Safety, efficacy and biodistribution of an engineered hepatotropic Adeno-Associated Virus mediated gene therapy for Ornithine Transcarbamylase Deficiency in Cynomolgus monkeys

Published 2021 View Full Article

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Title
Safety, efficacy and biodistribution of an engineered hepatotropic Adeno-Associated Virus mediated gene therapy for Ornithine Transcarbamylase Deficiency in Cynomolgus monkeys
Authors
Keywords
adeno-associated virus, ornithine transcarbamylase deficiency, engineered capsid, biodistribution, liver, AAV, AAVLK03, non-human primates, cynomolgus macaque, immune response
Journal
Molecular Therapy-Methods & Clinical Development
Volume -, Issue -, Pages -
Publisher
Elsevier BV
Online
2021-09-14
DOI
10.1016/j.omtm.2021.09.005

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