakovleva T, Bakalkin G. Asymmetry of the endogenous opioid system in the human anterior cingulate: a putative molecular basis for lateralization of emotions and pain. Cereb Cortex. 2015 Jan;25(1):97-108. doi: 10.1093/cercor/bht204. Epub 2013 Aug 19. PMID: 23960211 .

Fransson R, Sköld C, Kratz JM, Svensson R, Artursson P, Nyberg F, Hallberg M, Sandström A. Constrained H-Phe-Phe-NH2 analogues with high affinity to the substance P 1-7 binding site and with improved metabolic stability and cell permeability. J Med Chem. 2013 Jun 27;56(12):4953-65. doi: 10.1021/jm400209h. Epub 2013 Jun 17. PMID: 23735006.

Nyberg F, Hallberg M. Growth hormone and cognitive function. Nat Rev Endocrinol. 2013 Jun;9(6):357- 65. doi: 10.1038/nrendo.2013.78. Epub 2013 Apr 30. Review. PMID: 23629538.

Adem A, Al Haj M, Benedict S, Yasin J, Nagelkerke N, Nyberg F, Yandle TG, Frampton CM, Lewis LK, Nicholls MG, Kazzam E. ANP and BNP responses to dehydration in the one-humped camel and effects of blocking the renin-angiotensin system. PLoS One. 2013;8(3):e57806. doi: 10.1371/journal.pone.0057806. Epub 2013 Mar 13. PMID: 23516417.

Rhodin A, Grönbladh A, Ginya H, Nilsson KW, Rosenblad A, Zhou Q, Enlund M, Hallberg M, Gordh T, Nyberg F. Combined analysis of circulating β-endorphin with gene polymorphisms in OPRM1, CACNAD2 and ABCB1 reveals correlation with pain, opioid sensitivity and opioid-related side effects. Mol Brain. 2013 Feb 12;6:8. doi: 10.1186/1756-6606-6-8. PMID: 23402298.

Dominguez CA, Kalliomäki M, Gunnarsson U, Moen A, Sandblom G, Kockum I, Lavant E, Olsson T, Nyberg F, Rygh LJ, Røe C, Gjerstad J, Gordh T, Piehl F. The DQB1 *03:02 HLA haplotype is associated with increased risk of chronic pain after inguinal hernia surgery and lumbar disc herniation. Pain. 2013 Mar;154(3):427-33. doi: 10.1016/j.pain.2012.12.003. Epub 2012 Dec 13. PMID: 23318129.

Enhamre-Brolin E, Carlsson A, Hallberg M, Nyberg F. Growth hormone reverses streptozotocin-induced cognitive impairments in male mice. Behav Brain Res. 2013 Feb 1;238:273-8. doi: 10.1016/j.bbr.2012.10.036. Epub 2012 Nov 1. PMID: 23124136.

Grönbladh A, Johansson J, Nöstl A, Nyberg F, Hallberg M. GH improves spatial memory and reverses certain anabolic androgenic steroid-induced effects in intact rats. J Endocrinol. 2013 Jan 2;216(1):31-41. doi: 10.1530/JOE-12-0315. Print 2013 Jan. PMID: 23092877.

Johansson J, Grönbladh A, Nyberg F, Hallberg M. Application of in vitro [35S]GTPγ-S autoradiography in studies of growth hormone effects on opioid receptors in the male rat brain. Brain Res Bull. 2013 Jan;90:100-6. doi: 10.1016/j.brainresbull.2012.09.008. Epub 2012 Oct 11. PMID: 23063719.

Isaksson J, Nilsson KW, Nyberg F, Hogmark A, Lindblad F. Cortisol levels in children with attention-deficit/hyperactivity disorder. J Psychiatr Res. 2012 Nov;46(11):1398-405. doi: 10.1016/j.jpsychires.2012.08.021. Epub 2012 Sep 10. PMID:22974590.

Dr. Robert Clarke, Ph.D., D.Sc.

Robert Clarke, Ph.D., D.Sc. is an internationally recognized leader in breast cancer research. Dr. Clarke is the Dean for Research and Professor of Oncology at Georgetown University Medical Center. His research interests include understanding the cellular and molecular mechanisms driving antiestrogen resistance and hormone independence in breast cancer. He also is interested in drug resistance (cytotoxic, endocrine), drug/hormone interactions, and the application of molecular profiling (e.g., transcriptome, proteome, tissue array analyses) to predict breast cancer phenotypes and identify functionally relevant gene signaling networks. As an expert in cancer systems biology, Dr. Clarke leads several multinational molecular medicine studies in breast cancer, in collaboration with colleagues at the Georgetown Lombardi Comprehensive Cancer Center, Virginia Tech, and the University of Edinburgh, Scotland. He has served as chair of peer-review study sections for NIH and the Department of Defense, and currently serves on the editorial boards of over a dozen international peer review journals. Dr. Clarke is a Fellow of the Royal Society of Chemistry, a Fellow of the Royal Society of Medicine, and a Fellow of the Royal Society of Biology in the U.K.

Dr. Ted Dawson MD, PhD

  • Leonard and Madlyn Abramson Professor of Neurodegenerative Diseases, Johns Hopkins University
  • Scientific Director, Institute for Cell Engineering, Johns Hopkins University School of Medicine
  • Co-Director, Neuroregeneration Program, Institute for Cell Engineering, Johns Hopkins University School of Medicine

Dr. Dawson is collaborating with AGT to develop a gene therapeutic to arrest the development of Parkinson’s disease (PD) and other neurological disorders.

The main focus of the Dawson Laboratory at Johns Hopkins University is to unravel the molecular basis of neurodegeneration, which parallels Dr. Dawson’s clinical interest in neurodegenerative diseases.

The laboratory uses genetic, cell biological and biochemical approaches to explore the pathogenesis of Parkinson’s disease (PD) and other neurologic disorders. They also investigate several discrete mechanisms involved in cell death, including the role of nitric oxide as an endogenous messenger, the function of poly (ADP-ribose) polymerase-1 and apoptosis inducing factor in cell death, and how endogenous cell survival mechanisms protect neurons from death.

There are five broad areas of investigation:

  1. The study of the mechanisms of neuronal cell death;
  2. Nitric oxide (NO) signaling ;
  3. The study of novel cell death and cell survival pathways;
  4. The study of the molecular basis of Parkinson’s disease;
  5. Testing innovative neuroprotective and neurorestorative strategies in PD patients.

Dr. Dawson and his team originally identified NO as a major player in neuronal cell death, and they are investigating NO-death and NO-survival signaling pathways. They have shown that poly (ADP-ribose) polymerase (PARP) is a major target of NO-mediated neuronal injury and that selective inhibitors or knockout of PARP are profoundly neuroprotective in animal models of stroke and PD.

They recently identified a novel caspase-independent pathway of programmed cell death and showed that apoptosis inducing factor (AIF) is a critical cell death effector that acts downstream of NO/PARP. Current studies are focusing on the molecular mechanisms and identification of downstream targets of AIF’s actions and exploration of the role of other caspase-independent cell death effectors. The team is also investigating the mechanism by which PARP activation triggers AIF release and AIF kills cells.

PD is a common disorder of the nervous system that afflicts patients later in life with tremor, slowness of movement, gait instability, and rigidity. Loss of dopamine neurons accounts for the major signs and symptoms of PD, and mutations in at least five genes including a-synuclein, parkin, PINK1, DJ-1 and LRRK2 are responsible for rare Mendelian forms of PD. In addition to the progressive loss of dopamine neurons, PD is characterized by neurodegeneration throughout the central nervous system and by the accumulation of a-synuclein and other proteins in structures called Lewy bodies and Lewy neurites. Despite genetic advances in our understanding of PD, it is primarily considered a sporadic disorder with no known cause. Current evidence suggests that mitochondrial complex I abnormalities may be one of the major contributors to sporadic PD. Much as the discovery of dopamine deficiency led to potent treatments for motor symptoms, we believe that recent discoveries concerning the role of specific genes in Parkinson disease pathology will lead to the next revolution in disease therapy. Accordingly, the role of these genes in the pathogenesis of PD; and how mitochondrial complex I deficiency potentially leads to pathologic derangements in the function of these proteins has become a major focus of the Dawson Laboratory.

