Research Objective The origins of brain vascular mural cells are unknown. This proposal will identify mural stem cells in the developing human and mouse brain and determine their impact on…
Research Objective The research objectives are to identify causal genes for chemotherapy-induced mitochondrial toxicity and neurodegeneration in sensory neurons and drugs that target this toxicity. Impact These studies will open…
Research Objective We will develop the first human stem cell model of sporadic ALS (sALS) to identify disease mechanisms in the most common form of ALS and to discover drugs…
Research Objective To develop a new cell therapy for the brain by transplanting the brain's immune cells Impact The brain cell therapy we envision could be used to treat rare…
Research Objective We are developing a strategy to characterize the disease-relevance of hundreds of mutations across diverse genetic backgrounds using stem cells Impact Understanding how mutations impact cellular function can…
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…
Research Objective Development of a CRISPR genome editing therapy for ALS caused by C9orf72 and the delivery vehicle required to bring this novel therapy to patients. Impact A cure for…
Research Objective The therapeutic candidate we aim to discover under this award is clomipramine, an antidepressant that might help reduce a harmful protein in the nerve cells of patients with…
Research Objective Allogeneic human induced pluripotent stem cell-derived glial enriched progenitor cell therapy to treat mild traumatic brain injury Impact Prior to this study, glial cell-based therapies have never been…
Research Objective The research proposed here will support the development of NGL-101, a cell therapy for treating adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP). Impact Successful development of…
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