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

Summary: 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...

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Detalles Bibliográficos
Main Authors: Li Fang, Alex Mas Monteys, Alexandra Dürr, Megan Keiser, Congsheng Cheng, Akhil Harapanahalli, Pedro Gonzalez-Alegre, Beverly L. Davidson, Kai Wang
Formato: Artigo
Idioma:English
Publicado: Elsevier 2023-01-01
Series:HGG Advances
Subjects:
Acceso en liña:http://www.sciencedirect.com/science/article/pii/S266624772200063X