Mentor: Dr. Julien Rossignol
Combination Stem Cell Therapy for Huntington’s Disease
Huntington’s disease (HD) is a fatal neurodegenerative disease caused by an unstable, extended CAG trinucleotide repeat that causes progressive degeneration of medium spiny neurons in the brain. There is currently no known cure or effective treatment for this devastating disease. Cell replacement therapy has been studied as a potential treatment for the symptoms of HD in rodent models. In the present study, induced pluripotent stem cells and mesenchymal stem cells (MSCs) will be genetically engineered to over express Human BDNF (hBDNF) and co-transplanted into the striata of R6/2 mice to access potential therapeutic effects. The purpose of this study is therefore to confirm the therapeutic efficacy and functional recovery of bone marrow-derived-MSCs genetically engineered to over express hBDNF in the R6/2 mouse model of HD.