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Clinical Considerations for Capsid Choice in the Development of Liver-Targeted AAV-Based Gene Transfer

10 Sept 2019

Abstract excerpt

As gene transfer with adeno-associated virus (AAV) vectors is starting to enter clinical practice, this review examines the impact of vector capsid choice in liver-directed gene transfer for hemophilia. Given that there are multiple clinical trials completed and ongoing in this field, it is important to review the clinical evidence, particularly as a range of AAV-vector serotypes including AAV2, AAV5, AAV8, and...

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PMID: 31660419
DOI: 10.1016/j.omtm.2019.08.015

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