Human Stem Cell Use: iPS Cell
Repurposing Aminoadamantane Nitrate Compounds to Inhibit SARS-CoV-2 Viral Activity and to Protect the Brain from Viral-Related Damage
Research Objective The objective is to screen a series of aminoadamantane nitrate compounds for their ability to protect hiPSC-derived neurons from SARS-CoV-2-related damage and to block SARS-CoV-2 activity. Impact If successful, our screen would identify a drug candidate for further development that would protect neurons from SARS-CoV-2-related damage and also inhibit SARS-CoV-2 activity. Major Proposed […]
Identifying a lead compound for COVID19 using high throughput screening with lung stem cell organoids
Research Objective We propose to use a lung stem cell based organoid to identify a new compound for COVID-19 by screening a library of FDA approved compounds that could be repurposed for COVID-19 infection. Impact If successful, we will find a therapy to treat COVID-19 infection and prevent the lung complications that are so deadly. […]
Safety “Clinical Trial” of the Cardiac Liability of COVID19 Polytherapy
Autologous therapy for Parkinson’s disease: single cell RNAseq for in depth characterization of transplanted cells
Gene-corrected human microglia for the treatment of adult onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP)
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 NGL-101 will result in a life-altering therapy for patients suffering from ALSP and provide proof of concept for treating other microglia diseases. Major Proposed Activities […]
Inhibitory interneurons derived from human induced pluripotent stem cells to treat stroke
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 survival, differentiation, and minimal migration within the peri-infarct brain region and were unable to restore neurological functions after stroke. Major Proposed Activities MILESTONE 1: Determine […]
Drug discovery for gastrointestinal motility disorders using hPSC-derived enteric ganglioids
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 motility disorders including chronic constipation, achalasia and gastroparesis. Major Proposed Activities Assessment of candidate compounds on stem cell derived models from diverse genetic backgrounds Evaluation […]
Development of a VAV2 antisense oligonucleotide (ASO) treatment for ALS
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 Activities Compare the in vitro efficacy of up to 10 lead candidate VAV2 ASOs to rescue the survival of iNs from 30 locally and nationally […]
C9orf72 repeat expansion-tuned allelic suppression by CRISPRi as an ALS therapy
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 can be administered intravenously once, have long lasting effects, and is indicated for all ALS patients who carry repeat expansions of varying lengths and toxicity […]
Treatment of Myasthenic Syndrome due to Choline Acetyltransferase Deficiency Using AAV9-mediated Gene 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) gene. Impact Treatment of an incurable disease in infants and potential amelioration of symptoms of neuro degenerative diseases with deficient cholinergic system such as Alzheimer […]