The team is studying the genetic basis of PD by investigating the mechanisms by which mutations in familial-linked genes cause PD. Mutations in a-synuclein or LRRK2 cause autosomal dominant PD, and mutations in parkin, PINK1 or DJ-1 cause autosomal recessive PD. The team found that parkin is an ubiquitin E3 protein ligase and that disease-causing mutations inhibit its E3 activity. They identified CDCrel-1 and synphilin-1 as parkin substrates and have shown that parkin’s E3 ligase activity may be important in the formation of Lewy bodies, the pathologic hallmark of PD. Mutations in parkin are a risk factor in sporadic PD. The team discovered that S-nitrosylation of parkin impairs its function. This discovery links the more common sporadic form of PD with alterations in parkin function. To assess the role of parkin, PINK1, and DJ-1 in PD pathogenesis in vivo, the team has knocked out DJ-1, PINK1, LRRK2 and parkin, and they are identifying and characterizing protein targets of parkin and the biologic function of LRRK2, DJ-1 and PINK1.

They are interested in genes essential for cell survival. To this end, they have been studying the role of NO as a survival molecule. Taking advantage of their findings that NO plays an important role in activity-dependent neuroprotection, they focused on identifying genes that are regulated by NO’s activation of Ras, and more than 30 candidate neuroprotective genes have been identified. Understanding the mechanism by which these proteins regulate neuronal survival may lead to the identification of innovative therapies for the treatment of neurologic disorders.

Selected Publications

Andrabi SA, Umanah GK, Chang C, Stevens DA, Karuppagounder SS, Gagné JP, Poirier GG, Dawson VL, Dawson TM. Poly(ADP-ribose) polymerase-dependent energy depletion occurs through inhibition of glycolysis. Proc Natl Acad Sci U S A.2014 Jul 15;111(28):10209-14. doi: 10.1073/pnas.1405158111. Epub 2014 Jul 1. PMID: 24987120

Sagal J, Zhan X, Xu J, Tilghman J, Karuppagounder SS, Chen L, Dawson VL, Dawson TM, Laterra J, Ying M. Proneural transcription factor atoh1 drives highly efficient differentiation of human pluripotent stem cells into dopaminergic neurons. Stem Cells Transl Med. 2014 Aug;3(8):888-98. doi: 10.5966/sctm.2013-0213. Epub 2014 Jun 5. PMID: 24904172

Chen YC, Umanah GK, Dephoure N, Andrabi SA, Gygi SP, Dawson TM, Dawson VL, Rutter J. Msp1/ATAD1 maintains mitochondrial function by facilitating the degradation of mislocalized tail-anchored proteins. EMBO J. 2014 Jul 17;33(14):1548-64. doi: 10.15252/embj.201487943. Epub 2014 May 19. PMID: 24843043 [PubMed – in process]

Karuppagounder SS, Brahmachari S, Lee Y, Dawson VL, Dawson TM, Ko HS. The c-Abl inhibitor, nilotinib, protects dopaminergic neurons in a preclinical animal model of Parkinson’s disease. Sci Rep. 2014 May 2;4:4874. doi: 10.1038/srep04874. PMID: 24786396 [PubMed – in process]

Chi Z, Byrne ST, Dolinko A, Harraz MM, Kim MS, Umanah G, Zhong J, Chen R, Zhang J, Xu J, Chen L, Pandey A, Dawson TM, Dawson VL. Botch is a γ-glutamyl cyclotransferase that deglycinates and antagonizes Notch. Cell Rep. 2014 May 8;7(3):681-8. doi: 10.1016/j.celrep.2014.03.048. Epub 2014 Apr 24. PMID: 24767995

Chung, K.K.K, Y. Zhang, K. L. Lim, Y. Tanaka, H. Huang, J. Gao, C. A. Ross, V. L. Dawson and T. M. Dawson. “Parkin Ubiquitinates the a-Synuclein-Interacting Protein, Synphilin-1: Implications for Lewy Body Formation in Parkinson Disease.” Nature Medicine, 7:1144-1150 (2001). Yu, S.-W., H.-M. Wang, M.F. Poitras, C. Coombs, W.J. Bowers, H.J. Federoff, Guy G. Poirer, T.M. Dawson, V.L. Dawson “Mediation of PARP-1 Mediated Cell Death by Apoptosis Inducing Factor.” Science, 297: 259-263 (2002).

Dawson, T.M. and V.L. Dawson. “Molecular Pathways of Neurodegeneration in Parkinson’s Disease.” Science, 302: 819-822 (2003)

Chung, K.K.K., B. Thomas, X. Li, O. Pletnikova, J.C. Troncoso, L. Marsh, V. L. Dawson and T.M. Dawson. “S-Nitrosylation of Parkin Regulates Ubiquitination and Compromises Parkin’s Protective Function.” Science, 304:1328-1331 (2004).

Von Coelln, R., B. Thomas, J. M. Savitt, K.L. Lim, M. Sasaki, E. Hess, V.L. Dawson, T.M. Dawson. “Loss of Locus Coeruleus Neurons and Reduced Startle in Parkin Null Mice.” Proc. Natl. Acad. Sci. U.S.A., 101:10744-10749 (2004).

See his research profile at Ted Dawson Research Profile.

Julia Brown

Director

Julia Brown has extensive experience in development-stage pharmaceutical/biopharmaceutical and commercial-stage specialty pharmaceutical companies. She has served on the board of directors of nine drug development companies with a focus on governance and compensation. She currently serves on the board and as a member of the compensation and nominating and governance committees of Albireo Pharma, Inc., a publicly traded company with a focus in orphan pediatric liver diseases as well as other liver and gastrointestinal diseases and disorders. Her prior experience includes C-level executive roles with functional expertise in commercial operations, business and corporate development, partnering, strategic planning and general management. Julia earned her pharmaceutical industry acumen through 25 years with Eli Lilly in progressively more senior roles ranging from research to general manager of a product division.

Qi Tang

Director

Qi Tang has developed manufacturing and import/export businesses throughout China and the United States and has invested several times throughout AGT’s growth.  Ms. Tang is a real estate investor, entrepreneur, and financial investor who brings contacts and significant business expertise in her position as Director.

Aaron Galvin

Director

Aaron Galvin earned his BS and MS from the Massachusetts Institute of Technology (MIT), joining the MIT Lincoln Labs to pioneer innovations to radar as well as developing technologies for various classified national security projects.  After twelve years of US Government service, Mr. Galvin was recruited as Vice President of Research and Development for Aritech, a private, emerging electronics company, where he developed unique analog signal processing technology, resulting in nearly 150 patents and allowing his company to become the predominant, worldwide supplier of ultrasonic motion detectors for commercial and home security systems.  Aritech was acquired by ADT (American District Telegraph).  Mr. Galvin was retained by ADT as Vice President of Advanced Development.  Mr. Galvin founded ADT’s New England Research Laboratories (NERL), which he led until his retirement.  Mr. Galvin remains active in retirement, spending time inventing solutions to real-world problems that catch his interest and attention.

Tommy Thompson

Director and Senior Advisor

Tommy Thompson, former Secretary of the United States Department of Health and Human Services, worked fervently to modernize healthcare delivery as the former Governor of Wisconsin. An advocate for better health, Thompson has been at the forefront of technology and healthcare solutions. He is a leader in the fight against HIV/AIDS and brings nearly 30 years of experience solving complex health challenges to the AGT board, where he will guide investment, research and commercialization strategies. Previously, he worked as partner at Akin Gump law firm and was chairman of Deloitte’s global healthcare practice.

