title: Therapeutic Effect of Berberine on Huntington's Disease Transgenic Mouse Model.
abstract: Huntington disease (HD) represents a family of neurodegenerative diseases that are caused by misfolded proteins. The misfolded proteins accumulate in the affected brain regions in an age-dependent manner to cause late-onset neurodegeneration. Transgenic mouse models expressing the HD protein, huntingtin, have been widely used to identify therapeutics that may retard disease progression. Here we report that Berberine (BBR), an organic small molecule isolated from plants, has protective effects on transgenic HD (N171-82Q) mice. We found that BBR can reduce the accumulation of mutant huntingtin in cultured cells. More importantly, when given orally, BBR could effectively alleviate motor dysfunction and prolong the survival of transgenic N171-82Q HD mice. We found that BBR could promote the degradation of mutant huntingtin by enhancing autophagic function. Since BBR is an orally-taken drug that has been safely used to treat a number of diseases, our findings suggest that BBR can be tested on different HD animal models and HD patients to further evaluate its therapeutic effects.
authors.affiliation: Department of Human Genetics, Emory University School of Medicine, 615 Michael Street, Atlanta, GA 30322, United States of America; Graduate Program of Microbiology and Molecular Genetics, Emory University, Atlanta, GA 30322, United States of America.
authors.forename: Wenxiao
authors.initials: W
authors.lastname: Jiang
authors.affiliation: Department of Human Genetics, Emory University School of Medicine, 615 Michael Street, Atlanta, GA 30322, United States of America; Department of Neurology, Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan 430032, China.
authors.forename: Wenjie
authors.initials: W
authors.lastname: Wei
authors.affiliation: Department of Human Genetics, Emory University School of Medicine, 615 Michael Street, Atlanta, GA 30322, United States of America.
authors.forename: Marta A
authors.initials: MA
authors.lastname: Gaertig
authors.affiliation: Department of Human Genetics, Emory University School of Medicine, 615 Michael Street, Atlanta, GA 30322, United States of America.
authors.forename: Shihua
authors.initials: S
authors.lastname: Li
authors.affiliation: Department of Human Genetics, Emory University School of Medicine, 615 Michael Street, Atlanta, GA 30322, United States of America.
authors.forename: Xiao-Jiang
authors.initials: XJ
authors.lastname: Li
mesh_terms: D000818:Animals; D001343:Autophagy / Q000187:drug effects; D001522:Behavior, Animal; D001599:Berberine / Q000627:therapeutic use*; D004195:Disease Models, Animal; D057809:HEK293 Cells; D006801:Humans; D006816:Huntington Disease / Q000188:drug therapy*; D051379:Mice; D008822:Mice, Transgenic
chemical_list: D001599:Berberine