James Wilson (scientist)

James M. Wilson is a biomedical researcher with expertise in gene therapy.  Dr. Wilson graduated from Albion College (B.A., Chemistry) and the University of Michigan (MD, PhD).[1]  He completed residency training in Internal Medicine at the Massachusetts General Hospital followed by a postdoctoral fellowship at the Whitehead Institute.[2]

Dr. Wilson serves as the Director of the Gene Therapy Program, Rose H. Weiss Professor and Director of the Orphan Disease Center, and Professor of Medicine and Pediatrics at the Perelman School of Medicine at the University of Pennsylvania.[3]

Dr Wilson was the director of a gene therapy clinical trial that led to the death of 18-year-old Jesse Gelsinger in 1999. In a 2005 legal settlement, Wilson's clinical research was restricted and placed under supervision until 2010.[4]

Scientific Contributions

Dr. Wilson’s research involves the development of viral-based gene therapies for genetic diseases including cystic fibrosis, infantile epilepsies, storage diseases, liver metabolic diseases, neurodegenerative diseases, ocular diseases, and infectious diseases. A major research focus is the generation of novel vectors for improved transduction efficiencies and regulated expression, as well as the elucidation of host immune responses to viral vectors.[5]

His recent work emphasizes the creation of vectors for in vivo gene therapy concentrating on adeno-associated viruses (AAV). This work began with the recent discovery in his laboratory of a new family of primate AAVs; over 120 new AAV capsids were rescued as latent genomes from primate tissues and studied for their biology and potential as vectors.[6][7] This has led to an enhanced understanding of vector host interactions and a new generation of vectors with substantially improved performance profiles beyond that provided from the original 6 AAV isolates.  Most recently, Dr. Wilson’s laboratory has used AAV to accomplish successful in vivo genome editing.[8][9]

At the present time, his laboratory’s translational research portfolio includes more than 30 orphan disease programs.[10]

Recent Honors and Awards

2013    Gene Therapy Pioneer Award, Human Gene Therapy[11]

2014    Jane M. Glick Graduate Student Teaching Award, University of Pennsylvania[12]

2014    William Osler Patient Oriented Research Award, University of Pennsylvania[13]

2014    Top 20 Translational Researcher in 2013 based on paper and patent output (#2), Nature Biotechnology[14]

2015    PA BIO 2015 Scientific Achievement Award[15]

2015    Top 20 Translational Researchers in 2014 based on paper and patent output (#17), Nature Biotechnology[16]

2016    Top 20 Translational Researchers in 2015 based on paper and patent output (#7), Nature Biotechnology[17]

2017    Top 20 Translational Researchers in 2016 based on paper and patent output (#10), Nature Biotechnology[18]

2018    Top 20 Translational Researchers in 2017 based on paper and patent output (#2), Nature Biotechnology[19]

Community Outreach

Dr. Wilson is a Trustee at Albion College[20] and is the founder and President of a 501(c)3 called Health Through Fitness in Orphan Diseases.[21]

Under his leadership, the Orphan Disease Center organizes the Million Dollar Bike Ride in Philadelphia each year to raise money for rare disease research and bring together patients and families from the rare disease community. http://orphandiseasecenter.med.upenn.edu/million-dollar-bike-ride/

Gene Therapy Program at the University of Pennsylvania: https://gtp.med.upenn.edu/

Orphan Disease Center at the University of Pennsylvania: http://orphandiseasecenter.med.upenn.edu/

