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Strategies to improve safety profile of AAV vectors

1 Nov 2022

Abstract excerpt

Adeno-associated virus (AAV) vectors are currently used in four approved gene therapies for Leber congenital amaurosis (Luxturna), spinal muscular atrophy (Zolgensma), aromatic L-amino acid decarboxylase deficiency (Upstaza) and Haemophilia A (Roctavian), with several more therapies being investigated in clinical trials. AAV gene therapy has long been considered extremely safe both in the context of...

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PMID: 39086961
DOI: 10.3389/fmmed.2022.1054069

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