Research Objective We propose to evaluate both gene addition with viral vectors and CRISPR-based gene editing of a patient's own bone marrow stem cells as a curative therapy for autosomal…
Research Objective This DISC2 tests a strategy to target survival and self-renewal in glioma stem cells (GSC), and will identify a therapeutic candidate for safety, dosing and efficacy testing. Impact…
Research Objective This proposal will develop a cell-based therapy that can restore vision in retinal degeneration associated blindness such as Stargardt disease and age-related macular degeneration. Impact Chemically induced method…
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 Allogeneic Human induced pluripotent stem cells-derived inhibitory interneuron therapy product encapsulated in a hyaluronan/VEGF nanoparticle hydrogel for the treatment of stroke Impact Previous stem cell-based technologies had poor…
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…
Research Objective Our goal is to use stem cell models to identify a small molecule drug candidate for GI motility disorders. Impact The candidate will be used for severe gastrointestinal…
Research Objective Patient specific stem cells (iPSCs) to model ALS and identify a broadly acting therapeutic intervention Impact Heterogenous patient population, dire need for broadly acting therapeutic interventions Major Proposed…
Research Objective We aim to discover an adeno-associated viral (AAV) CRISPRi gene therapy for amyotrophic lateral sclerosis (ALS) patients with hexanucleotide repeat expansions in the C9orf72 gene. Impact This therapy…
Research Objective Use of a viral vector to transport a normal gene to children with severe motor and respiratory disabilities caused by an inborn defect of the choline acetyltransferase (ChAT)…
