Adeno-associated virus gene therapy

Adeno-associated virus (AAV) gene therapy uses a modified version of a virus called adeno-associated virus (AAV) to deliver a working copy of a defective gene into the cells relevant for a given disease. AAV has been shown to deliver therapeutic genes to cells in the eye, liver, brain, muscle and other organs.

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NCATS Platform Vector Gene Therapy (PaVe-GT): Why We Need PaVe-GT
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American Society of Gene and Cell Therapy: Vector 101
Adeno-Associated Virus (AAV) as a Vector for Gene Therapy [2017 published article]