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{{DISPLAYTITLE:James M. Wilson (scientist)}}
'''James M. Wilson''' is an American geneticist and medical researcher at the [[University of Pennsylvania]], known for his work on [[gene therapy]], where he was regarded as one of the field's most prominent researchers<ref name=wired>[https://www.wired.com/2013/08/the-fall-and-rise-of-gene-therapy-2/ Gene Therapy Emerges From Disgrace to Be the Next Big Thing, Again] by Carl Zimmer August 13, 2013 Wired</ref> until ethical concerns arose around the death of a patient in one of his trials.<ref name=wired /><ref name="James M. Wilson 2009 727–728">{{citation|title=A History Lesson for Stem Cells |author=James M. Wilson |journal=[[Science (journal)|Science]] |volume=324 |pages=727–728 |year=2009 |jstor=20493877 |registration=yes |doi=10.1126/science.1174935}}</ref>


'''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).<ref>{{Cite web|url=https://www.med.upenn.edu/apps/faculty/index.php/g275/p9746|title=James M. Wilson {{!}} Faculty {{!}} About Us {{!}} Perelman School of Medicine {{!}} Perelman School of Medicine at the University of Pennsylvania|website=www.med.upenn.edu|language=en|access-date=2018-09-28}}</ref>  He completed residency training in Internal Medicine at the [[Mass General Hospital|Massachusetts General Hospital]] followed by a postdoctoral fellowship at the Whitehead Institute.<ref>{{Cite web|url=https://www.med.upenn.edu/apps/faculty/index.php/g350/p9746|title=James M. Wilson - Faculty Biosketch|website=www.med.upenn.edu|access-date=2018-09-28}}</ref>  
==Biography==
Wilson earned his BA in chemistry at [[Albion College]] in 1977, and his MD and PhD at the [[University of Michigan]] in 1984, where he studied the genetics of [[Lesch-Nyhan syndrome]].<ref name=Uni_Pennsylvania /> He did a residency in internal medicine at [[Mass General Hospital]] and a postdoctoral fellowship at [[MIT]] with Richard Mulligan.<ref>http://online.liebertpub.com/doi/full/10.1089/hum.2014.2511</ref>


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.<ref>{{Cite web|url=https://gtp.med.upenn.edu/about/our-team|title=University of Pennsylvania Gene Therapy Program {{!}} Our Team|website=gtp.med.upenn.edu|language=en|access-date=2018-09-28}}</ref>
As of 2017, he is the director of the [[University of Pennsylvania]] Gene Therapy Program and Orphan Disease Center.<ref name=Uni_Pennsylvania>[http://www.med.upenn.edu/apps/faculty/index.php/g5165284/p9746 James M. Wilson], University of Pennsylvania</ref><ref>[http://www.scientificamerican.com/article/gene-therapy-an-interview/ Gene therapy]; an interview Scientific American</ref> His research interests include genetic disorders of [[lipid metabolism]], and gene therapy for [[dyslipidemia]]s, [[coronary artery disease]], [[congestive heart failure]], pandemic viruses, lysosomal storage diseases, and editing delivery technology.<ref name=Uni_Pennsylvania />


==Scientific Contributions==
== OTD clinical trial ==
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.<ref>{{Cite web|url=https://gtp.med.upenn.edu/|title=University of Pennsylvania Gene Therapy Program {{!}} Home|website=gtp.med.upenn.edu|language=en|access-date=2018-09-28}}</ref>
In 1997, Wilson, [[Steven Raper]], and [[Mark Batshaw]], all physicians at the [[University of Pennsylvania]], began enrolling patients in a clinical trial using experimental [[gene therapy]] to treat [[ornithine transcarbamylase deficiency]]. In 1998, after a patient experienced liver toxicity as a side effect of the treatment, they altered the clinical trial agreement to allow patients to continue receiving treatment after experiencing liver toxicity. As the administered doses increased, subsequent patients experienced liver toxicity on a regular basis.<ref>http://www.fda.gov/downloads/iceci/enforcementactions/enforcementstory/enforcementstoryarchive/ucm091079.pdf</ref>


