Research Objective This research will discover whether transplantation of stem cell-derived microglia can be used to treat Sanfilippo syndrome, a devastating and currently untreatable childhood neurological disease. Impact If successful,…
Research Objective The candidate is a fixed dose binary small molecule drug combination, consisting of two agents that act synergistically on a multipotent stem cell population in the CNS to…
Research Objective We propose to develop and validate a therapy for spinal cord injuries in which human stem cell-derived neural cells is injected into the injured spinal cord using an…
Research Objective The goal of this grant is to develop a gene therapy for a rare painful disorder, Inherited Erythromelalgia (IEM). Impact There are currently no FDA approved drugs for…
Research Objective A drug-stem cell combination therapy wherein the drug will direct and promote the delivery and distribution of stem cells to the disease site for the optimal therapeutic effect…
Research Objective AAV9-Cas13 gene therapy for Angelman syndrome using a first-in-kind mechanism of action that will safely and permanently restore expression of endogenous UBE3A that is deficient in CNS neurons.…
Research Objective These preclinical studies will discover the efficacy of stem cell-derived, nanoscale, extracellular vesicles (candidate) to treat adverse effects of cancer therapy on brain function and cognition. Impact Stem…
Research Objective This grant proposes development of a stem cell based therapy that is derived from human induced pluripotent stem cells. These cells are in the form of a brain…
Research Objective A cell therapy product comprised of inhibitory neurons that can migrate, integrate and restore neurologic function after traumatic brain injury. Impact Traumatic brain injury Major Proposed Activities Examine…
Research Objective We propose to investigate the transplantation of pluripotent stem cell derived microglia as a potential therapy for the devastating neurological disease; Adult-onset leukoencephalopathy (ALSP/HDLS). Impact The most immediately…
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