CORDIS Project
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This project explores the use of antisense oligonucleotides to correct genetic mutations causing familial hypertrophic cardiomyopathy. It focuses on improving the delivery of these therapeutic agents to heart cells derived from human stem cells.
Familial hypertrophic cardiomyopathy (HCM) is the most common inherited heart disease and is predominately caused by mutations in genes that encode proteins associated with cardiac sarcomeres, the functional unit critical for basic contractile heart function.
Nearly two-thirds of individuals with HCM experience mild to no symptoms, whereas the remaining one-third experience dyspnea, chest pain, or arrythmias that can progress into heart failure, causing sudden cardiac death, particularly in youn…
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