Ontology highlight
ABSTRACT:
SUBMITTER: Yang S
PROVIDER: S-EPMC5490741 | biostudies-literature | 2017 Jun
REPOSITORIES: biostudies-literature
Yang Su S Chang Renbao R Yang Huiming H Zhao Ting T Hong Yan Y Kong Ha Eun HE Sun Xiaobo X Qin Zhaohui Z Jin Peng P Li Shihua S Li Xiao-Jiang XJ
The Journal of clinical investigation 20170619 7
Huntington's disease is a neurodegenerative disorder caused by a polyglutamine repeat in the Huntingtin gene (HTT). Although suppressing the expression of mutant HTT (mHTT) has been explored as a therapeutic strategy to treat Huntington's disease, considerable efforts have gone into developing allele-specific suppression of mHTT expression, given that loss of Htt in mice can lead to embryonic lethality. It remains unknown whether depletion of HTT in the adult brain, regardless of its allele, cou ...[more]