Predicted deleterious variants in the human genome relevant to gene therapy with adeno-associated virus vectors

Predicted deleterious variants in the human genome relevant to gene therapy with adeno-associated virus vectors

Based on the observation that humans have variable responses of gene expression with the same dose of an adeno-associated vector, we hypothesized that there are deleterious variants in genes coding for processes required for adeno-associated virus (AAV)-mediated gene transfer/expression that may ham...

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Bibliographic Details
Main Authors: Mahboubeh R. Rostami, Philip L. Leopold, Jenifer M. Vasquez, Miguel de Mulder Rougvie, Alya Al Shakaki, Ali Ait Hssain, Amal Robay, Neil R. Hackett, Jason G. Mezey, Ronald G. Crystal
Format: Article
Language:English
Published: Elsevier 2023-12-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
AAV
gene therapy
genetic variants
viral infection pathway
whole genome sequencing
populations
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050123001754

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http://www.sciencedirect.com/science/article/pii/S2329050123001754

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