Investigational Assay for Haplotype Phasing of the Huntingtin Gene

Investigational Assay for Haplotype Phasing of the Huntingtin Gene

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) repeat. WVE-120101 and WVE-120102 are investigation...

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Bibliographic Details
Main Authors: Nenad Svrzikapa, Kenneth A. Longo, Nripesh Prasad, Ramakrishna Boyanapalli, Jeffrey M. Brown, Daniel Dorset, Scott Yourstone, Jason Powers, Shawn E. Levy, Aaron J. Morris, Chandra Vargeese, Jaya Goyal
Format: Article
Language:English
Published: Elsevier 2020-12-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
antisense oligonucleotide
Huntington’s disease
long-read sequencing
mRNA
single-nucleotide polymorphism
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050120301881

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http://www.sciencedirect.com/science/article/pii/S2329050120301881

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