A systematic review of adeno-associated virus gene therapies in neurology: the need for consistent safety monitoring of a promising treatment

Adeno-associated virus (AAV) gene therapies are generating much excitement in the rare disease field, particularly for previously untreatable neurological conditions. Efficacy has been claimed for several gene therapy products and the number of trials is rapidly increasing. However, reports of sever...

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Номзүйн дэлгэрэнгүй
Үндсэн зохиолчид: Horton, RH, Saade, D, Markati, T, Harriss, E, Bönnemann, CG, Muntoni, F, Servais, L
Формат: Journal article
Хэл сонгох:English
Хэвлэсэн: BMJ Publishing Group 2022