Article
A novel humanized mouse model of Huntington disease for preclinical development of therapeutics targeting mutant huntingtin alleles.
Human molecular genetics - 15 Mar 2017
Southwell Amber L, Skotte Niels H, Villanueva Erika B, Østergaard Michael E, Gu Xiaofeng, Kordasiewicz Holly B, Kay Chris, Cheung Daphne, Xie Yuanyun, Waltl Sabine, Dal Cengio Louisa, Findlay-Black Hailey, Doty Crystal N, Petoukhov Eugenia, Iworima Diepiriye, Slama Ramy, Ooi Jolene, Pouladi Mahmoud A, Yang X William, Swayze Eric E, Seth Punit P, Hayden Michael R
Abstract excerpt
Huntington disease (HD) is a neurodegenerative disease caused by a mutation in the huntingtin (HTT) gene. HTT is a large protein, interacts with many partners and is involved in many cellular pathways, which are perturbed in HD. Therapies targeting HTT directly are likely to provide the most global benefit. Thus there is a need for preclinical models of HD recapitulating human HTT genetics. We previously...
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