NDLI: 30 Years Later-Reflections on Human Gene Therapy: An Interview with Terry Flotte, MD.

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30 Years Later-Reflections on Human Gene Therapy: An Interview with Terry Flotte, MD.

Content Provider	Semantic Scholar
Author	Davies, Kevin
Copyright Year	2020
Abstract	Dr. Flotte: This same timeframe overlaps with my own career journey. In the ’80s, I was a pediatric resident at JohnsHopkins anddevelopeda passion for trying to impact cystic fibrosis (CF). I became a pediatric pulmonology fellow just before the discovery of the CFTR gene in 1989. I had the opportunity to take time out from my clinical training for about 3 years and get work in the laboratory of a basic virologist, Barrie Carter, who was at National Institute of Diabetes Digestive and Kidney Diseases. My mission was part of the first wave of trying to make batches of recombinant adeno-associated virus (AAV) that were free of wild-type virus. That was the primordial stage wherein gene therapy was very much about virology, and then when the CFTR gene became available in ’89 to work on addressing that problem. That anecdote shows that I am a physician-scientist, a physician first, who has seen the platforms associated with virology, molecular biology, and RNA biology as potential tools in a clinical armamentarium. I happened to be at NIH in 1989, when Steve Rosenberg did the first tumor-infiltrating lymphocytes from patients with melanoma and did the gene marking with the retrovirus with the neo gene, which is nontherapeutic. I was also there when Mike Blaese and French Anderson were doing the first adenosine deaminase severe combined immune deficiency patients. I was a bystander. They were trying to learn how to grow viruses, but I was a witness along the way to some of these developments. As the journal came out in 1990, I thought it was phenomenal that the decision was made to call the first journal in the field Human Gene Therapy, which is a very purposeful thing— not Gene Therapy or Gene Technology. The human side of it has always been, for me, the driving motivation. We have moved on in my laboratory, and in my own leadership in leading first a gene therapy center, then a genetics institute, the department of pediatrics, and now a medical school for the past 21 years, to see Terry Flotte, MD Editor-in-Chief, Human Gene Therapy Editor’s note: Terry Flotte, MD, wears a number of hats in addition to being the Editor-in-Chief of Human Gene Therapy. He is the Dean of the School of Medicine, Provost, and Executive Deputy Chancellor of the University of Massachusetts Medical School in Worcester, MA. Like the journal, Dr. Flotte has been in the gene therapy trenches for three decades. In addition to leading a talented group of researchers and physicians, he is involved in several pioneering clinical trials, including Tay–Sachs disease. Here, in conversation with Dr. Kevin Davies, Dr. Flotte reflects on the ups and downs of gene therapy and the new vision for Human Gene Therapy.
Starting Page	5
Ending Page	8
Page Count	4
File Format	PDF HTM / HTML
DOI	10.1089/hum.2019.29097.int
PubMed reference number	31967919
Journal	Medline
Volume Number	31
Issue Number	1-2
Alternate Webpage(s)	https://www.liebertpub.com/doi/pdf/10.1089/hum.2019.29097.int
Alternate Webpage(s)	https://doi.org/10.1089/hum.2019.29097.int
Journal	Human gene therapy
Language	English
Access Restriction	Open
Content Type	Text
Resource Type	Biography

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