Goal 2: Reduce Human Disease

Submitted by (@nhlbiforumadministrator1)

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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26 down votes
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Goal 2: Reduce Human Disease

Submitted by (@freddigoldman)

How can we more safely deliver stem cells to Sickle Cell patients

Newer therapies using gene correction, rather than gene addition, are needed for sickle cell disease. Even with this potential advantage, there needs to be a way to safely deliver gene corrected HSC to the sickle cell patient. Chemotherapy is poorly tolerated, and often is the reason patients do not choose the BMT option. What is the status of other less toxic non myeloablative approaches, and how can they best be ...more »

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51 net votes
67 up votes
16 down votes
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Goal 3: Advance Translational Research

Submitted by (@nhlbiforumadministrator)

Expediting Gene and Cell Therapies to the Clinics

What methodologies will best enhance the translation of technologies for gene and cell therapies into potential products for clinical application and commercial development? In considering a strategy for NHLBI investment in gene therapy, it is important to note that we are only at the beginning of a revolution that will eventually impact biomedical research across a broad range of specialties. NHLBI/NIH needs to create ...more »

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66 net votes
97 up votes
31 down votes
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Goal 1: Promote Human Health

Submitted by (@nhlbiforumadministrator)

Rejuvenating Old Hearts and Lungs

Is there a true "mechanome" affecting normal heart function that could be studied to uncover important mechanically-stimulated pathways relevant to tissue development, maintenance, and repair in vivo? Are there mechanomics that predict and control fundamental biological processes linked to the body's natural healing and regeneration machinery? What if we could leverage 3D tissue engineering advances to rejuvenate aging ...more »

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27 net votes
43 up votes
16 down votes
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