Article
Allele-specific suppression of mutant huntingtin using antisense oligonucleotides: providing a therapeutic option for all Huntington disease patients.
PloS one - 1 Jan 2014
Skotte Niels H, Southwell Amber L, Østergaard Michael E, Carroll Jeffrey B, Warby Simon C, Doty Crystal N, Petoukhov Eugenia, Vaid Kuljeet, Kordasiewicz Holly, Watt Andrew T, Freier Susan M, Hung Gene, Seth Punit P, Bennett C Frank, Swayze Eric E, Hayden Michael R
Abstract excerpt
Huntington disease (HD) is an inherited, fatal neurodegenerative disorder caused by a CAG repeat expansion in the huntingtin gene. The mutant protein causes neuronal dysfunction and degeneration resulting in motor dysfunction, cognitive decline, and psychiatric disturbances. Currently, there is no disease altering treatment, and symptomatic therapy has limited benefit. The pathogenesis of HD is complicated and...
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