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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Detalles Bibliográficos
Autores principales: 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
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2020
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7648085/
https://www.ncbi.nlm.nih.gov/pubmed/33209959
http://dx.doi.org/10.1016/j.omtm.2020.09.003

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7648085/
https://www.ncbi.nlm.nih.gov/pubmed/33209959
http://dx.doi.org/10.1016/j.omtm.2020.09.003

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