AAV5-miHTT Gene Therapy Demonstrates Broad Distribution and Strong Human Mutant Huntingtin Lowering in a Huntington’s Disease Minipig Model-Reference-Cited by-同舟云学术

AAV5-miHTT Gene Therapy Demonstrates Broad Distribution and Strong Human Mutant Huntingtin Lowering in a Huntington’s Disease Minipig Model

Published:2018-09 Issue:9 Volume:26 Page:2163-2177
ISSN:1525-0016
Container-title:Molecular Therapy
language:en
Short-container-title:Molecular Therapy

Author:

Evers Melvin M.,Miniarikova Jana,Juhas Stefan,Vallès Astrid,Bohuslavova Bozena,Juhasova Jana,Skalnikova Helena Kupcova,Vodicka Petr,Valekova Ivona,Brouwers Cynthia,Blits Bas,Lubelski Jacek,Kovarova Hana,Ellederova Zdenka,van Deventer Sander J.,Petry Harald,Motlik Jan,Konstantinova Pavlina

Funder

PIGMOD Center’s

National Sustainability Programme

Czech Ministry of Education, Youth and Sports

CHDI Foundation

Publisher

Elsevier BV

Subject

Drug Discovery,Pharmacology,Genetics,Molecular Biology,Molecular Medicine

Reference72 articles.

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2. Huntington’s disease (HD): the neuropathology of a multisystem neurodegenerative disorder of the human brain;Rüb;Brain Pathol.,2016