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 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 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 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…
Research Objective We will empower stem cell biologists to generate iPSC-derived neurons faster and with enhanced maturation by enabling optical cell stimulation and triggering activity-dependent maturation processes Impact Our project…
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 We propose to discovery and evaluate antisense gene therapy for specific mutations underlying debilitating or life-threatening neurodevelopmental diseases including epilepsy and autism syndromes. Impact The conditions are four…
Research Objective We will screen for modifiers of the response to misfolded αSyn and Aβ, and their cognate antibodies. Development of drugs to combat this inflammation is important in neurodegenerative…
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