Efficient and Precise Processing of the Optimized Primary Artificial MicroRNA in a Huntingtin-Lowering Adeno-Associated Viral Gene Therapy In Vitro and in Mice and Nonhuman Primates
Author:
Affiliation:
1. Voyager Therapeutics, Cambridge, Massachusetts, USA
2. Sanofi-Genzyme, Framingham, Massachusetts, USA.
Publisher
Mary Ann Liebert Inc
Subject
Genetics,Molecular Biology,Molecular Medicine
Link
https://www.liebertpub.com/doi/pdf/10.1089/hum.2021.221
Reference59 articles.
1. Regional Specificity of Brain Atrophy in Huntington's Disease
2. Huntington's disease: from molecular pathogenesis to clinical treatment
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4. Sustained effects of nonallele-specificHuntingtinsilencing
5. Sustained Therapeutic Reversal of Huntington's Disease by Transient Repression of Huntingtin Synthesis
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