Project Objective: Proof of Concept
Pluripotent Stem Cells for Tendon Tissue Engineering
Research Objective We propose to develop a bio-tendon engineered from differentiated pluripotent stem cells for the repair of tendon injuries and degeneration. Impact Rotator cuff tears are the most common causes of shoulder pain that require surgery. However, failure rates range from 20% to 90%. A successful tendon repair will have a major impact on […]
Microgel encapsulated iPSC-derived notochordal cells to treat intervertebral disc degeneration and low back pain
Research Objective We aim to discover an injectable, rejuvenating treatment for painful intervertebral disc degeneration using microtissue-encapsulated iPSC-derived notochordal cells (iNCs) using large animal model Impact Our treatment candidate may allow for a non-invasive stem cell therapy, targeting the underlying pathogenesis of intervertebral disc degeneration, the leading cause of chronic back pain in adults. Major […]
A Novel Therapy for Sanfilippo B
Research Objective To develop a Stem Cell therapy for Sanfilippo B syndrome. Impact There is no treatment for Sanfilippo syndrome, and other therapeutic approaches have failed in clinics. This proposal will develop a stem cell based therapy for Sanfilippo syndrome. Major Proposed Activities Generation of universal donor Embryonic Stem Cells (ESC H1) using state of […]
Novel Lipid Nanoparticles for Enhancing eNOS Synthesis for Cardioprotection Post Myocardial Infarction
Research Objective Our therapeutic candidate is a lipid nanoparticle that delivers a therapeutic dose of mRNA to the human heart, which transiently transfects of cells within the heart to improve function after an MI. Impact There is evidence for eNOS therapy as a cardioprotectant post MI; however, the progression of to the clinic has stalled […]
Autologous stem cell-derived interneuron cell therapy for spinal cord injury (SCI)
Research Objective Functional restoration following spinal cord injury using defined excitatory and inhibitory spinal interneuron progenitor cell transplantation Impact The development of a stem cell derived progenitor cell therapy with disease modifying potential for spinal cord injury Major Proposed Activities Define and characterize DI4 GABA and V3 Glut spinal interneuron progenitor cells derived from pluripotent […]
A treatment for Rett syndrome using glial-restricted neural progenitor cells
Research Objective We developed a novel glial-restricted neural progenitor cells transplantation strategy as a treatment for Rett syndrome, reverting neuronal alterations caused by genetic mutations. Impact There are no disease-modifying therapies for Rett syndrome. Our therapeutic, if successful, will be a first-in-class treatment for this devastating neurological disorder and potentially others. Major Proposed Activities Characterization […]
Modified RNA-Based Gene Therapy for Cardiac Regeneration Through Cardiomyocyte Proliferation
Research Objective Efficacious and safe intramyocardial delivery of modified mRNA encoding cell cycle regulators as a gene therapy for cardiac regeneration through resident cardiomyocyte proliferation. Impact This project would provide disease-modifying gene therapy for people with heart failure due to loss of cardiac muscle, a leading cause of deaths in the US, using novel modified […]
An hematopoietic stem-cell-based approach to treat HIV employing CAR T cells and anti-HIV broadly neutralizing antibodies.
Research Objective We propose to transduce hematopoietic stem cells with vectors that encode chimeric antigen receptors targeting HIV for T cells and anti-HIV broadly neutralizing antibodies for B and/or plasma cells. Impact Recent methods are limited by the rise of escape mutants against a single CAR. Our approach solves this issue by the ability to […]
Bioengineering human stem cell-derived beta cell organoids to monitor cell health in real time and improve therapeutic outcomes in patients
Research Objective We will generate nanoprobe-containing stem cell-derived human beta cells that can be monitored in real time in response to inflammatory stress upon transplantation in patients with type 1 diabetes. Impact Our product will replace donor islets for cell replacement therapy in patients with type 1 diabetes, and will provide a readout of cell […]
Enabling non-genetic activity-driven maturation of iPSC-derived neurons
Research Objective We will empower stem cell biologists to generate iPSC-derived neurons faster and with enhanced maturation by enabling optical cell stimulation and triggering activity-dependent maturation processes Impact Our project will address such critical bottlenecks as insufficient maturity of iPSC-derived neurons that limits their utility in age-related neurological disorders that manifest later in life. Major […]