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Safety and efficacy of an engineered hepatotropic AAV gene therapy for ornithine transcarbamylase deficiency in cynomolgus monkeys. (2021)

First Author: Baruteau J

Attributed to: Development of rAAV-mediated gene therapy for a severe paediatric metabolic liver disease: Ornithine Transcarbamylase deficiency. funded by MRC

Abstract

No abstract provided

Bibliographic Information

Digital Object Identifier: http://dx.doi.org/10.1016/j.omtm.2021.09.005

PubMed Identifier: 34703837

Publication URI: http://europepmc.org/abstract/MED/34703837

Type: Journal Article/Review

Volume: 23

Parent Publication: Molecular therapy. Methods & clinical development

ISSN: 2329-0501