Therapeutic/Technology: Cell delivery or targeting

Overcoming barriers for airway stem cell gene therapy for Cystic Fibrosis

Research Objective This research will allow the targeting of airway stem cells for long lived gene therapy for Cystic Fibrosis and for other airway diseases Impact We will overcome the barriers to accessing airway basal stem cells for gene correction for Cystic Fibrosis (CF) and use a new gene correction strategy to correct >99% of […]

Developing a microglia replacement therapy

Research Objective To develop a new cell therapy for the brain by transplanting the brain's immune cells Impact The brain cell therapy we envision could be used to treat rare genetic metabolic diseases, Multiple Sclerosis, brain tumors, as well as common neurodegenerative diseases including Alzheimer's disease. Major Proposed Activities Finding the best source of human […]

Engineering AAV capsids for transduction of neural and muscle stem cells

Research Objective The studies will identify and characterize new gene therapy vectors able to deliver gene editing components to stem cells. to enable treatment of diseases involving both muscle and brain. Impact New AAV capsids that target muscle and neural stem cells will enhance the number of neurological diseases able to be treated with AAV-based […]

Development of Anti-COVID RNAi Therapeutics Using Human iPSC-Derived Alveolar Epithelial Cells

Research Objective To optimize a new approach to deliver Anti-COVID siRNAs into human iPSC-derived lung cells that can selectively kill the COVID virus Impact Our proposal, if successful, will solve the siRNA delivery problem and rapidly open the door to Anti-COVID siRNA therapeutics. Major Proposed Activities Complete synthesis of a new delivery device called a […]

Prodrug innovation to target muscle stem cells and enhance muscle regeneration

Research Objective To target therapeutics to muscle stem cells, the building blocks of skeletal muscle. Impact Drugs, genes and gene editing strategies can be delivered directly to muscle stem cells to alleviate disease. Major Proposed Activities Synthetic peptides based on the ectodomains of Myomaker will be synthesized, with a fluorophore conjugated for tracking. Alternatively, anti-Myomaker […]

Novel Platforms to Enhance In Vivo Delivery of Skeletal Muscle Progenitor Cells from Human Pluripotent Stem Cells

Research Objective Delivery of muscle stem cells presents a major roadblock for therapy. We explore novel approaches to increase the efficiency of delivering and monitoring muscle stem cells derived from hPSCs. Impact Development of enhanced monitoring and delivery platforms will greatly accelerate translational strategies aimed at delivering muscle stem cells for transplantation to patients with […]

Injectable Hydrogels for the Delivery, Maturation, and Engraftment of Clinically Relevant Numbers of Human Induced Pluripotent Stem Cell-Derived Neural Progenitors to the Central Nervous System

One critical bottleneck in the translation of regenerative medicine into the clinic is the efficient delivery and engraftment of transplanted cells. While direct injection is the least invasive method for cell delivery, it commonly results in the survival of only 5-20% of cells. Studies suggest that delivery within a carrier gel may enhance cell viability, […]

Identification and isolation of transplantable human hematopoietic stem cells from pluripotent cell lines; two steps from primitive hematopoiesis to transplantable definitive cells, and non-toxic conditioning of hosts for hematopoeitic stem cell transp…

A goal of stem-cell therapy is to transplant into a patient “tissue-specific” stem cells, which can regenerate a particular type of healthy tissue (e.g., heart or blood cells). A major obstacle to this goal is obtaining tissue-specific stem cells that (1) are available in sufficient numbers; and (2) will not be rejected by the recipient. […]

Development of a clinical-grade extracorporeal liver support system using human induced pluripotent stem cell-derived hepatic cells

Liver failure is the fourth leading cause of adult death in California. Because liver cells can regenerate, some patients with liver failure could be saved without having to undergo organ transplantation if their liver function could be supported temporarily. Here, we propose to develop a device to support these patients called the “extracorporeal liver support […]