Therapeutic Candidate or Device Autologous blood stem cells edited to restore iduronidase expression Indication Severe Mucopolysaccharidosis Type 1 (MPS1/ Hurler's syndrome) Therapeutic Mechanism Autologous blood stem cells undergo genome editing…
Therapeutic Candidate or Device Transduced Hematopoietic Stem Cells from Peripheral Blood Stem Cells of adults and pediatric patients with cystinosis Indication Autologous hematopoietic stem cell gene therapy for patients with…
Therapeutic Candidate or Device hESC-derived pancreatic progenitor cells delivered in a device that allows direct vascularization of the cell therapy Indication high-risk type 1 diabetes including "brittle" diabetes and hypoglycemia…
The goal of our CIRM-funded Early Translational (ETA) grant was to engineer a product to improve healing in diabetic foot ulcers, a devastating consequence of diabetes that occurs in about…
We are developing a stem cell-derived replacement cell therapy for insulin-requiring diabetes. Through a process known as directed differentiation, embryonic stem cells are turned into pancreatic cells in the laboratory.…
The intestine performs the essential function of absorbing food and water into the body. Without a functional intestine, children and adults cannot eat normal meals, and these patients depend on…
The intestine performs the essential function of absorbing food and water into the body. Without a functional intestine, children and adults cannot eat normal meals, and these patients depend on…
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…
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