The Children's Hospital Corporation, doing business as Boston Children's Hospital, received a $705,402 Project Grant award from the National Institute of Neurological Disorders and Stroke (NINDS) under the Extramural Research Programs in the Neurosciences and Neurological Disorders (CFDA 93.853) federal grant program. The award supports research to evaluate the efficacy and safety of an adeno-associated virus (AAV) gene therapy construct for treating succinic semialdehyde dehydrogenase deficiency (SSADHD), a rare genetic metabolic disorder. The project aims to measure SSADH restoration, neuronal function, and other disease-relevant endpoints in SSADHD patient-derived induced pluripotent stem cell (iPSC) neurons and an SSADHD mouse model. The goal is to determine if the AAV-based gene therapy can sufficiently restore SSADH activity and reverse the SSADHD phenotype, paving the way for a potential curative treatment for this unmet medical need.
Generated 3/4/25, 7:33 AM