Adeno-associated virus vector as a platform for gene therapy delivery

Adeno-associated virus vector as a platform for gene therapy delivery

Adeno-associated virus vector as a platform for gene therapy delivery, Published online: 01 February 2019; doi:10.1038/s41573-019-0012-9

Adeno-associated virus (AAV) vectors represent the leading platform for therapeutic gene delivery, with two recombinant AAV gene therapy products having gained regulatory approval in Europe or the United States. Here, Gao and colleagues discuss the fundamentals of AAV and vectorology, focusing on current therapeutic strategies, clinical progress and ongoing challenges.

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