Molecular Characterization of hESC and hIPSC-Derived Spinal Motor Neurons
One of the main objectives of stem cell biology is to create physiologically relevant cell types that can be used to either facilitate the study of or directly treat human…
Defining the molecular mechanisms of somatic cell reprogramming
The development of methods to “reprogram” adult cells such as skin cells by simultaneously expressing four specific factors — Oct3/4, Sox2, c-Myc and Klf4 — in order to create cells…
Molecular Mechanisms of Reprogramming towards Pluripotency
Stem cell biology and its applications to cell-based therapies, since its inception 30 years ago, has been hindered by the immunological considerations of rejection of non-autologous cells in patients, as…
Developing a universal CRISPR gene therapy approach to treat C9orf72 ALS
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…
Drug discovery for Charcot Marie Tooth Disease using hPSC-derived Schwann cells
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…
Modulating cardiac myosin heavy chain isoform expression for treating cardiomyopathies
Research Objective To discover CRISPR-based genetic strategies that will directly and therapeutically modify cardiac myosin heavy chain isoform expression to treat HF and cardiomyopathies. Impact Heart Failure and Cardiomyopathies Major…
Human induced pluripotent stem cells-derived glial enriched progenitors for the treatment of mild traumatic brain injury
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…
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…
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…
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…
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