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...
Main Authors: | , , , , , , , , |
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Formato: | Artigo |
Idioma: | English |
Publicado: |
Elsevier
2023-01-01
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Series: | HGG Advances |
Subjects: | |
Acceso en liña: | http://www.sciencedirect.com/science/article/pii/S266624772200063X |