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…
Research Objective We propose to discover a tool that will utilize patient specific iPSC-derived human mini-guts to identify personalized antifibrotic treatments in pediatric Crohn’s disease patients Impact The major bottleneck…
Research Objective This proposal will deliver a small molecule therapeutic candidate for the treatment of Myelodysplastic Syndromes and will act by inducing differentiation on mutated hematopoietic stem cells. Impact This…
Research Objective We propose a gene therapy for the treatment of a severe infantile epilepsy called CDKL5 Deficiency Disorder using CRISPR-mediated epigenetic editing Impact A transformative treatment for females affected…
Research Objective A new therapeutic option for DOCK8 deficiency using autologous human hematopoietic stem cells modified through either lentiviral gene addition or CRISPR/Cas9 based gene editing. Impact Allogeneic HSCT is…
Research Objective Locally delivered formulation of RSpo1 protein as an activator of Lgr5+ epithelial stem cells in chemotherapy- or radiation therapy-induced oral mucositis Impact Oral mucositis Major Proposed Activities RSpo1…
Research Objective Develop a stem cell-based platform that safely and efficiently delivers viruses that specifically kill tumor cells and restore immune activity in patients with advanced cancer. Impact Overcome the…
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…
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