Article
Allele-Specific Knockdown of Mutant Huntingtin Protein via Editing at Coding Region Single Nucleotide Polymorphism Heterozygosities.
Human gene therapy - 1 Jan 2022
Oikemus Sarah R, Pfister Edith L, Sapp Ellen, Chase Kathryn O, Kennington Lori A, Hudgens Edward, Miller Rachael, Zhu Lihua Julie, Chaudhary Akanksh, Mick Eric O, Sena-Esteves Miguel, Wolfe Scot A, DiFiglia Marian, Aronin Neil, Brodsky Michael H
Abstract excerpt
Huntington's disease (HD) is a devastating, autosomal dominant neurodegenerative disease caused by a trinucleotide repeat expansion in the huntingtin (HTT) gene. Inactivation of the mutant allele by clustered regularly interspaced short palindromic repeats (CRISPR)-Cas9 based gene editing offers a possible therapeutic approach for this disease, but permanent disruption of normal HTT function might compromise...
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