Dr. Steven Dobrowolski, PhD

Associate Professor of Pathology, University of Pittsburgh School of Medicine

  • Dujols V, Kusukawa N, McKinney JT, Dobrowolski SF, Wittwer CT (2006) High-resolution melting analysis for scanning and genotyping., in Real Time PCR. Tevfik D, ed.
  • Dobrowolski SF, E Ellingson C, Caldovic L, Tuchman M. (2007) Streamlined assessment of gene variants by high resolution melt profiling utilizing the ornithine transcarbamylase gene as a model system. Hum Mut. 28(11):1133-1140
  • Dobrowolski SF, Ellingson C, Coyne T, Grey J, Martin R, Naylor EW, Koch R, Levy HL. (2007) Mutations in the phenylalanine hydroxylase gene identified in 95 patients with phenylketonuria using novel systems of mutation scanning and specific genotyping based upon thermal melt profiles. Mol Gen Metab. 91(3):218-227.
  • Gundry CN, Dobrowolski SF, Martin YR, Robbins TC, Nay LM, Boyd N, Coyne T, Wall MD, Wittwer CT, Teng DH. (2008) Base-pair neutral homozygotes can be discriminated by calibrated high-resolution melting of small amplicons. Nucleic Acids Res. 36(10):3401-3408.
  • Mitchel S, Dobrowolski SF, Tuchman M, Coyne T, Summar M. (2009) Genetic Variation in the Urea Cycle: A Model Resource for Investigating Key Candidate Genes for Common Diseases. Hum Mut. 30(1):56-60.
  • Dobrowolski SF, Pey AL, Koch R, Levy HL, Ellingson C, Naylor EW, Martinez A. (2009) Biochemical characterization of mutant phenylalanine hydroxylase enzymes and correlate to clinical presentation in hyperphenylalanemic patients. J Inherited Met Dis 32;1-21.
  • Kaput J, Cotton RGH, Hardman L, Dobrowolski SF et al. (2009) Planning the Human Variome Project: The Spain Report. Hum Mut. 30:4, 496–510.
  • Dobrowolski SF, Borski K, Ellingson C; Koch R, Levy H, Naylor EW. (2009) A limited spectrum of severe phenylalanine hydroxylase mutations observed in phenylketonuria patients in western Poland and the implications to treatment with 6R tetrahydrobiopterin. J Hum Genet. 54(6):335-9
  • Dobrowolski SF, Gray J, Ellingson C. (2009) Identifying sequence variants in the human mitochondrial genome using high resolution melting for variant scanning and specific genotyping. Hum Mut. 30:6, 891–898
  • Dobrowolski SF, Andersen HS, Andresen BS (2010) The PAH c.30C>G synonymous variation is a pathological mutation that functions by creating a common splicing silencer motif. Mol Gen Metab. 100:4, 316-23.
  • Olsen RJ, Dobrowolski SF, Kjeldsen M, Hougaard D, Simonsen H, Gregersen N, Andresen BS. (2010) High-resolution melting analysis, a simple and effective method for reliable mutation scanning and frequency studies in the ACADVL gene. J Inherited Met Dis. 33:3, 247-60.
  • Dobrowolski SF, Heintz C, Miller T, Ellingson C, Ellingson C, Ozer I, Gokcay G, Baykal T, Thony B, Demirkol M, Blau N (2011) Molecular Genetics and Impact of residual Phenylalanine Hydroxylase Activity on Tetrahydrobiopterin-Responsiveness in Turkish PKU Population. Mol Gen Metab. 102:2 116-21.

Dr. Jerry Vockley, MD, PhD

  • Division Chief of Medical Genetics at Children’s Hospital of Pittsburgh of the University of Pittsburgh Medical Center
  • Cleveland Family Professor of Pediatric Research at the University of Pittsburgh School of Medicine
  • Distinguished Editor, NIH Director’s New Innovator Award Editorial Board
  • Associate Editor of Molecular Genetics and Metabolism

Collaborating with AGT on Phenylketonuria (PKU)

Dr. Jerry Vockley is a highly respected thought leader in the field of genetic research as a result of his internationally acclaimed work in medical genetics and inborn errors of metabolism. He has published more than 185 scientific articles in peer-reviewed journals. His laboratory has been responsible for identifying and characterizing the molecular basis of multiple new inborn errors of metabolism.  The National Institutes of Health has awarded Dr. Vockley continuous funding to support his important work since 1992.

After receiving his bachelor’s degree in biology at Carnegie-Mellon University, Dr. Vockley went on to receive his medical degree and doctorate in Genetics from the University of Pennsylvania School of Medicine.  He completed his pediatric residency at the University of Colorado Health Science Center, and his postdoctoral fellowship in Human Genetic and Pediatrics at Yale University School of Medicine in New Haven, Connecticut. Before assuming his current position in Pittsburgh, Dr. Vockley was Chair of Medical Genetics in the Mayo Clinic School of Medicine.

Dr. Vockley is board-certified in pediatrics, clinical genetics and biochemical/molecular genetics. He has has received numerous honors for his work. His professional and scientific society memberships include the American Society for Clinical Investigation, Society for Inherited Metabolic Disorders, American Society of Human Genetics, American Academy of Pediatrics, American Association for the Advancement of Science and the Society for the Study of Inborn Errors of Metabolism. He is the past president of the Society for Inherited Metabolic Disorders and the International Congress on Inborn Errors of Metabolism

Selected Publications

Wang Y, Mohsen AW, Mihalik SJ, Goetzman ES, Vockley J. (2010). Evidence for physical association of mitochondrial fatty acid oxidation and oxidative phosphorylation complexes. The Journal of biological chemistry. 285: 29834-41. PMC2943265.

Wolfe LA, Morava E, He M, Vockley J, Gibson KM. (2012). Heritable disorders in the metabolism of the dolichols: A bridge from sterol biosynthesis to molecular glycosylation. Am J Med Genet C Semin Med Genet. 160C: 322-8.

Mazariegos G, Shneider B, Burton B, Fox IJ, Hadzic N, Kishnani P, Morton DH, McIntire S, Sokol RJ, Summar M, White D, Chavanon V, Vockley J. (2014). Liver transplantation for pediatric metabolic disease. Mol Genet Metab.111: 418-27. 24495602.

Vockley J, Andersson HC, Antshel KM, Braverman NE, Burton BK, Frazier DM, Mitchell J, Smith WE, Thompson BH, Berry SA. (2014). Phenylalanine hydroxylase deficiency: diagnosis and management guideline. Genet Med.16: 188-200. 24385074.

Schiff M, Haberberger B, Xia C, Mohsen AW, Goetzman ES, Wang Y, Uppala R, Zhang Y, Karunanidhi A, Prabhu D, Alharbi H, Prochownik EV, Haack T, Haberle J, Munnich A, Rotig A, Taylor RW, Nicholls RD, Kim JJ, Prokisch H, Vockley J. (2015). Complex I assembly function and fatty acid oxidation enzyme activity of ACAD9 both contribute to disease severity in ACAD9 deficiency. Hum Mol Genet. 24: 3238-47. 4424958.

Vockley J, Marsden D, McCracken E, DeWard S, Barone A, Hsu K, Kakkis E. (2015). Long-term major clinical outcomes in patients with long chain fatty acid oxidation disorders before and after transition to triheptanoin treatment-A retrospective chart review. Mol Genet Metab. 116: 53-60. PMC4561603.

Dr. Dean Felsher, MD, PhD

  • Stanford University School of Medicine
  • Director, Stanford Translational Research and Applied Medicine Program
  • Professor of Medicine (Oncology) and Pathology

Collaborating with AGT on Liver Cancer

Dr. Dean W. Felsher is currently a Professor in the Division of Oncology, Departments of Medicine and Pathology at Stanford University. He obtained his B.A. in Chemistry at the University of Chicago and his MD PhD from the University of California, Los Angeles. He conducted his Oncology Fellowship training at the University of California San Francisco.

Dr. Felsher’s laboratory pursues both basic science and translational research studies investigating how oncogenes initiate and sustain tumorigenesis and uses these insights towards the development of new therapies for cancer. He has pioneered strategies utilizing the Tetracycline regulatory System to develop conditional transgenic mouse model systems of tumorigenesis to address three questions:

  • How does oncogene activation initiate tumorigenesis?
  • When does oncogene inactivation reverse tumorigenesis?
  • What are the mechanisms by which tumors escape dependence upon oncogenes?

To date, he established a paradigm suggesting that the inactivation of the single oncogene, MYC, can be sufficient to reverse the process of tumorigenesis, in lymphoma, leukemia, osteogenic sarcoma and hepatocellular carcinoma. Even genomically complex tumors were found to regress upon MCY inactivation. Moreover, he has shown that MYC oncogene inactivation uncovers pluripotent differentiation of tumor cells into normal cellular lineages. In some cases, this differentiation results in the permanent loss of a neoplastic phenotype and even brief MYC inactivation induces sustained tumor regression. In other cases, some of the tumor cells appear to retain the capacity for neoplastic features upon oncogene reactivation, hence existing in a state of tumor dormancy

The experimental model system that he has developed has contributed to several new principles of tumor biology, including the notion of the reversibility of cancer, the idea that oncogene inactivation can result in the uncovering of stem cell features of tumors, the idea that oncogene inactivation can either revoke tumorigenesis or induce a state of tumor dormancy, the notion that cellular senescence programs and angiogenic pathways play an important role in the mechanism of oncogene addiction, and finally, the role of the immune system in contributing to many of these mechanisms of oncogene addiction.