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.<ref>{{Cite journal|last=Gao|first=Guang-Ping|last2=Alvira|first2=Mauricio R.|last3=Wang|first3=Lili|last4=Calcedo|first4=Roberto|last5=Johnston|first5=Julie|last6=Wilson|first6=James M.|date=2002-09-03|title=Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy|url=https://www.ncbi.nlm.nih.gov/pubmed/12192090|journal=Proceedings of the National Academy of Sciences of the United States of America|volume=99|issue=18|pages=11854–11859|doi=10.1073/pnas.182412299|issn=0027-8424|pmid=12192090}}</ref><ref>{{Cite journal|last=Gao|first=Guangping|last2=Vandenberghe|first2=Luk H.|last3=Alvira|first3=Mauricio R.|last4=Lu|first4=You|last5=Calcedo|first5=Roberto|last6=Zhou|first6=Xiangyang|last7=Wilson|first7=James M.|date=2004-6|title=Clades of Adeno-associated viruses are widely disseminated in human tissues|url=https://www.ncbi.nlm.nih.gov/pubmed/15163731|journal=Journal of Virology|volume=78|issue=12|pages=6381–6388|doi=10.1128/JVI.78.12.6381-6388.2004|issn=0022-538X|pmid=15163731}}</ref> 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.<ref>{{Cite journal|last=Yang|first=Yang|last2=Wang|first2=Lili|last3=Bell|first3=Peter|last4=McMenamin|first4=Deirdre|last5=He|first5=Zhenning|last6=White|first6=John|last7=Yu|first7=Hongwei|last8=Xu|first8=Chenyu|last9=Morizono|first9=Hiroki|date=2016-3|title=A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice|url=https://www.ncbi.nlm.nih.gov/pubmed/26829317|journal=Nature Biotechnology|volume=34|issue=3|pages=334–338|doi=10.1038/nbt.3469|issn=1546-1696|pmc=PMC4786489|pmid=26829317}}</ref><ref>{{Cite journal|last=Wang|first=Lili|last2=Smith|first2=Jeff|last3=Breton|first3=Camilo|last4=Clark|first4=Peter|last5=Zhang|first5=Jia|last6=Ying|first6=Lei|last7=Che|first7=Yan|last8=Lape|first8=Janel|last9=Bell|first9=Peter|date=2018-9|title=Meganuclease targeting of PCSK9 in macaque liver leads to stable reduction in serum cholesterol|url=https://www.ncbi.nlm.nih.gov/pubmed/29985478|journal=Nature Biotechnology|volume=36|issue=8|pages=717–725|doi=10.1038/nbt.4182|issn=1546-1696|pmid=29985478}}</ref>
In 1999, [[Jesse Gelsinger]] died after receiving a gene therapy treatment. Due to his blood ammonia levels being above the maximum allowed for the study, he should not have been allowed to enroll. Further investigation showed improper consent forms and that Wilson had significant financial stakes in the success of the trial.<ref name=wired /><ref name="James M. Wilson 2009 727–728"/> Wilson agreed to a five-year ban on participating in clinical trial research.<ref>https://www.bloomberg.com/news/articles/2015-07-29/doctor-whose-drug-killed-teen-returns-as-gene-tech-booms-again</ref>

At the present time, his laboratory’s translational research portfolio includes more than 30 orphan disease programs.<ref>{{Cite web|url=https://gtp.med.upenn.edu/translational-research-lab|title=University of Pennsylvania Gene Therapy Program {{!}} Translational Research Lab|website=gtp.med.upenn.edu|language=en|access-date=2018-09-28}}</ref>  

== Recent Honors and Awards ==
2013    Gene Therapy Pioneer Award, ''Human Gene Therapy''<ref>{{Cite web|url=https://home.liebertpub.com/news/top-12-pioneer-awards-for-seminal-work-in-gene-and-cell-therapy-selected-by-blue-ribbon-panel-essays-to-be-published-in-human-gene-therapy/1350|title=Mary Ann Liebert, Inc., publishers {{!}} News|website=home.liebertpub.com|language=en|access-date=2018-09-28}}</ref>

2014    Jane M. Glick Graduate Student Teaching Award, University of Pennsylvania<ref>{{Cite web|url=http://pathology.med.upenn.edu/news/awards-dr-james-wilson|title=Awards for Dr. James Wilson {{!}} University of Pennsylvania {{!}} Pathology and Laboratory Medicine|website=pathology.med.upenn.edu|language=en|access-date=2018-09-28}}</ref>

2014    William Osler Patient Oriented Research Award, University of Pennsylvania<ref>{{Cite web|url=http://pathology.med.upenn.edu/news/awards-dr-james-wilson|title=Awards for Dr. James Wilson {{!}} University of Pennsylvania {{!}} Pathology and Laboratory Medicine|website=pathology.med.upenn.edu|language=en|access-date=2018-09-28}}</ref>

2014    Top 20 Translational Researcher in 2013 based on paper and patent output (#2), ''Nature Biotechnology''<ref>{{Cite web|url=https://www.nature.com/articles/nbt.2986/tables/1|title=Table 1: Top translational researchers in 2013, ranked by total patents at their current affiliation.|website=www.nature.com|language=en|access-date=2018-09-28}}</ref>

2015    PA BIO 2015 Scientific Achievement Award<ref>{{Cite web|url=https://almanac.upenn.edu/archive/volumes/v61/n26/honors-other-things.html|title=3/17/15, Honors & Other Things - Almanac, Vol. 61, No. 26|website=almanac.upenn.edu|access-date=2018-09-28}}</ref>

2015    Top 20 Translational Researchers in 2014 based on paper and patent output (#17), ''Nature Biotechnology''<ref>{{Cite web|url=https://www.nature.com/articles/nbt.3335/tables/1|title=Table 1: Top 20 researchers in 2014|website=www.nature.com|language=en|access-date=2018-09-28}}</ref>

