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AAV gene delivery to the spinal cord: serotypes, methods, candidate diseases, and clinical trials

18 Dec 2017

Abstract excerpt

INTRODUCTION: Adeno-associated viral (AAV) vector-mediated gene delivery to the spinal cord has finally entered the pathway towards regulatory approval. Phase 1 clinical trials using AAV gene therapy for pediatric disorders - spinal muscular atrophy (SMA) and giant axonal neuropathy (GAN) - are now underway. AREAS COVERED: This review addresses the latest progress in the field of AAV gene delivery to the spinal...

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PMID: 29249183
DOI: 10.1080/14712598.2018.1416089

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