Dr. Felsher’s work has led to major contributions to our understanding of the molecular basis of cancer and useful towards the rational development of new therapeutics to treat cancer.

Selected Publications

Zhongwei Cao, Hua Fan-Minogue, David I. Bellovin, Aleksey Yevtodiyenko, Julia Arzeno, Qiwei Yang, Sanjiv Sam Gambhir, and Dean W. Felsher. ‘MYC Phosphorylation, Activation, and Tumorigenic Potential in Hepatocellular Carcinoma are Regulated by HMG-CoA Reductase‘; Cancer Res. Mar 15, 2011; 71(6): 2286–2297. 10.1158/0008-5472.CAN-10-3367

Li Y, Choi PS, Felsher DW. Oncogene addiction: resetting the safety switch? Oncotarget. 2014 Sep 20. [Epub ahead of print] PubMed PMID: 25275297.

Li Y, Choi PS, Casey SC, Felsher DW. Activation of cre recombinase alone can induce complete tumor regression. PLoS One. 2014 Sep 10;9(9):e107589. doi: 10.1371/journal.pone.0107589. eCollection 2014. PubMed PMID: 25208064; PubMed Central PMCID: PMC4160265.

Li Y, Choi PS, Casey SC, Dill DL, Felsher DW. MYC through miR-17-92 suppresses specific target genes to maintain survival, autonomous proliferation, and a neoplastic state. Cancer Cell. 2014 Aug 11;26(2):262-72. doi: 10.1016/j.ccr.2014.06.014. PubMed PMID: 25117713.

Casey SC, Li Y, Fan AC, Felsher DW. Oncogene withdrawal engages the immune system to induce sustained cancer regression. J Immunother Cancer. 2014 Jul 15;2:24. doi: 10.1186/2051-1426-2-24. eCollection 2014. Review. PubMed PMID: 25089198; PubMed Central PMCID: PMC4118610.

Choi PS, Li Y, Felsher DW. Addiction to multiple oncogenes can be exploited to prevent the emergence of therapeutic resistance. Proc Natl Acad Sci U S A. 2014Aug 12;111(32):E3316-24. doi: 10.1073/pnas.1406123111. Epub 2014 Jul 28. PubMed PMID: 25071175; PubMed Central PMCID: PMC4136575.

Eberlin LS, Gabay M, Fan AC, Gouw AM, Tibshirani RJ, Felsher DW, Zare RN. Alteration of the lipid profile in lymphomas induced by MYC overexpression. Proc Natl Acad Sci U S A. 2014 Jul 22;111(29):10450-5. doi: 10.1073/pnas.1409778111. Epub 2014 Jul 3. PubMed PMID: 24994904; PubMed Central PMCID: PMC4115527.

Gabay M, Li Y, Felsher DW. MYC activation is a hallmark of cancer initiation and maintenance. Cold Spring Harb Perspect Med. 2014 Jun 2;4(6). pii: a014241. doi: 10.1101/cshperspect.a014241. PubMed PMID: 24890832.

Ye D, Shuhendler AJ, Cui L, Tong L, Tee SS, Tikhomirov G, Felsher DW, Rao J. Bioorthogonal cyclization-mediated in situ self-assembly of small-molecule probes for imaging caspase activity in vivo. Nat Chem. 2014 Jun;6(6):519-26. doi: 10.1038/nchem.1920. Epub 2014 Apr 28. PubMed PMID: 24848238; PubMed Central PMCID: PMC4031611.

Casey SC, Li Y, Felsher DW. An essential role for the immune system in the mechanism of tumor regression following targeted oncogene inactivation. Immunol Res. 2014 May;58(2-3):282-91. doi: 10.1007/s12026-014-8503-6. PubMed PMID: 24791942.

Li Y, Casey SC, Felsher DW. Inactivation of MYC reverses tumorigenesis. J Intern Med. 2014 Jul;276(1):52-60. doi: 10.1111/joim.12237. Review. PubMed PMID: 24645771; PubMed Central PMCID: PMC4065197.

Cao Z, Ding BS, Guo P, Lee SB, Butler JM, Casey SC, Simons M, Tam W, Felsher DW, Shido K, Rafii A, Scandura JM, Rafii S. Angiocrine factors deployed by tumor vascular niche induce B cell lymphoma invasiveness and chemoresistance. Cancer Cell. 2014 Mar 17;25(3):350-65. doi: 10.1016/j.ccr.2014.02.005. PubMed PMID: 24651014; PubMed Central PMCID: PMC4017921.

Nwabugwu CI, Rakhra K, Felsher DW, Paik DS. A tumor-immune mathematical model of CD4+ T helper cell dependent tumor regression by oncogene inactivation. Conf Proc IEEE Eng Med Biol Soc. 2013;2013:4529-32. doi: 10.1109/EMBC.2013.6610554. PubMed PMID: 24110741.

Rakhra K, Felsher DW. Generation of a tetracycline regulated mouse model of MYC-induced T-cell acute lymphoblastic leukemia. Methods Mol Biol. 2013;1012:221-35. doi: 10.1007/978-1-62703-429-6_15. PubMed PMID: 24006068.

Ansari C, Tikhomirov GA, Hong SH, Falconer RA, Loadman PM, Gill JH, Castaneda R, Hazard FK, Tong L, Lenkov OD, Felsher DW, Rao J, Daldrup-Link HE. Development of novel tumor-targeted theranostic nanoparticles activated by membrane-type matrix metalloproteinases for combined cancer magnetic resonance imaging and therapy. Small. 2014 Feb 12;10(3):566-75, 417. doi: 10.1002/smll.201301456. Epub 2013 Aug 27. PubMed PMID: 24038954; PubMed Central PMCID: PMC3946335.

Fan AC, O’Rourke JJ, Praharaj DR, Felsher DW. Real-time nanoscale proteomic analysis of the novel multi-kinase pathway inhibitor rigosertib to measure the response to treatment of cancer. Expert Opin Investig Drugs. 2013 Nov;22(11):1495-509. doi: 10.1517/13543784.2013.829453. Epub 2013 Aug 12. Review. PubMed PMID: 23937225; PubMed Central PMCID: PMC4111569.

Casey SC, Bellovin DI, Felsher DW. Noncanonical roles of the immune system in eliciting oncogene addiction. Curr Opin Immunol. 2013 Apr;25(2):246-58. doi: 10.1016/j.coi.2013.02.003. Epub 2013 Apr 6. Review. PubMed PMID: 23571026; PubMed Central PMCID: PMC3683588.

Das, B., Kashino, S. S., Pulu, I., Kalita, D., Swami, V., Yeger, H., Felsher, D. W., Campos-Neto, A. “CD271(+) bone marrow mesenchymal stem cells may provide a niche for dormant Mycobacterium tuberculosis”. Science translational medicine.2013; 5 (170): 170ra13-?

Tran, P. T., Shroff, E. H., Burns, T. F., Thiyagarajan, S., Das, S. T., Zabuawala, T., Chen, J., Cho, Y., Luong, R., Tamayo, P., Salih, T., Aziz, K., Adam, S. J., Vicent, S., Nielsen, C. H., Withofs, N., Sweet-Cordero, A., Gambhir, S. S., Rudin, C. M., Felsher, D. W. “Twist1 Suppresses Senescence Programs and Thereby Accelerates and Maintains Mutant Kras-Induced Lung Tumorigenesis”. PLOS GENETICS 2012; 8 (5)

Bachireddy, P., Rakhra, K., Felsher, D. W. “Immunology in the clinic review series; focus on cancer: multiple roles for the immune system in oncogene addiction”. CLINICAL AND EXPERIMENTAL IMMUNOLOGY 2012; 167 (2): 188-194

DeChristopher, B. A., Fan, A. C., Felsher, D. W., Wender, P. A. “’Picolog,’ a Synthetically-Available Bryostatin Analog, Inhibits Growth of MYC-Induced Lymphoma In Vivo” ONCOTARGET 2012; 3 (1): 58-66

Choi, P. S., van Riggelen, J., Gentles, A. J., Bachireddy, P., Rakhra, K., Adam, S. J., Plevritis, S. K., Felsher, D. W. “Lymphomas that recur after MYC suppression continue to exhibit oncogene addiction”. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA 2011; 108 (42): 17432-17437

Dr. Shyam Kottilil, PhD

Education and Training

  • 1991, MD – Medicine, Medical College, Trichur
  • 1998, PhD – Immunology, Memorial University, Newfoundland
  • 2000, Resident – Internal Medicine, Brown University, Providence, Rhode Island
  • 2003, Fellow – Infectious Diseases, National Institutes of Health, Bethesda, Maryland

Dr. Shyam Kottilil MD. Ph.D. is the Associate Director of the Division of Clinical Care and Research at the Institute of Human Virology (IHV), University of Maryland, Baltimore MD. Dr. Kottilil also serves as the Director of the Clinical Trials Unit, Division of Infectious Diseases at the IHV. Dr. Kottilil continues to work as the Scientific Director of the NIH-District of Columbia Program for AIDS Progress. He received his medical degree from Govt Medical College, Trichur, India, Ph.D (immunology of HIV infection) from Memorial University of Newfoundland, Canada, completed a residency in Internal Medicine from Brown University, Rhode Island and a fellowship in infectious diseases rom the National Institutes of Health under Dr. Anthony S. Fauci.

