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)…
Research Objective The objective of this research proposal is to develop a lead AAV candidate for an optogenetic vision restoration therapy for patients suffering from blindness due to loss of…
Research Objective Genetically modified cells that help control immune reactions Impact Improved treatment for patients undergoing transplantation Major Proposed Activities To identify the optimal source of regulatory T cells for…
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 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…
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