Research Objective We propose to discover genome- and epigenome-edited allogeneic T cells engineered to selectively target JCV as a potentially lifesaving treatment for progressive multifocal leukoencephalopathy (PML). Impact If successful,…
Research Objective Use human cell therapy insights, specifically CDC-secreted EV analysis, to develop a noncoding RNA chemical entity for myocarditis treatment. Impact Key knowledge gap is how to recruit adaptive…
Research Objective Our candidate product PSCA-CAR_s15 uiNK is derived from transduction of iPSCs selected from the most ideal source and episomally reprogrammed from mature NK cells or CD34+ cells. Impact…
Research Objective A Treatment for Artemis-deficient Severe Combined Immunodeficiency using Non-Viral CRISPR-driven Safe Harbor Transgenesis in Hematopoietic Stem Cells Impact We aim to develop a novel genome editing based therapy…
Research Objective To develop a lipid nanoparticle/mRNA complex that can safely and efficiently edit muscle stem cells in utero, correct the dystrophin mutation, and develop a treatment for Duchenne muscular…
Research Objective The objective is to define a final therapeutic candidate for an effective gene therapy for mutations of the creatine transporter SLC6A8, a major cause of X-linked intellectual disability…
Research Objective To develop a Stem Cell therapy for Sanfilippo B syndrome. Impact There is no treatment for Sanfilippo syndrome, and other therapeutic approaches have failed in clinics. This proposal…
Research Objective Our therapeutic candidate is a lipid nanoparticle that delivers a therapeutic dose of mRNA to the human heart, which transiently transfects of cells within the heart to improve…
Research Objective Functional restoration following spinal cord injury using defined excitatory and inhibitory spinal interneuron progenitor cell transplantation Impact The development of a stem cell derived progenitor cell therapy with…
Research Objective Efficacious and safe intramyocardial delivery of modified mRNA encoding cell cycle regulators as a gene therapy for cardiac regeneration through resident cardiomyocyte proliferation. Impact This project would provide…
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