He has conducted several pivotal investigator initiated studies in hepatitis C and has authored over 200 peer-reviewed publications. Dr. Kottilil serves as one of the founding members of the US National hepatitis C treatment guidance panel. He is a Fellow of the American College of Physician (FACP), Infectious Diseases Society of America (FIDSA). He has received numerous awards including National Institute of Allergy and Infectious Diseases (NIAID) Meritorious service award for Innovations in Science (2014) and NIH Outstanding Mentor award (2013). Dr. Kottilil has mentored over 30 MDs and Ph.Ds during his academic career.

Highlighted Publications

  1. Kottilil S, Polis MA, Kovacs JA. HIV Infection, Hepatitis C infection, and HAART: Hard Clinical Choices. JAMA. (2004), 292:243-50.
  2. Moir S, Malaspina A, Pickeral OK, Donoghue ET, Vasquez J, Miller NJ, Krishnan SR, Planta MA, Turney JF, Justement JS, Kottilil S, Dybul M, Mican JM, Kovacs C, Chun TW, Birse CE, and Fauci AS. Survival of B Cells of HIV-viremic Patients Mediated by Altered Expression of Receptors of the TNF Super family. J Exp Med. (2004), 200:587-99.
  3. Fauci AS Mavilio D, and Kottilil S. NK cells in HIV infection: Paradigm for Protection or Targets for Ambush. Nat Rev Immunol. (2005), 5: 835-43.
  4. Arthos J, Cicala C, Martinelli E, Macleod K, Van Ryk D, Wei D, Xiao Z, Veenstra T, Conrad TP, Lempicki RA, Cruz C, Censoplano N, Chung E, Reitano KN, Kottilil S, Goode D, Fauci AS. The HIV-1 Envelope Binds to a4b7 integrin, the gut mucosal homing receptor. Nat Immunol. (2008), 9:301-309.
  5. Moir S, Ho J, Malaspina A, Wang W, DiPoto AC, O’Shea MA, Roby G, Kottilil S, Arthos J, Proschan MA, Chun TW, Fauci AS. Evidence for HIV-associated B cell exhaustion in a dysfunctional memory B cell compartment in HIV-infected viremic individuals. J Exp Med. (2008), 205:1797-805.
  6. Kottilil S., Yan MY Reitano KN, Zhang X, Lempicki RA, Roby G, Daucher M, Yang J, Ghany M, Polis MA, and Fauci AS. HIV and HCV infections induce distinct immunologic imprints with implications for the pathogenesis of liver disease and therapeutic response to interferon-alpha. Hepatology (2009), 50:34-45
  7. Hadigan C and Kottilil S. Hepatitis C virus infection and coinfection with human immunodeficiency virus: challenges and advancements in management. JAMA (2011), 306:294-301.
  8. Naggie S, Osinusi A, Katsounas A, Lempicki RA, Herrmann E, Schlaak JF, Shivakumar B, Masur H, Polis MA, Kottilil S. Dysregulation Of Host Innate Immunity In HIV/HCV Genotype 1 Infected IL28B CT/TT Haplotype Patients Associated With Depressed Viral Kinetics And Therapeutic Response. Hepatology (2012), 56:444-54.

Dr. Jose Bordon, MD, PhD, AAHIVS

Providence Hospital

Dr. Jose Bordon is a board certified infectious disease specialist, focusing on treatment of individuals with HIV infections. He offers patients high quality care in a professional and compassionate manner. Dr. Bordon earned his degree in medicine from National University of Cordoba Medical School of Argentina and completed his residency in internal medicine at George Washington University Medical Center/Providence Hospital in Washington, D.C. He completed his infectious disease fellowship at the University of Maryland Medical School in Baltimore, MD. Among his notable affiliations, Dr. Bordon is on the Editorial Board of Case Reports in Pulmonology and a Journal Reviewer for numerous journals, includingClinical Infectious Diseases. His areas of interest include HIV infections, viral Hepatitis, and Hepatitis C. “My desire is to help educate HIV-infected patients on the most current available treatments. Working together hand-in-hand with the patient, we can maximize a high quality, functional lifestyle.” – Jose Bordon, M.D., Ph.D., AAHIVS

Dr. John J. Rossi, PhD

  • Lidow Family Research Chair
  • Morgan & Helen Chu Dean’s Chair of the Irell & Manella Graduate School of Biological Sciences
  • Dean, Irell & Manella Graduate School of Biological Sciences
  • Chair and Professor, Department of Molecular and Cellular Biology
  • Associate Director for Education & Training, Comprehensive Cancer Center

Collaborating with AGT on HIV/AIDS

Dr. John J. Rossi is an internationally recognized leader in the field of molecular biology. A pioneer in clinical research with nucleic acids and in the development of RNA-based therapies for human disease, Dr. Rossi was involved in the first human trials testing genetically altered stem cells for cancer therapy—work that has already led to the discovery of new treatments.

Dr. Rossi received a BA in biology from the University of New Hampshire, and a PhD in microbial genetics from the University of Connecticut. Following a post-doctoral fellowship at Brown University Medical School, Dr. Rossi joined Southern California’s City of Hope (COH) Comprehensive Cancer Center as an assistant research scientist to apply his theoretical research to the search for new treatments for HIV, AIDS-related lymphoma, pancreatic cancer, and liver cancer. In 1992, he was named chairman of the Division of Biology and, in 1993, COH recognized his groundbreaking work at the molecular level in the battle against AIDS and other major diseases by naming him to its Gallery of Medical and Scientific Achievement. Dr. Rossi also received the American Association for Clinical Chemistry Outstanding Speaker Award in 1991, and was honored by the National Institute of Allergy and Infectious Diseases Division of AIDS with their Merit Award in 1992. For three years Dr. Rossi served as head of the department of gene therapy at Loma Linda University Medical Center, and currently holds an adjunct professorship at the University’s Department of Biochemistry and Microbiology.

In addition to holding leadership positions at a range of esteemed educational and biomedical research organizations, Dr. Rossi serves as editor for a variety of gene therapy and biomedical publications including Human Gene Therapy, Silence, JBC, and BMC Biotechnology, as well as publishing more than 250 scientific papers including seminal discoveries regarding therapeutic applications of small, regulatory RNA in cancer and infectious diseases.

Recent Publications

Takahashi, M., X. Wu, M. Ho, P. Chomchan, JJ. Rossi, JC. Burnett, and J. Zhou. “High throughput sequencing analysis of RNA libraries reveals the influences of initial library and PCR methods on SELEX efficiency”. Sci Rep, 2016. 22(6):33697. doi: 10.1038/srep33697.

