Longitudinal study revealing motor, cognitive and behavioral decline in a transgenic minipig model of Huntington's disease.

ABSTRACT: Huntington's disease (HD) is an inherited devastating neurodegenerative disease with no known cure to date. Several therapeutic treatments for HD are in development, but their safety, tolerability and efficacy need to be tested before translation to bedside. The monogenetic nature of this disorder has enabled the generation of transgenic animal models carrying a mutant huntingtin (mHTT) gene causing HD. A large animal model reflecting disease progression in humans would be beneficial for testing the potential therapeutic approaches. Progression of the motor, cognitive and behavioral phenotype was monitored in transgenic Huntington's disease minipigs (TgHD) expressing the N-terminal part of human mHTT. New tests were established to investigate physical activity by telemetry, and to explore

SUBMITTER: Baxa M 

PROVIDER: S-EPMC6918771 | biostudies-literature | 2019 Dec

REPOSITORIES: biostudies-literature