A Comprehensive Haplotype Targeting Strategy for Allele-Specific HTT Suppression in Huntington Disease.
Sign Up to like & getrecommendations! Published in 2019 at "American journal of human genetics"
DOI: 10.1016/j.ajhg.2019.10.011
Abstract: Huntington disease (HD) is a fatal neurodegenerative disorder caused by a gain-of-function mutation in HTT. Suppression of mutant HTT has emerged as a leading therapeutic strategy for HD, with allele-selective approaches targeting HTT SNPs now… read more here.
Keywords: comprehensive haplotype; htt; allele specific; strategy ... See more keywords