New Cell Lines for Huntington’s Disease
Huntington’s disease (HD) is a devastating neurodegenerative disease with a 1/10,000 disease risk that always leads to death. These numbers do not fully reflect the large societal and familial cost…
MSC engineered to produce BDNF for the treatment of Huntington’s disease
A team at UC Davis plans to use bone marrow derived mesenchymal stem cells to deliver a growth factor, called BDNF, to patients' brains in order to reduce the death…
Control of OCT4 abundance and function in human stem cells
Research Objective Our work will reveal an essential pathway that establishes precise levels of the OCT4 transcription factor and protects stem cell identity despite genetic or environmental stress. Impact Our…
RNA-directed therapy for Huntington’s disease
Research Objective We develop a novel adeno-associated viral (AAV) vector-delivered RNA-targeting therapeutic for elimination of toxic RNA causative of Huntington’s disease. Impact There are no disease-modifying therapies for Huntington’s disease.…
Discovery of therapeutics for Huntington’s Disease
Research Objective The objective of the proposed research is to perform 3 independent hESC-based screens to identify drug candidates for Huntington’s Disease. Impact There are currently no effective treatments for…
hNSC-mediated delivery of ApiCCT1 as a candidate therapeutic for Huntington’s disease
Research Objective The therapeutic candidate is a human Neural Stem Cell that secretes a protein, ApiCCT1, that aids in the prevention of disease phenotypes, for application in treatment of Huntington's…
An hESC-derived hNSC Therapeutic for Huntington’s Disease
Therapeutic Candidate or Device The therapeutic candidate is a human Neural Stem Cell product to prevent or delay disease symptoms for treatment of Huntington's disease (HD). Indication Huntington’s disease, a…
Frances Saldaña: Huntington’s Disease Patient Advocate
n a span of seven years, Frances Saldaña lost her three children to Huntington's disease, a devastating neurodegenerative disease that has no cure. Despite her suffering, Saldaña continues to advocate…
