Goal 2: Reduce Human Disease

Gene Therapies to Revitalize/Regenerate Cardiac Function

There is a need to examine the use of recombinant DNA to the heart for correction of genetic abnormalities or restoration of normal signaling pathways to prevent heart failure. However, gene therapy is a complex process and more studies are needed in which tissue targeting, route of delivery, regulation of target gene expression, therapeutic dose, and identification of robust biomarkers are further investigated.

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Is this idea a Compelling Question (CQ) or Critical Challenge (CC)? : Critical Challenge (CC)

Details on the impact of addressing this CQ or CC :

Successful gene therapy could restore or improve the condition of heart failure patients, especially when medications have been unsuccessful.

Feasibility and challenges of addressing this CQ or CC :

There have already been human trials of gene therapy in heart failure patients with positive outcomes.

Improvements in cardiac revascularization and medical therapies have significantly reduced cardiovascular-related deaths; however, the number of patients developing heart failure (HF) has steadily increased. One explanation is that surgery and medical therapies are palliative, but do not address the molecular pathogenesis of HF.

Name of idea submitter and other team members who worked on this idea : NHLBI Staff

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Idea No. 287