References

  1. ↑ "James M. Wilson | Faculty | About Us | Perelman School of Medicine | Perelman School of Medicine at the University of Pennsylvania". www.med.upenn.edu. Retrieved 2018-09-28.
  2. ↑ "James M. Wilson - Faculty Biosketch". www.med.upenn.edu. Retrieved 2018-09-28.
  3. ↑ "University of Pennsylvania Gene Therapy Program | Our Team". gtp.med.upenn.edu. Retrieved 2018-09-28.
  4. ↑ Check, E. (2005). "Sanctions agreed over teenager's gene-therapy death." Nature volume 433, page 674 (17 February 2005)
  5. ↑ "University of Pennsylvania Gene Therapy Program | Home". https://www.nature.com. Retrieved 2018-10-11. External link in |website= (help)
  6. ↑ Gao, Guang-Ping; Alvira, Mauricio R.; Wang, Lili; Calcedo, Roberto; Johnston, Julie; Wilson, James M. (2002-09-03). "Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy". Proceedings of the National Academy of Sciences of the United States of America. 99 (18): 11854–11859. doi:10.1073/pnas.182412299. ISSN 0027-8424. PMC 129358. PMID 12192090.
  7. ↑ Gao, Guangping; Vandenberghe, Luk H.; Alvira, Mauricio R.; Lu, You; Calcedo, Roberto; Zhou, Xiangyang; Wilson, James M. (2004-6). "Clades of Adeno-associated viruses are widely disseminated in human tissues". Journal of Virology. 78 (12): 6381–6388. doi:10.1128/JVI.78.12.6381-6388.2004. ISSN 0022-538X. PMC 416542. PMID 15163731. Check date values in: |date= (help)
  8. ↑ Yang, Yang; Wang, Lili; Bell, Peter; McMenamin, Deirdre; He, Zhenning; White, John; Yu, Hongwei; Xu, Chenyu; Morizono, Hiroki (2016-3). "A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice". Nature Biotechnology. 34 (3): 334–338. doi:10.1038/nbt.3469. ISSN 1546-1696. PMC 4786489. PMID 26829317. Check date values in: |date= (help)
  9. ↑ Wang, Lili; Smith, Jeff; Breton, Camilo; Clark, Peter; Zhang, Jia; Ying, Lei; Che, Yan; Lape, Janel; Bell, Peter (2018-9). "Meganuclease targeting of PCSK9 in macaque liver leads to stable reduction in serum cholesterol". Nature Biotechnology. 36 (8): 717–725. doi:10.1038/nbt.4182. ISSN 1546-1696. PMID 29985478. Check date values in: |date= (help)
  10. ↑ "University of Pennsylvania Gene Therapy Program | Translational Research Lab". gtp.med.upenn.edu. Retrieved 2018-09-28.
  11. ↑ "Mary Ann Liebert, Inc., publishers | News". home.liebertpub.com. Retrieved 2018-09-28.
  12. ↑ "Awards for Dr. James Wilson | University of Pennsylvania | Pathology and Laboratory Medicine". pathology.med.upenn.edu. Retrieved 2018-09-28.
  13. ↑ "Awards for Dr. James Wilson | University of Pennsylvania | Pathology and Laboratory Medicine". pathology.med.upenn.edu. Retrieved 2018-09-28.
  14. ↑ "Table 1: Top translational researchers in 2013, ranked by total patents at their current affiliation". www.nature.com. Retrieved 2018-09-28.
  15. ↑ "3/17/15, Honors & Other Things - Almanac, Vol. 61, No. 26". almanac.upenn.edu. Retrieved 2018-09-28.
  16. ↑ "Table 1: Top 20 researchers in 2014". www.nature.com. Retrieved 2018-09-28.
  17. ↑ "Table 1: Top 20 translational researchers in 2016". www.nature.com. Retrieved 2018-09-28.
  18. ↑ "Table 1: Top 20 translational researchers in 2016". www.nature.com. Retrieved 2018-09-28.
  19. ↑ "Table 1: Top 20 translational researchers in 2017". www.nature.com. Retrieved 2018-09-28.
  20. ↑ Perney, John. "Board of Trustees - Albion College". www.albion.edu. Retrieved 2018-09-28.
  21. ↑ "HTFOD.org | Heath Through Fitness in Orphan Diseases". htfod.org. Retrieved 2018-09-28.
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