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RAFT: A CRISPR-Free RNA Trans-Splicing Platform for MYH7 Correction in Hypertrophic Cardiomyopathy
Unmet Need Hypertrophic cardiomyopathy (HCM) is a common inherited heart disease affecting 1 in 500 individuals, frequently caused by mutations in the MYH7 gene. Current gene therapy approaches, including AAV-based gene replacement, face significant challenges…
Muscle-targeted gene therapy for SLC25A4 mitochondrial deficiency
Unmet Need There is a significant unmet need for disease-modifying therapies that address mitochondrial dysfunction in inherited cardiomyopathies and myopathies such as SLC25A4 deficiency. Existing treatments are limited to supportive care and heart transplantation, with…
Permanent blood pressure control through liver-directed gene editing
Unmet Need The World Health Organization estimates that high blood pressure (hypertension) results in approximately 9 million annual deaths worldwide. While current medications can lower blood pressure, they are limited by the need for lifelong…
Novel TRPV4-Targeted Therapy for Arrhythmic Cardiomyopathy
Unmet Need Arrhythmic cardiomyopathy (ACM) is a life-threatening condition characterized by fatty-fibrotic remodeling of the heart muscle, leading to arrhythmias, heart failure, and sudden cardiac death. While 60% of ACM cases are linked to known…
Precision Engineering of Neural Circuits via Designer Connexins for Long-Term Cellular Integration
Unmet Need Gap junctions are crucial for enabling the direct flow of ions and small molecules between cells, playing a significant role in synchronizing electrical activity in organs like the heart and brain. However, mutations…
A simple blood test that can predict heart failure earlier than current methods
Unmet Need Heart failure is the leading cause of morbidity and mortality in the developed world. Approximately 50% of the US population over the age of 45 is expected to have preclinical heart failure, and…
Targeted delivery of gene therapy via exosomes for cardiomyocyte reprogramming
Unmet Need Every year, close to 1 million Americans experience a heart attack. A heart attack results in the loss of specialized cells called cardiomyocytes, which are crucial for the heart’s contraction and rhythmic beating.…
Dark-Blood Delayed-Enhancement MRI: Improved Tissue Contrast and Blood-Pool Signal Suppression
Unmet Need Delayed-enhancement MRI (DE-MRI) is the standard for imaging myocardial infarction (MI), providing high-resolution maps of infarcted and viable myocardium. However, DE-MRI often suffers from poor differentiation between infarcted myocardium and the bright left…
ClinVar: Determining the factors influencing cardiomyopathy-associated gene variant call evolution
Description ClinVar is a freely accessible, public archive of reports of human variations classified for diseases and drug responses, with supporting evidence. ClinVar thus facilitates access to and communication about the relationships asserted between human…
Central Venous Catheter Insertion Device
Unmet Need Central Venous Catheterization (CVC) is a common procedure where a catheter is inserted into a large vein, typically in the neck, chest, or groin. With 5 million insertions a year, representing 8% of…
Metabolite biomarkers of human heart failure
Value Proposition Heart failure is a very common condition affecting approximately 5.1 million people in the United States. Due to its association with age, the incidence of heart failure, as well as the cost of…
Novel biomarkers to detect Venous Thrombosis without radiation exposure in elderly patients
Unmet Need Venous thrombosis/thromboembolism (VT/E), the formation of a blood clot in vein, can be a life-threatening condition with 600,000 incidences in the US per year and affecting an estimated 10 million people globally. VT/E…