UAB is participating in a Phase I/II trial of AMT 191, a one time gene therapy designed to enable patients with classic Fabry disease to produce their own missing enzyme, potentially reducing the need ...
The Director of the San Raffaele-Telethon Institute for Gene Therapy Luigi Naldini reflects on what a recent Nature study reveals and about the future of in vivo gene transfer “Our study provides an ...
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Unlocking the secrets of gene therapy delivery: New insights into genome ejection from AAV vectors
A research team at the University of Osaka has unveiled the molecular mechanism behind genome ejection from adeno-associated virus (AAV) vectors, a crucial delivery vehicle in gene therapy. Subscribe ...
A new approach to gene therapy for retinitis pigmentosa may help patients with advanced disease regain vision regardless of which genetic mutation is causing the condition. An optogenetic therapy ...
Gene editing refers to the precise alteration of an organism's DNA sequence at a specific locus. In cancer treatment, this technology is leveraged to modify the genetic makeup of cancer cells, either ...
Advocates stress that while gene therapy is causing a buzz in wealthy countries, African governments must prioritise ...
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