Project Objective: Research Insights
Enhanced Branching Morphogenesis and Pluripotent Cell Lineage Differentiation for Pediatric Regenerative Therapies
Research Objective Approximately 20,000 babies are born annually with kidney disease; the long-term outcome is poor. These studies address new ways to develop mini-kidney structures for transplantation to induce repair. Impact ~85% of people on the organ waitlist are in need of a kidney and there are insufficient donors. There is a pressing need to […]
Optimizing self-renewal signaling kinetics to stabilize ex vivo hematopoietic stem cell expansion
Research Objective We aim to develop conditions for stable expansion of blood stem cells outside of the body Impact Blood stem cells are a rare but necessary cell type for curative bone marrow transplantation and related gene therapies. Stable blood stem cell expansion will increase therapy availability and success Major Proposed Activities Validate a fully […]
Identification of antigenic neo-epitopes from in vitro reprogrammed human tissue precursors for regenerative therapy
Research Objective This study examine potential immunologic changes caused by cellular reprogramming that could present a barrier to clinical application of regenerative therapies. Impact Identification and evaluation of immunologic changes caused by cellular reprogramming provides critical information to maximize the efficacy and safety of regenerative cellualar therapies. Major Proposed Activities Identify changes to the repertoire […]
Generation of human airway stem cells by direct transcriptional reprogramming for disease modeling and regeneration
Research Objective We will generate human airway stem cells by direct transcriptional reprogramming of fibroblasts. We will use these induced airway stem cells to model motile cilia disease in a dish. Impact Generating airway stem cells through reprogramming will create a scalable and editable cell line from which we can derive airway epithelium, thus enabling […]
An exosome-based translational strategy to mitigate Alzheimer’s disease neuropathology
Research Objective These studies will determine whether stem cell derived exosomes (nano-scale vesicles) can be used to treat the symptoms of Alzheimer’s disease (AD). Impact Our stem cell-derived exosome therapy will provide a viable approach to ameliorate the relentless progression of AD that severely impacts quality of life for millions of patients and their families. […]
Reprogramming human stem cells for blood cell generation
Research Objective To create a universal donor blood cell line that can be used to produce human red blood cells for transplantation. Impact Successful completion of this work would create a safe, unrestricted source of universal donor human blood cells that could be used to improve healthcare and save lives throughout the world. Major Proposed […]
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 […]
Generation of expandable, self-renewing muscle stem cells for Duchenne Muscular Dystrophy
Research Objective The goal of this proposal is to define protocols to generate expandable, self-renewing human muscle stem cells (MuSC) from hiPS cells for Duchenne Muscular Dystrophy disease modeling and therapeutics. Impact The integration of STAT3i with current approaches to derive myogenic cells from hiPS cells would enable the generation of self-renewing MuSC that are […]
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 […]
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 […]