AAV5-miHTT Gene Therapy Demonstrates Sustained Huntingtin Lowering and Functional Improvement in Huntington Disease Mouse Models-Reference-Cited by-同舟云学术

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AAV5-miHTT Gene Therapy Demonstrates Sustained Huntingtin Lowering and Functional Improvement in Huntington Disease Mouse Models

Published:2019-06 Issue: Volume:13 Page:334-343
ISSN:2329-0501
Container-title:Molecular Therapy - Methods & Clinical Development
language:en
Short-container-title:Molecular Therapy - Methods & Clinical Development

Author:

Spronck Elisabeth A.,Brouwers Cynthia C.,Vallès Astrid,de Haan Martin,Petry Harald,van Deventer Sander J.,Konstantinova Pavlina,Evers Melvin M.

Publisher

Elsevier BV

Subject

Genetics,Molecular Biology,Molecular Medicine

Reference35 articles.

1. Huntington’s disease: from molecular pathogenesis to clinical treatment;Ross;Lancet Neurol.,2011

2. Huntington disease;Vonsattel;J. Neuropathol. Exp. Neurol.,1998

3. Huntington’s disease: new frontiers for molecular and cell therapy;Melone;Curr. Drug Targets,2005

4. Sustained therapeutic reversal of Huntington’s disease by transient repression of huntingtin synthesis;Kordasiewicz;Neuron,2012

5. Huntington disease: natural history, biomarkers and prospects for therapeutics;Ross;Nat. Rev. Neurol.,2014

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