Haplotyping SNPs for allele-specific gene editing of the expanded huntingtin allele using long-read sequencing

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Huntington’s disease (HD) is an autosomal dominant neurodegenerative disease caused by CAG trinucleotide repeat expansions in exon-1 of huntingtin (HTT). Currently, there is no cure for HD, and the clinical care of individuals with HD is focused on symptom management. Previously, we showed allele-sp...

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Detalles Bibliográficos
Autores principales: Fang, Li, Monteys, Alex Mas, Dürr, Alexandra, Keiser, Megan, Cheng, Congsheng, Harapanahalli, Akhil, Gonzalez-Alegre, Pedro, Davidson, Beverly L., Wang, Kai
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2022
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9574884/
https://www.ncbi.nlm.nih.gov/pubmed/36262216
http://dx.doi.org/10.1016/j.xhgg.2022.100146
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