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 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 will utilize human induced pluripotent stem cells derived from Duchenne muscular dystrophy (DMD) patients for drug testing and drug discovery for this rare genetic disease. Impact Diverse…
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 We aim to discover an injectable, rejuvenating treatment for painful intervertebral disc degeneration using microtissue-encapsulated iPSC-derived notochordal cells (iNCs) using large animal model Impact Our treatment candidate may…
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 We developed a novel glial-restricted neural progenitor cells transplantation strategy as a treatment for Rett syndrome, reverting neuronal alterations caused by genetic mutations. Impact There are no disease-modifying…
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…
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…
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