Therapeutic/Technology: Biologic
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 […]
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 […]
CRISPR/Cas9 nanoparticle enabled therapy for Duchenne Muscular Dystrophy in muscle stem cells
Research Objective Gene correction of muscle stem cells Impact These studies will develop a gene editing based therapy for one of the most prevalent lethal childhood disorders called Duchenne Muscular Dystrophy. Major Proposed Activities To identify the best MSNP-CRISPR candidates for CRISPR/Cas9 plasmid delivery in vitro to muscle stem cells To identify the best MSNP-STEM […]
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. […]
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 […]
Targeting progenitors in scar tissue to reduce chronic scar burden
Research Objective Develop novel strategies to treat heart scars by targeting progenitors that replenish scars Impact There currently is no therapy for treating scar tissue in the heart or any other organ. Our proposal would lead to the development of targeted approaches to reduce scar burden. Major Proposed Activities Identify progenitors in scar tissue (murine […]
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 […]
Activation of patient-specific endogenous myocardial repair through the exosomes generated from the hypoxic iPSC-derived cardiomyocytes (iCMs).
Research Objective This proposal will provide direct evidence of clinical implementation of patient-specific iPSC products by validating the efficacy of autologous, cell-free exosome therapy. Impact Five-year survival of heart failure is a dismal 50% and is top diagnosis of hospital admission. Exosomes offer a feasible and effective cell-free therapy by activating endogenous myocardial repair. Major […]
Noncoding RNA drug TY1 as a therapeutic candidate for scleroderma and systemic sclerosis
Translational Candidate Modified synthetic noncoding RNA molecule Area of Impact Systemic Sclerosis Mechanism of Action The mechanism of action of TY1 is alleviating cell stress and damage through enhancing genes that alleviate cell stress which, in turn, control inflammation and fibrosis in diseases tissue. Unmet Medical Need Systemic sclerosis is an incurable disease with no […]
Pro-regenerative infusible ECM biomaterial for treating acute myocardial infarction
Translational Candidate Injectable biomaterial derived from the natural scaffolding of pig hearts Area of Impact Improving the quality of life of patients with heart attacks Mechanism of Action The proposed mechanism of action is through recruitment of the body's own stem cells and reducing inflammation to heal the heart. Unmet Medical Need The prevalence of […]