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...
Main Authors: | Horton, RH, Saade, D, Markati, T, Harriss, E, Bönnemann, CG, Muntoni, F, Servais, L |
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Format: | Journal article |
Language: | English |
Published: |
BMJ Publishing Group
2022
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