Cell Line Generation: Embryonic Stem Cell
Therapeutic immune tolerant human islet-like organoids (HILOs) for Type 1 Diabetes
Research Objective Development of immune tolerant human islet-like organoids for transplantation into diabetic patients. Impact Our proposal will progress the development of an unlimited, reproducible source of immune tolerant engineered islets for transplantation into Type I diabetics. Major Proposed Activities Demonstrate efficacy of immune tolerant HILOs in humanized diabetic mice Demonstrate safety of immune tolerant […]
Identification and characterization of the optimal human neural stem cell line (hNSC) for the treatment of traumatic brain injury (TBI) 2.0.
Research Objective We propose to discover the optimal human neural stem cell candidate for traumatic brain injury. 4 hNSC products (2 ES derived & 2 fetal) will be compared with TBI/vehicle controls, & then each other. Impact Traumatic brain injury (TBI) affects more Americans than brain, breast, colon, lung and prostate cancer combined ! There […]
A Novel Tissue Engineering Technique to Repair Degenerated Retina
Research Objective Transplantation of human embryonic stem cell (hESC) derived retina organoids (hESC-RO) together with hESC derived retinal pigment epithelium (hESC-RPE) to treat advanced retinal degeneration diseases Impact Based on the ‘proof of concept’ experiments in animal models, this novel approach can be translated into a therapeutic product for the treatment of advanced human retinal […]
Morphological and functional integration of stem cell derived retina organoid sheets into degenerating retina models
Translational Candidate Retina organoid sheets (ROs) derived from CSC14 human embryonic stem cells (NIH registry line #0284) manufactured under GMP conditions Area of Impact Retinitis PIgmentosa (RP) (irreversible loss of photoreceptors) due to mutation of photoreceptors and/or other retinal genes Mechanism of Action Proposed mechanism of action is cell replacement, combined with trophic effects. Transplanted […]
Human Stem-Cell Based Development of a Potent Alzheimer’s Drug Candidate
Over 6 million people in the US suffer from Alzheimer’s disease (AD). There are no drugs that prevent the death of nerve cells in AD, nor has any drug been identified that can stimulate nerve cell replacement in aged human brain. Importantly, even if nerve cells could be replaced, the toxic environment of the AD […]
Clinical Development of a Cell Therapy for Diabetes
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 pancreatic cells are loaded into a delivery device, which is essentially a small envelope made with a semi-permeable membrane, not unlike a flat tea […]
Alpha Stem Cell Clinic for the Development of Regenerative Therapies
The proposed alpha clinic will bring together an outstanding team of physician-scientists with substantial clinical trials experience including stem cell and other cellular treatments of blood diseases and others. This team will also draw on our unique regional competitive advantages derived from our history of extensive collaboration with investigators at many nearby first-class research institutions […]
A suite of engineered human pluripotent stem cell lines to facilitate the generation of hematopoietic stem cells
Our goal is to develop tools that address major bottlenecks that have prevented the generation of blood forming stem cells in culture for therapeutic use. To help overcome these bottlenecks, we will generate a suite of human embryonic stem cell reporter lines that can be used to monitor key milestones in blood stem cell development. […]
Development of a Relevant Pre-Clinical Animal Model as a Tool to Evaluate Human Stem Cell-Derived Replacement Therapies for Motor Neuron Injuries and Degenerative Diseases
Motor neurons degenerate and die as a consequence of many conditions, including trauma to the spinal cord and its nerve roots and degenerative diseases such as amyotrophic lateral sclerosis and spinal muscular atrophy. Paralysis and in many cases death may result from a loss of motor neurons. No effective treatments are available for these patients. […]
Generation of a functional thymus to induce immune tolerance to stem cell derivatives
Stem cell research offers the promise of replacing missing or damaged tissues in the treatment of disease. Stem-cell-derived transplants still face problems with rejection as in traditional organ transplants. Several drugs can prevent rejection but also suppress the immune system, leaving patients vulnerable to infections and cancer. To avoid rejection without using drugs requires re-educating […]