Research Objective We aim to solve a 30 year old problem, which is to understand using human stem cells how certain genetic mutations cause human disease. Impact We currently don't…
Research Objective We are building a foundational platform for developing genome editing technologies that increase patient coverage by 20-40X and can be applied to over 700 genetic diseases in diverse…
Research Objective We will build a foundational model and experimental platform to catalog all genes that promote and protect against PAH, with the potential to extend to many other developmental…
Research Objective Greenstone Biosciences will use patient-derived stem cells to discover safe and effective drugs for cardiovascular disease. Impact The use of patient-derived stem cells to discover novel drug targets…
Research Objective To discover CRISPR-based genetic strategies that will directly and therapeutically modify cardiac myosin heavy chain isoform expression to treat HF and cardiomyopathies. Impact Heart Failure and Cardiomyopathies Major…
Research Objective Use human cell therapy insights, specifically CDC-secreted EV analysis, to develop a noncoding RNA chemical entity for myocarditis treatment. Impact Key knowledge gap is how to recruit adaptive…
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…
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…
Research Objective The candidate is a gene therapy that delivers cardiac reprogramming factors to convert resident cardiac fibroblasts into functioning cardiac muscle. Thus, it is a regenerative cardiac gene therapy.…
Research Objective Modified synthetic noncoding RNA molecule Impact Heart failure with preserved ejection fraction Major Proposed Activities Lead optimizationPerform extensive preclinical testing and select a therapeutic candidate.Develop and test preliminary…
