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 The objective of this research is to define the final therapeutic candidate for effective hematopoietic stem cell gene therapy to treat severe alpha thalassemia that requires life-ling transfusions…
Research Objective The candidate is a gene therapy that delivers cardiac reprogramming factors to convert resident cardiac fibroblasts into functioning cardiac muscle. Thus, it is a regenerative cardiac gene therapy.…
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…
Research Objective The expected outcome is an exhaustion-resistant CAR-T cell, which persists long-term in a functional progenitor T cell state in the tumor microenvironment and can be used for cancer…
Research Objective We develop a novel adeno-associated viral (AAV) vector-delivered RNA-targeting therapeutic for elimination of toxic RNA causative of Huntington’s disease. Impact There are no disease-modifying therapies for Huntington’s disease.…
Research Objective This research will discover whether transplantation of stem cell-derived microglia can be used to treat Sanfilippo syndrome, a devastating and currently untreatable childhood neurological disease. Impact If successful,…
Research Objective AAV-SPL 2.0 is a gene therapy cure for SPLIS, a lethal childhood disorder of metabolism that causes kidney failure. Our gene therapy may also work in more common…
Research Objective We will use integrated gene editing techniques to develop a new CAR-T cell therapy for multiple myeloma treatment Impact Develop an improved CAR-T cell therapy for patients with…
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