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…
Research Objective Generating human liver using pig as a bioreactor Impact The livers produced will be used for transplantation into patients with end-stage liver disease, metabolic disorders, and for metastatic…
Research Objective The primary objective of this research is to test whether excitatory human V2a spinal interneurons engineered from PSCs can repair the damaged spinal cord and restore motor function.…
Research Objective We propose to develop a bio-tendon engineered from differentiated pluripotent stem cells for the repair of tendon injuries and degeneration. Impact Rotator cuff tears are the most common…
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 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 We will generate nanoprobe-containing stem cell-derived human beta cells that can be monitored in real time in response to inflammatory stress upon transplantation in patients with type 1…
Research Objective We will develop a gene therapy for a major inherited optic nerve disease and test the effectiveness of the treatment by analyzing healthy and patient stem cell-derived mini…
Research Objective Determine optimal islet transplant conditions and systemic treatments that promote graft survival upon transplantation into immune-competent diabetic subjects. Impact Our proposal will optimize the generation and viability of…
Research Objective Conditioned media from human iPSC-derived smooth muscle cell progenitors. This media exerts paracrine effect to restore damaged vaginal wall in patients with pelvic organ prolapse. Impact Pelvic organ…
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