Dao, P., J. Hoinka, M. Takahashi, J. Zhou, M. Ho, Y. Wang, F. Costa, JJ. Rossi, R. Backofen, J. Burnett, and TM. Przytycka. “AptaTRACE Elucidates RNA Sequence-Structure Motifs from Selection Trends in HT-SELEX Experiments”. Cell Syst, 2016. 3(1): p. 62-70. doi: 10.1016/j.cels.2016.07.003

Chen, C., P. Posocco, X. Liu, Q. Cheng, E. Laurini, J. Zhou, C. Liu, Y. Wang, J. Tang, VD. Col, T. Yu, S. Giorgio, M. Fermeglia, F. Qu, Z. Liang, JJ. Rossi, M. Liu, P. Rocchi, S. Pricl, and L. Peng. “siRNA Delivery: Mastering Dendrimer Self-Assembly for Efficient siRNA Delivery: From Conceptual Design to In Vivo Efficient Gene Silencing (Small 27/2016)”. Small, 2016. 12(27):3604. doi: 10.1001/smll.201670131

Chen, C., P. Posocco, X. Liu, Q. Cheng, E. Laurini, J. Zhou, C. Liu, Y. Wang, J. Tang, VD. Col, T. Yu, S. Giorgio, M. Fermeglia, F. Qu, Z. Liang, JJ. Rossi, M. Liu, P. Rocchi, S. Pricl, and L. Peng. “Mastering Dendrimer Self-Assembly for Efficient siRNA Delivery: From Conceptual Design to In Vivo Efficient Gene Silencing”. Small, 2016. 12(27): p. 3667-76. doi: 10.1002/smll.201503866

Yoon, S., KW. Huang, V. Reebye, P. Mintz, YW. Tien, HS. Lai, P. Sætrom, I. Reccia, P. Swiderski, B. Armstrong, A. Jozwiak, D. Spalding, L. Jiao, N. Habib, and JJ. Rossi. “Targeted Delivery of C/EBPα -saRNA by Pancreatic Ductal Adenocarcinoma-specific RNA Aptamers Inhibits Tumor Growth In Vivo”. Mol Ther, 2016. 24(6): p. 1106-16. doi: 10.1038/mt.2016.60

Zhou, J. and JJ. Rossi. “Evolution of Cell-Type-Specific RNA Aptamers Via Live Cell-Based SELEX”. Methods Mol Biol, 2016. 1421: p. 191-214. doi: 10.1007/978-1-4939-3591-8_16

Cui, Q., S. Yang, P. Ye, E. Tian, G. Sun, J. Zhou, G. Sun, X. Liu, C. Chen, K. Murai, C. Zhao, KT. Azizian, L. Yang, C. Warden, X. Wu, M. D’Apuzzo, C. Brown, B. Badie, L. Peng, AD. Riggs, JJ. Rossi, and Y. Shi. “Downregulation of TLX induces TET3 expression and inhibits glioblastoma stem cell self-renewal and tumorigenesis”. Na Commun., 2016. 3(7): 10637. doi: 10.1038/ncomms10637.

van den Berg, FT, JJ. Rossi, P. Arbuthnot, MS. Weinberg. “Design of Effective Primary MicroRNA Mimics With Different Basal Stem Conformations”. Mol Ther Nucleic Acids, 2016. 12(5): e278. doi: 10.1038/mtna.2016.53.

Bobbin, ML. and JJ. Rossi. “RNA Interference (RNAi)-Based Therapeutics: Delivering on the Promise?”. Annu Rev Pharmacol Toxicol, 2016. 56: p. 103-22. doi: 10.1146/annurev-pharmtox-010715-103633.

Jung, U., M. Takahashi, JJ. Rossi, and JC Burnett. “LGIT In Vitro Latency Model in Primary and T Cell Lines to Test HIV-1 Reactivation Compounds”. Methods Mol Biol., 2016. 1354: p. 255-64. doi: 10.1007/978-1-4939-3046-3_17.

Petz, LD., JC. Burnett, H. Li, S. Li, R. Tonai, M. Bakalinskaya, EJ. Shpall, S. Armitage, J. Kurtzberg, DM Regan, P. Clark, S. Querol, JA. Gutman, SR. Spellman, L. Gragert, and JJ. Rossi. “Progress toward curing HIV infection with hematopoietic cell transplantation”. Stem Cells Cloning, 2015. 28(8): p. 109-16. doi: 10.2147/SCCAA.S56050.

Jung, U., K. Urak, M. Veillette, ME. Nepveu-Traversy, QT. Pham, S. Hamel, JJ. Rossi, and L. Berthoux. “Preclinical Assessment of Mutant Human TRIM5α as an Anti-HIV-1 Transgene”. Hum Gene Ther, 2016. 26(10): p. 664-79. doi: 10.1089/hum.2015.059

Bobbin, ML., JC. Burnett, and JJ. Rossi. “RNA interference approaches for treatment of HIV-1 infection”. Genome Med, 2015. 7(1):50. doi: 10.1186/s13073-015-0174-y.

Li, Y., X. Wu, H. Gao, JM. Jin, AX. Li, YS. Kim, SK. Pal, RA. Nelson, CM. Lau, C. Guo, B. Mu, J. Wang, F. Wang, J. Wang, Y. Zhao, W. Chen, JJ. Rossi, LM. Weiss, and H. Wu. “Piwi-Interacting RNAs (piRNAs) Are Dysregulated in Renal Cell Carcinoma and Associated with Tumor Metastasis and Cancer-Specific Survival”. Mol Med, 2015. 13(21): p. 381-8. doi: 10.2119/molmed.2014.00203.

Zhou, J., JJ. Rossi, and KT. Shum. “Methods for assembling B-cell lymphoma specific and internalizing aptamer-siRNA nanoparticles via the sticky bridge”. Methods Mol Biol., 2015. 1297: p. 169-85. doi: 10.1007/978-1-4939-2562-9_12.

Rossi, JJ. “Twenty-five plus years of an RNA addiction”. RNA, 2015. 21(4): p. 721-2. doi: 10.1261/rna.050120.115

Finlay, J., CM. Roberts, G. Lowe, J. Loeza, JJ. Rossi, and CA. Glackin. “RNA-based TWIST1 inhibition via dendrimer complex to reduce breast cancer cell metastasis”. Biomed Res Int., 2015. 2015:382745. doi: 10.1155/2015/382745.

Takahashi, M., JC. Burnett, and JJ. Rossi. “Aptamer-siRNA chimeras for HIV”. Adv Exp Med Biol., 2015. 848: p. 211-34. doi: 10.1007/978-1-4939-2432-5_11.

Zhou, J., S. Satheesan, H. Li, MS. Weinberg, KV. Morris, JC. Burnett, and JJ. Rossi. “Cell-specific RNA aptamer against human CCR5 specifically targets HIV-1 susceptible cells and inhibits HIV-1 infectivity”. Chem Biol., 2015. 22(3): p. 379-90. doi: 10.1016/j.chembiol.2015.01.005.

Daniels, SM. L. Sinck, NJ. Ward, CE. Melendez-Peña, RJ. Scarborough, I. Azar, E. Rance, A. Daher, KM. Pang, JJ. Rossi, and A. Gatignol. “HIV-1 RRE RNA acts as an RNA silencing suppressor by competing with TRBP-bound siRNAs”. RNA Biol., 2015. 12(2): p. 123-35. doi: 10.1080/15476286.2015.1014759.

Dr. Robert Redfield, Jr., MD

  • Co-founder, Institute of Human Virology at the University of Maryland
  • Division Head and Professor of Medicine, Microbiology and Immunology
  • Chief of Infectious Diseases and Director of the HIV program
  • Presidential Advisory Council on HIV/AIDS

Collaborating with AGT on HIV/AIDS

Dr. Robert Redfield, Jr. has been actively engaged in clinical research and clinical care of chronic human viral infections, especially HIV. His dominant area of research interest is the development of novel biological approaches to the treatment of chronic viral pathogens with a particular focus of targeting host cell pathways for their therapeutic potential.

Several novel areas under active translation investigation include:

  • targeting key biochemical pathways of nucleotide biosynthesis to enhance activity of specific antiretroviral medication
  • Targeting cell activation with specific cell cycles as a primary treatment target
  • Use of G1 cell cycle agents to down-regulate expression of key HIV host cell receptors
  • Use of G1 cell cycles to enhance antiviral activity of HIV entry inhibitors
  • Use of HIV-specific proteins as therapeutic vaccines

Dr. Redfield is also actively involved in clinical research focused on the evaluation of alterative HIV-treatment strategies designed to improve treatment outcome and the evaluation of new commercially developed antiviral medications. The clinical research unit which he oversees has more than 30 active intervention trials. Dr. Redfield’s research focus is driven by his goal to develop durable treatment for persons living with HIV infection and other chronic viral infections in the US and throughout the world, including resource-limited settings.

Currently, Dr. Redfield oversees an extensive clinical program providing HIV care and treatment to more than 4,000 patients in the Baltimore–Washington community. He also oversees an extensive care and treatment program as part of the President’s Emergency Plan for AIDS Relief, which is active in nine (9) countries in Africa and the Caribbean and is now providing ARV treatment to more than 50,000 people living with HIV. Improvement in the outcome of treatment programs is driven by targeted program evaluation and operational research.

