ZINC FINGER NUCLEASE-BASED STEM CELL THERAPY FOR AIDS
Some years ago it was discovered that patients homozygous for a natural mutation (the Δ32 mutation) in the CCR5 gene are generally resistant to HIV infection by blocking virus entry…
Stem cell based treatment strategy for Age-related Macular Degeneration (AMD)
Retinal degeneration represents a group of blinding diseases that are increasingly impacting the health and well being of Californians. It is estimated that by 2020, over 450,000 Californians will suffer…
Development of Highly Active Anti-Leukemia Stem Cell Therapy (HALT)
Leukemias are cancers of the blood forming cells that afflict both children and adults. Many drugs have been developed to treat leukemias and related diseases. These drugs are often effective…
COVID-19 Antiviral Therapy to Block Direct Cell Injury and Associated Tissue Damage
Translational Candidate Berzosertib (VE-822), a safe drug candidate for treatment against COVID-19, will be investigated. Area of Impact The outcome of the proposed studies will have a significant health benefit…
Purification of Human Hematopoietic Stem Cells (HSCs) for Clinical Stem Cell Transplantation
Translational Candidate Two new monoclonal antibodies anti-CD34 and anti-CD90, and protocols to purify cancer-free and/or T cell free human HSC for clinical transplantation. Area of Impact The development of hematopoietic…
Development of a low-cost, clinical-grade iPS maintenance medium for enabling stem cell therapy manufacturing
Translational Candidate HiDef-B8, an iPS cell maintenance medium designed to reduce stem cell GMP manufacturing costs and risk. Area of Impact HiDef-B8 addresses scale-up manufacturing, by being lower-cost ($300/L) and…
Generation of human universal donor iPS cells
Translational Candidate Universal donor cell that is a genetically-engineered iPSC clone and is equipped with a safety switch. Area of Impact Regenerative medicine including replacement therapies affected by immune rejection…
T-Pure: Peripheral Blood Processing Tool for Point of Care CAR-T Manufacturing
Translational Candidate Tool kit added directly to peripheral blood allowing for purification of T cell enriched product suitable for (CAR)-T cell generation. Area of Impact The goal is to generate…
Cell Villages and Clinical Trial in a Dish with Pooled iPSC-CMs for Drug Discovery
Translational Candidate Human stem cells in a dish engineered into heart cells to supplement, refine, reduce, and/or ultimately replace human clinical trials. Area of Impact Increase genetic diversity of preclinical…
Human iPSC-derived micro-heart muscles for high-throughput cardiac drug discovery
Translational Candidate In vitro miniaturized array of heart muscle amenable for use in efficient high-throughput drug discovery and screening campaigns. Area of Impact Effective high-throughput screening of drugs on human…
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