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Autor:
Daniel Dorset, Ramakrishna Boyanapalli, Kenneth Longo, Jeffrey M. Brown, Aaron J. Morris, Nripesh Prasad, Scott Yourstone, Shawn Levy, Chandra Vargeese, Nenad Svrzikapa, Jaya Goyal, Jason Powers
Publikováno v:
Molecular Therapy. Methods & Clinical Development
Molecular Therapy: Methods & Clinical Development, Vol 19, Iss, Pp 162-173 (2020)
Molecular Therapy: Methods & Clinical Development, Vol 19, Iss, Pp 162-173 (2020)
Novel treatments for Huntington’s disease (HD), a progressive neurodegenerative disorder, include selective targeting of the mutant allele of the huntingtin gene (mHTT) carrying the abnormally expanded disease-causing cytosine-adenine-guanine (CAG)