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Article

Development challenges associated with rAAV-based gene therapies

1 Jan 2021

Abstract excerpt

The number of gene therapies in development continues to increase, as they represent a novel method to treat, and potentially cure, many diseases. Gene therapies can be conducted with an in vivo or ex vivo approach, to cause gene augmentation, gene suppression, or genomic editing. Adeno-associated viruses are commonly used to deliver gene therapies, but their use is associated with several manufacturing,...

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PMID: 33536390
DOI: 10.2131/jts.46.57

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