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Article

Permanent inactivation of Huntington's disease mutation by personalized allele-specific CRISPR/Cas9.

Human molecular genetics - 15 Oct 2016

Shin Jun Wan, Kim Kyung-Hee, Chao Michael J, Atwal Ranjit S, Gillis Tammy, MacDonald Marcy E, Gusella James F, Lee Jong-Min

Abstract excerpt

A comprehensive genetics-based precision medicine strategy to selectively and permanently inactivate only mutant, not normal allele, could benefit many dominantly inherited disorders. Here, we demonstrate the power of our novel strategy of inactivating the mutant allele using haplotype-specific CRISPR/Cas9 target sites in Huntington's disease (HD), a late-onset neurodegenerative disorder due to a toxic dominant...

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Identifiers

PMID: 28172889
DOI: 10.1093/hmg/ddw286
PMCID: PMC6078600

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