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

2017    Top 20 Translational Researchers in 2016 based on paper and patent output (#10), ''Nature Biotechnology''<ref>{{Cite web|url=https://www.nature.com/articles/nbt.4028/tables/1|title=Table 1: Top 20 translational researchers in 2016|website=www.nature.com|language=en|access-date=2018-09-28}}</ref>

2018    Top 20 Translational Researchers in 2017 based on paper and patent output (#2), ''Nature Biotechnology''<ref>{{Cite web|url=https://www.nature.com/articles/nbt.4237/tables/1|title=Table 1: Top 20 translational researchers in 2017|website=www.nature.com|language=en|access-date=2018-09-28}}</ref>

== Community Outreach ==
Dr. Wilson is a Trustee at Albion College<ref>{{Cite web|url=https://www.albion.edu/about-albion/board-of-trustees|title=Board of Trustees - Albion College|last=Perney|first=John|website=www.albion.edu|language=en-gb|access-date=2018-09-28}}</ref> and is the founder and President of a 501(c)3 called Health Through Fitness in Orphan Diseases.<ref>{{Cite web|url=http://htfod.org/|title=HTFOD.org {{!}} Heath Through Fitness in Orphan Diseases|website=htfod.org|language=en-US|access-date=2018-09-28}}</ref> 

Under his leadership, the Orphan Disease Center organizes the [http://orphandiseasecenter.med.upenn.edu/million-dollar-bike-ride/<nowiki> 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. </nowiki>

== Web Links ==
[https://gtp.med.upenn.edu/<nowiki>Gene Therapy Program at the University of Pennsylvania]</nowiki>

<nowiki>[http://orphandiseasecenter.med.upenn.edu/ Orphan Disease Center at the University of Pennsylvania]</nowiki>


==References==
==References==

Revision as of 21:19, 28 September 2018


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]

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.[4]

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.[5][6] 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.[7][8]

At the present time, his laboratory’s translational research portfolio includes more than 30 orphan disease programs.[9]  

Recent Honors and Awards

2013    Gene Therapy Pioneer Award, Human Gene Therapy[10]

2014    Jane M. Glick Graduate Student Teaching Award, University of Pennsylvania[11]

2014    William Osler Patient Oriented Research Award, University of Pennsylvania[12]

2014    Top 20 Translational Researcher in 2013 based on paper and patent output (#2), Nature Biotechnology[13]

2015    PA BIO 2015 Scientific Achievement Award[14]

2015    Top 20 Translational Researchers in 2014 based on paper and patent output (#17), Nature Biotechnology[15]

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

2017    Top 20 Translational Researchers in 2016 based on paper and patent output (#10), Nature Biotechnology[16]

2018    Top 20 Translational Researchers in 2017 based on paper and patent output (#2), Nature Biotechnology[17]

Community Outreach

Dr. Wilson is a Trustee at Albion College[18] and is the founder and President of a 501(c)3 called Health Through Fitness in Orphan Diseases.[19] 

Under his leadership, the Orphan Disease Center organizes the [http://orphandiseasecenter.med.upenn.edu/million-dollar-bike-ride/ 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.

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

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

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. ^ "University of Pennsylvania Gene Therapy Program | Home". gtp.med.upenn.edu. Retrieved 2018-09-28.
  5. ^ 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. PMID 12192090.
  6. ^ 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. PMID 15163731. {{cite journal}}: Check date values in: |date= (help)
  7. ^ 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. {{cite journal}}: Check date values in: |date= (help)CS1 maint: PMC format (link)
  8. ^ 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. {{cite journal}}: Check date values in: |date= (help)
  9. ^ "University of Pennsylvania Gene Therapy Program | Translational Research Lab". gtp.med.upenn.edu. Retrieved 2018-09-28.
  10. ^ "Mary Ann Liebert, Inc., publishers | News". home.liebertpub.com. Retrieved 2018-09-28.
  11. ^ "Awards for Dr. James Wilson | University of Pennsylvania | Pathology and Laboratory Medicine". pathology.med.upenn.edu. 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. ^ "Table 1: Top translational researchers in 2013, ranked by total patents at their current affiliation". www.nature.com. Retrieved 2018-09-28.
  14. ^ "3/17/15, Honors & Other Things - Almanac, Vol. 61, No. 26". almanac.upenn.edu. Retrieved 2018-09-28.
  15. ^ "Table 1: Top 20 researchers in 2014". www.nature.com. Retrieved 2018-09-28.
  16. ^ "Table 1: Top 20 translational researchers in 2016". www.nature.com. Retrieved 2018-09-28.
  17. ^ "Table 1: Top 20 translational researchers in 2017". www.nature.com. Retrieved 2018-09-28.
  18. ^ Perney, John. "Board of Trustees - Albion College". www.albion.edu. Retrieved 2018-09-28.
  19. ^ "HTFOD.org | Heath Through Fitness in Orphan Diseases". htfod.org. Retrieved 2018-09-28.
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