Project Objective: Proof of Concept
iPS-Interneuron Transplantation for Neural Repair after Stroke
Research Objective To determine if transplantation of iPS-interneurons cells (iPS-3i cells) enhances functional recovery in stroke. Impact Successful completion of the proposed studies will develop a brain repair therapy for stroke, an unmet clinical need with significant impact on society. Major Proposed Activities To determine the recovery effect of transplantation of iPS-3i cells in the […]
Examining the efficacy of GDF11 antibody as a rejuvenator of aged human muscle stem cell capacity and muscle repair .
Research Objective To examine the efficacy of blocking blood borne GDF11 activity to rejuvenate aged human muscle stem cell regenerative capacity. Impact This project will provide a proof-of-principle that GDF11 inhibition can boost aged human skeletal muscle repair, and facilitate its translational potential. Major Proposed Activities Determine GDF11 protein levels in human sera as a […]
Exosomal Y-RNAs as mediators of bioactivity of cardiac-derived cell therapy
Research Objective We propose to dissect the contribution of Y-RNAs, small non-coding RNA species enriched in CDC-exosomes, in mediating the effect of CDC-exosomes on cardioprotection and macrophage polarization. Impact Examining the contribution of highly represented RNA species in CDC-exo could allow a better understanding of the mechanism of action of CDC-exo and modulation of their […]
Development of 3D Bioprinting Techniques using Human Embryonic Stem Cells Derived Cardiomyocytes for Cardiac Tissue Engineering
Heart, stroke and other cardiovascular diseases are responsible for ~17 million deaths per year globally and this number is predicted to reach 23.3 million by 2030. Cardiovascular diseases impose a staggering annual cost of $300 billion on the U.S. health care system. Heart transplantation is the ultimate solution to end-stage heart failure. However, a major […]
Injectable Macroporous Matrices to Enhance Stem Cell Engraftment and Survival
Despite the great promise stem cells hold for regenerative medicine, the efficacy of stem cell-based therapies is greatly limited by poor cell engraftment and survival. To overcome this major bottleneck, the goal of this proposal is to validate the efficacy of novel microribbon (µRB)-based scaffolds for cell delivery. These scaffolds combine the injectability and cell […]
A small molecule tool for reducing the malignant potential in reprogramming human iPSCs and ESCs
This research project aims to solve a key bottleneck in the use of differentiated human embryonic stem cells and induced pluripotent stem cells for the regeneration and replacement of diseased or damaged tissues. This bottleneck is the potential of unintended transplants containing failed-to-differentiate stem cells developing into benign growths called teratomas, or worse, malignant teratocarcinomas. […]
Stem Cell-Derived Astrocyte Precursor Transplants in Amyotrophic Lateral Sclerosis
Combination therapy to Enhance Antisense Mediated Exon Skipping for Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) affects 1 in every 3,500 boys worldwide. DMD is caused by mutations in the gene encoding dystrophin, a protein key to muscle health. DMD patients are typically weaker than normal by age 3, and with progressive muscle weakness most loose the ability to walk by age 11. DMD progresses to complete […]
Use of human iPSC-derived neurons from Huntington’s Disease patients to develop novel, disease-modifying small molecule structural corrector drug candidates targeting the unique, neurotoxic conformation of mutant huntingtin
The long-term objective of this project is to develop a drug to treat Huntington’s disease (HD), the most common inherited neurodegenerative disorder. Characterized by involuntary movements, personality changes and dementia, HD is a devastatingly progressive disease that results in death 10–20 years after disease onset and diagnosis. No therapy presently exists for HD; therefore, this […]
Gene therapy-corrected autologous hepatocyte-like cells from induced pluripotent stem cells for the treatment of pediatric single enzyme disorders
Liver transplantation (LT) has been used to treat a variety of liver diseases. Within hours after birth, neonates can present with disorders of the urea cycle (UCDs), the critical metabolic liver pathway needed to detoxify waste nitrogen from the diet and cellular turnover. The overall incidence of UCDs is estimated to be 1 in 8200 […]