Dr. Redfield serves as a member of the Presidential Advisory Council on HIV/AIDS as well as being the Chair of the International Subcommittee. He has also recently completed terms on NIH’s Advisory Council for the Office of AIDS Research and the Advisory Board of the NIH’s Fogarty International Center.

Selected Publications

Redfield R R, Markham PD, Salahuddin SZ, Sarngadharan MG, Bodner AJ, Folks TM, Ballou WR, Wright DC, Gallo RC. Frequent Transmission of HTLV-III Among Spouses of Patients with AIDS-Related Complex and AIDS. JAMA 1985; 253(11):1571-1573.

Redfield R R, Markham PD, Salahuddin SZ, Wright DC, Sarngadharan MG, Gallo RC. Heterosexually Acquired HTLV III/LAV Disease (AIDS-Related Complex and AIDS). JAMA 1985; 254 (15); 2094-2096.

Redfield R R, Wright DC, Tramont EC. The Walter Reed Staging Classification for HTLV-III, LAV Infection. N Engl J Med 1986; 314: 131-132.

Redfield R R, Birx DL, Ketter N, Polonis V, Davis C, Smith G, Johnson S, Fowler A, Brundage JF, Wierzba T, Shafferman A, Volvovitz F, Oster C, Tramont EC, Burke DS. HIV Vaccine Therapy Phase I Safety and Immunogenicity Evaluation of Post Infection Immune Modification by Active Immunization with Recombinant GP 160. N Eng J Med.1991; 324: 1677-1684.

Michael N, Morrow P, Mosca J, Vahey MT, Burke DS, Redfield R R. Induction of HIV-1 Expression in Chronically Infected Cells is Associated Primarily with a Shift in RNA Spacing Patterns. J Virology 1991: 65 (7084).

Michael NL, Vahey M, Burke DS, Redfield R R. “Viral DNA and mRNA Expression Correlate with the State of Human Immunodeficiency Virus (HIV) Type 1 Infection in Humans: Evidence for Viral Replication in all Stages of HIV Disease”. J of Virology 1992; 66 (1): 310-316.

Michael NL, d’Arcy L, Ehrenberg PK, Vahey MT, Birx DL, Redfield R R. 1994. “Naturally Occurring Genotypes of the HIV-1 Long Terminal Repeat Display, A Wide Range of Transcriptional Competency”. J of Virology 1994; 68: 3163-3174.

Margolis D, Heredia A, Gaywee J, Drusano G, Oldach D, Redfield R R “Abacavir and mycophenolic acid, an inhibitor of inosine monophosphate dehydrogenase, have profound and synergistic anti-HIV activity. J AIDS”. 1999;21:362-370.

Heredia A, Margolis D, Oldach D, Hazen R, Nhut L, Redfield R. “Abacavir in combination with the inosine monophosphate dehydrogenase (IMPDH)-inhibitor mycophenolic acid is active against multidrug-resistant HIV-1”. J AIDS. 1999;22:406-412.

Birx DL, Loomis-Price LD, Aronson N, Brundage J, Davis C, Deyton L, Garner R, Gordin F, Henry D, Holloway W, Kerkering T, Luskin-Hawk R, McNeil J, Michael N, Foster Pierce P, Poretz D, Ratto-Kim S, Renzullo P, Ruiz N, Sitz K, Smith G, Tacket C, Thompson M, Tramont E, Yangco B, Yarrish R, Redfield RR. “Efficacy testing of recombinant human immunodeficiency virus (HIV) gp160 as a therapeutic vaccine in early-stage HIV-1-infected volunteers. rgp160 Phase II Vaccine Investigators”. J Infect Dis. 2000; 181:881-9.

Heredia A, Davis C, Redfield RR. “Synergistic inhibition of HIV-1 in activated and resting peripheral blood mononuclear cells, monocyte-derived macrophages, and selected drug-resistant isolates with nucleoside analogues combined with a natural product, Resveratrol”. J AIDS. 2000; JAIDS, 2000; 25(3):246-255.

Davis C, Heredia A, Le N, Dominique JK, Redfield RR. “Differential Human Immunodeficiency Virus-suppressive Activity of Reverse Transcription Inhibitors in Resting and Activated Peripheral Blood Lymphocytes”. JHV. May/June 2001;4(3):113-22.

Heredia A, Davis C, Amoroso A, Dominique JK, Le N, Klingebiel E, Reardon E, Zella D, Redfield RR. “Induction of G1 Cell Cycle Arrest in Peripheral Blood Mononuclear Cells Results in Increased Extracellular Levels of RANTES, MIP-1α and MIP-1β: a Strategy to Inhibit Replication of R5 strains of HIV- 1”. Proc. Nat’l. Acad. Sci. USA, 2003;100:4179-4184

Heredia A, Amoroso A, Davis C, Le N, Reardon E, Dominique JK, Klingebiel E, Gallo RC, Redfield RR. “Rapamycin causes downregulation of CCR5 and accumulation of anti-HIV ß -chemokines: an approach to suppress R5 strains of HIV-1”. Proc. Nat’l Acad. Sci. USA, 2003;100:10411-10416

Alonso Heredia, Charles Davis, Douty Bamba, Nhut Le, Muhammad Y. Gwarzo, Mariola Sadowska, Robert C. Gallo, Robert R. Redfield “Indirubin-3’-monoxime, a derivative of a Chinese antileukemia medicine, inhibits P-TEFb function and HIV-1 replication”. AIDS 2005, 19:2087-2095

Irene Tennant

Director of Clinical Affairs

Irene Tennant is Director of Clinical Affairs for American Gene Technologies.  Irene brings more than 20 years of experience in clinical research and operations, and has led teams in support of the development of biopharma products and pharmaceutical first in class treatments in gene medicine, oncology, and infectious diseases.  With a keen knowledge of the clinical pathway from early stage development to licensure, Irene has managed a number of pivotal projects, overseeing every stage of clinical activity leading to successful FDA approvals.  Irene earned her Bachelor of Science in Biology from Cornell University and her Master of Science in Neurobiology from New York University.

C. Neil Lyons, CPA

Chief Financial Officer & Executive Vice President

C. Neil Lyons, CPA has more than 30 years of experience in finance, strategy, raising capital, SEC compliance, and corporate governance in both public and private companies, with over 10 years of that leading finance for publicly-traded, clinical-stage biotechnology companies. Mr. Lyons has raised over $125 million in capital for biotechnology companies with a career-to-date deal sheet exceeding $1 billion. Mr. Lyons practiced public accounting with Deloitte for the first 10 years of his career and worked in the information technology and communications industries (Honeywell, Verizon and Alcatel) prior to entering the biotechnology industry in 2005.

Dr. Gary Mansfield, PhD

Vice President of Preclinical Development

Gary Mansfield, PhD is VP of Preclinical Development for American Gene Technologies. He has 20 years’ experience in cell and gene therapy product development and has worked in a range of biotechnology settings from seed stage start-ups to late stage clinical companies.  Dr. Mansfield has extensive experience spanning preclinical strategy and operations, CRO/CMO oversight, translational research, manufacturing and regulatory affairs.

Dr. Mansfield has worked at Intronn, VIRxSYS, WuXi AppTec and Mesoblast. At Intronn he was part of the original scientific team that pioneered a novel gene therapy platform for editing endogenous RNA and was intimately involved in the due diligence process leading up to the acquisition of Intronn by VIRxSYS Corporation a pioneering cell and gene therapy company that performed first-in-man clinical trials using lentiviral vectors. Dr. Mansfield also worked at the CRO WuXi AppTec as scientific director overseeing the areas of complex biologics including cell and gene therapies.  Following this Dr. Mansfield worked for Mesoblast in New York City, a late stage clinical, publicly traded biotech company using mesenchymal stem cells to treat a variety of disorders. Dr. Mansfield was chair of the preclinical strategy and operations committee at Mesoblast tasked with developing strategies for each therapeutic area, formulating operating standards, selection of study sites, and review of IND enabling programs. He has served on the IACUC and Institutional Biosafety Committee of external clinical stage biotechnology company’s and established and chaired Institutional Biosafety Committees at two of his previous company positions.  He has also been a senior representative in the due diligence process with major biopharmaceutical companies.

Dr. Mansfield received his PhD from the University of Nottingham in the UK and performed his postdoctoral studies at King’s College London, Harvard University and Duke University before entering the biotechnology arena.  He has published 28 papers and is inventor on 10 biotechnology patents.

