# Gene Therapy
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gene therapy is medical technology that aims to produce a therapeutic effect through the manipulation of gene expression or through altering the biological properties of living cells.
The first attempt at modifying human DNA was performed in 1980, by Martin Cline, but the first successful nuclear gene transfer in humans, approved by the National Institutes of Health, was performed in May 1989. The first therapeutic use of gene transfer as well as the first direct insertion of human DNA into the nuclear genome was performed by French Anderson in a trial starting in September 1990.
One trial may be enough. One uninterpretable trial is not
Episode Summary
In the inaugural episode of Humaneva Insights, host Patrick McManus speaks with Dr. Brandon Fletcher, Head of Medical Affairs and Cell & Gene Therapy at Kapadi, about when a single adequate and well-controlled clinical trial may be sufficient to support an FDA regulatory decision in…
Ep. 218: Beyond Symptom Control: Gene Therapy for Rare Neurological Disorders
Moderator: Kleopas Kleopa (Nicosia, Cyprus)
Guest: Jo Ng (London, UK)
In this episode, Kleopas Kleopa speaks with Jo Ng about gene therapy for rare neurological disorders. They discuss the clinical potential of AAV-based therapies, key diagnostic and therapeutic considerations including early dia…
Gene Therapy - Helen Anderson
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Gene Therapy’s Future: Will Patient Engagement Eclipse Science? | Hypothesize That!
Is scientific innovation enough to drive the future of gene therapy, or is patient engagement the missing key? Join host Nicola Hill as she explores this question with Laurence Woollard, a rare disease advocate, and UCB’s Global Patient Engagement Lead, Dr. Emma Lane. Together, they discuss the cri…
100% Response Rate in ALL: Vironexis's Gene Therapy T-Cell Engager (But There's a Catch)
Welcome to The Biotech Voyager. It's the show that covers what's next in biotech, mapping the early-stage signals before their big news.Hosted by Benjamin McLeod and Jeff Martin, PhD.
In this episode, the hosts navigate an exceptionally busy day in biotech, examining multiple groundbreaking signals…
Episode 516: Gene Therapy in the Retina Space with Dr. David Kirn
Disclosures: This episode was sponsored by Cited. Cited is PubMed meets Instagram Stories for retina specialists, free to download, made for hearing what your peers think about the latest research. Find it at https://apps.apple.com/us/app/cited-for-retina/id6764145960.
What Cell and Gene Therapy Investors Need to See First | Phil Vanek & David Crean
Phil Vanek, PhD, is Chief Commercialization Officer of the International Society for Cell & Gene Therapy (ISCT), chair of the CGT Catapult Technology Advisory Board and founder of Redline Bioadvisors. David H. Crean, PhD, MBA, is founder and managing partner of Cardiff Advisory, a general partner a…
Ohio’s Biotech Boom: Gene Therapy, Investment & the Race with China
Ohio is emerging as a major U.S. life sciences hub, with growing investment in biotech, gene therapy, biomanufacturing and pharmaceutical innovation. But what does it take to build a competitive biotech ecosystem outside traditional hubs like Boston and San Francisco?In this episode of the Making M…
Never Lose Sight: Angela Ricketts on Bringing Light to Rare Eye Diseases
We welcome Angela Ricketts, an author, business owner, and nonprofit founder, ahead of the show’s 400th episode. Angela discusses her son’s diagnosis at age five with choroideremia, an ultra-rare, progressive blinding eye disease affecting boys and men. She contrasts families who wait to accept the…
Roller Coasters, AEDs and Gene Therapy: Your Questions for Dr. Ackerman
Can a child with long QT syndrome ride roller coasters? Should families carry a personal AED? And where does gene therapy for long QT stand?
In this episode of Wild Beats, host Jen Berko brings your questions to Dr. Michael Ackerman, director of Mayo Clinic's Windland Smith Rice Genetic Heart Rhyth…
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