Intracerebral Gene Therapy Using AAVrh.10-hARSA Recombinant Vector to Treat Patients with Early-Onset Forms of Metachromatic Leukodystrophy: Preclinical Feasibility and Safety Assessments in Nonhuman Primates

Title
Intracerebral Gene Therapy Using AAVrh.10-hARSA Recombinant Vector to Treat Patients with Early-Onset Forms of Metachromatic Leukodystrophy: Preclinical Feasibility and Safety Assessments in Nonhuman Primates
Authors
Keywords
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Journal
Human Gene Therapy Clinical Development
Volume 26, Issue 2, Pages 113-124
Publisher
Mary Ann Liebert Inc
Online
2015-03-11
DOI
10.1089/humc.2014.139

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