Disease Focus: Metabolic Disorders

Development of immune invisible beta cells as a cell therapy for type 1 diabetes through genetic modification of hESCs

Research Objective Development of hESC-derived pancreatic beta cells that are protected from allogeneic and autoimmune attack into a cell therapy for type 1 diabetes (T1D) Impact Cell therapy of T1D is challenged by immune rejection. Therefore, we will develop pancreatic progenitors derived from genetically modified hESCs that can evade allogeneic and autoimmune responses. Major Proposed […]

Preclinical development of AAV vector-mediated in vivo hepatic reprogramming of myofibroblasts as a therapy for liver fibrosis

Research Objective An intravenously injectable virus that converts the scar cells responsible for liver cirrhosis into the cells that provide most of the liver’s function, thereby preventing or reversing liver failure. Impact The proposed research will develop a new therapy for liver cirrhosis, which can be cured by liver transplantation, but there are not enough […]

Designing a cellular niche for transplantation of human embryonic stem cell-derived beta cells

Research Objective The expected outcome of these studies is a cellular therapeutic for Type I Diabetes: engineered human islets for transplant into patients, surpassing the function of beta cells or progenitors alone. Impact The proposed studies would address key bottlenecks in cell replacement therapy for Type I Diabetes — issues with cellular engraftment, survival, and […]

Preclinical development of human hepatocyte progenitor cells for cell therapy

Research Objective Determine if human hepatocyte progenitor cells, which exist in the normal adult liver, can be maintained and expanded in vitro while maintaining in vivo regenerative capacity. Impact Cell transplantation therapy can be an effective alternative treatment for severe liver diseases to liver transplantation, which is severely limited by the lack of available donor […]

Thin Film Encapsulation Devices for Human Stem Cell derived Insulin Producing Cells

Research Objective We propose to develop a macroencapsulation technology, based on flexible nanoporous thin films, to support the long term viability and function of human stem cell derived insulin producing cells. Impact Encapsulation devices that maintain function of stem cell derived islets can address challenges with current cell therapy for Type I Diabetics, including islet […]

Developing a personalized approach to beta cell replacement for patients with a genetic form of diabetes

Research Objective To correct a gene mutation in a patient's stem cells and produce functional replacement cells for the patient to cure their diabetes. Impact WE expect that this project can serve as a model for developing new treatments for patients with certain forms of genetic diabetes. Major Proposed Activities To understand how the patient's […]

Generation of bile duct-competent transplantable human liver organoids

Research Objective Generation of human stem cell-derived mini livers capable of exporting bile into the gallbladder after transplantation into the liver Impact Mini livers capable of normal bile export would have potential for therapy of diseases in which bile export is impaired like Alagille syndrome Major Proposed Activities Generation of mini livers using human stem […]

Enhanced Autologous Pancreatic Islet Transplantation and Survival for Diabetes Mellitus Therapy

Translational Candidate 'Pseudoislets' derive from human islets, but compared to islets have superior survival, function, and diabetes reversal after transplantation. Area of Impact Pseudoislets could transform islet replacement strategies in diabetes by increasing the number and durable function of transplanted islet cells. Mechanism of Action Transplantation of replacement human islet cells is approved in type […]

MPS II: Plasma cell delivery of iduronate sulfatase

Translational Candidate The patient’s own B cells will be engineered to express the therapeutic enzyme needed for care in Mucopolysaccharidosis type II (MPS II) patients Area of Impact MPS II, a rare genetic disease causing multi-organ symptoms and death by age 15, if not treated. Current treatment does not address major symptoms. Mechanism of Action […]

Personalized Cell Therapy for Diabetes

Translational Candidate Preclinical studies will develop patient specific stem cell-derived islets that secrete insulin & other islet hormones for regulation of blood sugar Area of Impact Genetically matched stem cell derived islets could provide treatment for diabetes without the need for immunosuppression or implantable devices. Mechanism of Action The stem cell-derived islets contain insulin-producing pancreatic […]