Selected Publications

D’Costa J, Mansfield SG, and Humeau L. Lentiviral vectors in clinical trials Curr Opin Mol Ther 11:554-64 (2009).

Rodriguez-Martin T, Anthony K, Garcia-Blanco MA, Mansfield SG, Anderton BH, Gallo JM. Correction of tau mis-splicing caused by FTDP-17 MAPT mutations by spliceosome-mediated RNA trans-splicing. Hum Mol Genet 18:3266-73 (2009).

Wang J, Mansfield SG, Cote CA, Jiang PD, Weng K, Amar MJ, Brewer BH Jr, Remaley AT, McGarrity GJ, Garcia-Blanco MA, and Puttaraju M. Trans-splicing into highly abundant albumin transcripts for production of therapeutic proteins in vivoMol Ther (2009).

Liu X, Luo M, Zhang LN, Yan Z, Zak R, Ding W, Mansfield SG, Mitchell LG, and Engelhardt JF. Spliceosome-mediated RNA trans-splicing with recombinant adeno-associated virus partially restores cystic fibrosis transmembrane conductance regulator function to polarized human cystic fibrosis airway epithelial cells. Hum Gene Ther 16:1116-23 (2005).

Rodriguez-Martin T, Garcia-Blanco MA, Mansfield SG, Grover AC, Hutton M, Yu Q, Zhou J, Anderton BH, and Gallo JM. Reprogramming of tau alternative splicing by spliceosome-mediated RNA trans-splicing: implications for tauopathies. Proc Natl Acad Sci USA 102:15659-64 (2005).

Mansfield SG, Chao H, and Walsh CE. RNA repair using spliceosome-mediated RNA trans-splicing.  Trends Mol Med 10:263-8 (2004).

Mansfield, SG, Clark RH, Puttaraju M, Kole J, Cohn JA, Mitchell LG, and Garcia-Blanco MA. 5′ exon replacement and repair by spliceosome-mediated RNA trans-splicing. RNA 9:1290-7 (2003).

Chao H, Mansfield SG, Bartel RC, Hiriyanna S, Mitchell LG, Garcia-Blanco MA, and Walsh CE. Phenotype correction of hemophilia A mice by spliceosome-mediated RNA trans-splicing. Nat Med 9:1015-9 (2003).

Liu X, Jiang Q, Mansfield SG, Puttaraju M, Zhang Y, Zhou W, Cohn JA, Garcia-Blanco MA, Mitchell LG, and Englehardt JF. Partial correction of endogenous DeltaF508 CFTR in human cystic fibrosis airway epithelia by spliceosome-mediated RNA trans-splicing. Nat Biotech 20:47-52 (2002).

Mansfield SG, Kole J, Puttaraju M, Yang CC, Garcia-Blanco MA, Cohn JA, and Mitchell LG. Repair of CFTR mRNA by spliceosome-mediated RNA trans-splicing. Gene Ther 7:1885-95 (2000).

Puttaraju M, Jamison S, Mansfield SG, Garcia-Blanco MA, and Mitchell LG. Spliceosome-mediated RNA trans-splicing as a tool for gene therapy. Nature Biotech 17:246-252 (1999).

Dr. David Pauza, PhD

Chief Science Officer

David Pauza, PhD is Chief Science Officer for American Gene Technologies and Professor of Medicine at the University of Maryland Medical School in Baltimore. Prior to joining AGT, Dr. Pauza was Associate Director for the university’s prestigious Institute of Human Virology and Co-Leader of the Greenebaum Cancer Center Program in Viral Oncology. He is an internationally recognized expert in human virology and viral diseases including HIV, arenaviruses, poxviruses and herpesviruses. Dr. Pauza is also recognized as an expert in viral immunology, particularly the role for innate immune cells including NK and gamma delta T cells. He was honored as the United Nations Scientific, Cultural and Educational Organization’s Visiting Professor in Biotechnology at the University of Rome and the William J. Way Visiting Professor at the Duke University Center for AIDS Research. Dr. Pauza received the Chernow Foundation Award from the American Foundation for AIDS Research and is Honorary Member of the Gold Key Society. Dr. Pauza has been funded continuously by the N.I.H. for nearly 30 years.

Dr. Pauza is recognized for his outstanding efforts as a scientific thought leader, educator and consultant. He was an empaneled member of the Vaccine Study Section for the N.I.H. and has been active as a reviewer of federal grants and programs. He has been and continues on the editorial boards of several scientific journals, has consulted for a number of small to medium biotechnology companies and been an advisor to numerous health advocacy organizations including the AIDSResearch Alliance. Dr. Pauza is Chair of the National Scientific Advisory Board for the Southwest National Primate Research Center and serves on the Executive Advisory Board for the Chantal Biya Center for International Research in Yaounde, Cameroon. Formerly, he was an Expert Consultant in Immunology to the China Integrated Program on AIDS Research and helped to organize the 2013 Moscow Week in Virology – that included an international scientific conference and educational experiences for Russian infectious disease physicians. He mentored 16 students in their PhD research and trained 22 postdoctoral fellows.

Dr. Pauza received his PhD in 1981 from the Department of Molecular Biology, University of California Berkeley in the laboratory of Howard K. Schachman, PhD and had postdoctoral training at the Laboratory of Molecular Biology, Cambridge, England under Sydney Brenner, MD. In 1985 he started the HIV/AIDS research program at the Salk Institute for Biological Studies in La Jolla, California then moved to the University of Wisconsin-Madison in 1990 to build a diverse program on HIV/AIDS there. In 2000 he was recruited to the Institute of Human Virology in Baltimore, Maryland before joining AGT as a consultant (2012) and subsequently as Chief Science Officer (2015). He has published more than 150 scientific papers and holds 3 US patents

Selected Publications

Li, H., H. Peng, P. Ma, Y. Ruan, B. Su, X. Ding, C. Xu, C.D. Pauza, and Y. Shao. “Association between Vgamma2Vdelta2 T cells and disease progression after infection with closely related strains of HIV in China”. Clin Infect Dis, 2008. 46(9): p. 1466-72.

Riedel, D.J., M.M. Sajadi, C.L. Armstrong, J.S. Cummings, C. Cairo, R.R. Redfield, and C.D. Pauza. “Natural viral suppressors of HIV-1 have a unique capacity to maintain gammadelta T cells”. Aids, 2009. 23(15): p. 1955-64.

Hebbeler, A.M., N. Propp, C. Cairo, H. Li, J.S. Cummings, L.P. Jacobson, J.B. Margolick, and C.D. Pauza. “Failure to restore the Vgamma2-Jgamma1.2 repertoire in HIV-infected men receiving highly active antiretroviral therapy (HAART)”. Clin Immunol, 2008. 128(3): p. 349-57.

Cummings, J.S., C. Cairo, C. Armstrong, C.E. Davis, and C.D. Pauza. “Impacts of HIV infection on Vgamma2Vdelta2 T cell phenotype and function: a mechanism for reduced tumor immunity in AIDS”. J Leukoc Biol, 2008. 84(2): p. 371-9.

Alexander, A.A., A. Maniar, J.S. Cummings, A.M. Hebbeler, D.H. Schulze, B.R. Gastman, C.D. Pauza, S.E. Strome, and A.I. Chapoval. “Isopentenyl pyrophosphate-activated CD56+ {gamma}{delta} T lymphocytes display potent antitumor activity toward human squamous cell carcinoma”. Clin Cancer Res, 2008. 14(13): p. 4232-40.

Maniar, A., X. Zhang, W. Lin, B.R. Gastman, C.D. Pauza, S.E. Strome, and A.I. Chapoval. “Human gammadelta T lymphocytes induce robust NK cell-mediated antitumor cytotoxicity through CD137 engagement”. Blood. 116(10): p. 1726-33.

Jeffrey A. Galvin

CEO and Founder

Jeffrey A. Galvin is the CEO and Co-founder of American Gene Technologies™ (AGT). He earned his BA degree in Economics from Harvard in 1981. He has more than 30 years of business and entrepreneurial experience including founder or executive positions at a variety of Silicon Valley startups. Several of his companies were taken public and/or sold to public companies, including one in the medical-technology arena that was sold to Varian, the leading maker of linear accelerators used in cancer therapy. Following his startup experience, he retired to become an Angel Investor in real estate and high tech. He came out of retirement to found and fund AGT after meeting Roscoe Brady at NIH.

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