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
Published in:Mol Ther Methods Clin Dev
Main Authors: Svrzikapa, Nenad, Longo, Kenneth A., Prasad, Nripesh, Boyanapalli, Ramakrishna, Brown, Jeffrey M., Dorset, Daniel, Yourstone, Scott, Powers, Jason, Levy, Shawn E., Morris, Aaron J., Vargeese, Chandra, Goyal, Jaya
Format: Artigo
Language:Inglês
Published: American Society of Gene & Cell Therapy 2020
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Original Article
Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC7648085/
https://ncbi.nlm.nih.gov/pubmed/33209959
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2020.09.003
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