Project Objective: Proof of Concept
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 […]
CRISPR/dCas9 mutant targeting SNCA promoter for downregulation of alpha-synuclein expression as a novel therapeutic approach for Parkinson’s disease
Research Objective Discovery of a novel therapeutic candidate for Parkinson’s disease which modifies gene expression using human stem cell-derived neurons to halt the neurodegenerative disease process. Impact Stopping the neurodegenerative process of Parkinson’s disease is a critical unmet medical need. Our approach is based on novel gene engineering technology that modifies expression of key target […]
Direct Cardiac Reprogramming for Regenerative Medicine
Research Objective To develop a gene therapy product to deliver cardiac reprogramming factors into the heart for regeneration of new heart muscle. Impact The proposed candidate would regenerate heart muscle for the 23 million adult and pediatric patients with heart failure, for whom there are currently no disease-modifying therapeutic approaches. Major Proposed Activities Successful conversion […]
hNSC-mediated delivery of ApiCCT1 as a candidate therapeutic for Huntington’s disease
Research Objective The therapeutic candidate is a human Neural Stem Cell that secretes a protein, ApiCCT1, that aids in the prevention of disease phenotypes, for application in treatment of Huntington's disease (HD). Impact No treatment currently exists that can slow or prevent the unrelenting progression of Huntington’s disease, a devastating brain disease, therefore a completely […]
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 […]
Immunotherapy for HIV infection using engineered hematopoietic stem/progenitor cells
Research Objective The therapeutic candidate proposed here is hematopoietic stem/progenitor cells engineered to encode for HIV-specific T cell receptors. Impact The success of the proposed studies will test the efficacy of an approach to provide long-lasting functional cure for HIV infection, obviating the need for anti-retroviral therapy. Major Proposed Activities Test if engineered hematopoietic stem/progenitor […]
MSC delivery of an artificial transcription factor to the brain as a treatment for Angelman Syndrome
Research Objective Mesenchymal stem cells will be used to deliver an artificial transcription factor to neurons in the brain to treat a genetic disease. Impact It could lead directly to a treatment for Angelman Syndrome, but the approach could be used to alter gene expression in almost any brain disorder. It could overcome the brain […]
Scalable, Defined Production of Oligodendrocyte Precursor Cells to Treat Neural Disease and Injury
Research Objective The goal of this proposal is to develop an optimized, scalable process to manufacture high quality oligodendrocyte precursor cells (OPCs) from human pluripotent stem cells for treating human disease. Impact OPCs have therapeutic potential for spinal cord injury, restoration of cognitive function after cancer radiation therapy, inherited demyelinating disease, and potentially multiple sclerosis. […]
Novel Rejuvenated T Cell Immunotherapy for Lung Cancer
Research Objective Through this project, we would like to evaluate how this T-iPSC-based immunotherapy that we have developed can eliminate lung cancer cells effectively in vivo using xenografted SCID mice. Impact This novel T-iPSC-based immunotherapy will provide another effective treatment for lung cancer and possible other malignancies by supplying unlimited number of young and active […]
Gingival mesenchymal stem cells as a novel treatment modality for periodontal tissue regeneration
Research Objective To develop a novel regenerative and adhesive hydrogel encapsulating patient's' gingival stem cell which can potentially be used as an adhesive dental hydrogel for periodontal tissue regeneration. Impact Upon successful completion, this project will introduce a promising treatment approach for maxillofacial defects presenting an innovative treatment modality for periodontal tissue regeneration. Major Proposed […]