Goal 3: Advance Translational Research

To facilitate innovation and accelerate research translation, knowledge dissemination, and implementation science that enhances public health.

Goal 3: Advance Translational Research

Submitted by (@joyce.bischoff)

To find specific medical therapies to treat the wide array of human vascular malformations and vascular tumors.

Vascular malformations and vascular tumors, together referred to as vascular anomalies, comprise a complex and wide array of diseases in which there is a fundamental disruption in blood and lymphatic vasculature. The lesions disrupt organ function, destroy tissue, cause bleeding, increase infections and can threaten life. At present, there are some medical therapies but none are specifically targeted to an underlying ...more »

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Goal 3: Advance Translational Research

Submitted by (@nhlbiforumadministrator)

Maximizing Previous Investment in Existing Cohorts

Everyone would like to see integration of genomic, metabolomic, epigenomic, proteomic, transcriptomic, etc. data analyzed in the context of clinical disease, environmental influences, and even end-organ effects (lung versus heart or blood as an example). Rarely can this occur on small cohorts, but rarely are funds available to take maximum use of existing large cohorts and the samples and information collected within ...more »

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Goal 3: Advance Translational Research

Submitted by (@j.m.miano)

Integrating New Genomic Discoveries with Genome Editing Towards Personalized Medicine

The human genome is a veritable digital library of information that includes millions of regulatory elements and the expansive classes of long and short noncoding RNAs. These noncoding sequences represent a rich source of sequence variation (eg, SNPs), but the role such sequence variants play in the control of gene expression or noncoding RNA function is poorly understood. Many noncoding sequence variants will prove to ...more »

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6 net votes
16 up votes
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Goal 3: Advance Translational Research

Submitted by (@dkagr0)

Funding for synthesis and screening of potentially therapeutic molecules

Currently, there are limited, if none, funding resources to synthesize and screen potentially therapeutic molecules, based on supportive findings in cells, biopsy tissues from the patients with the disease in question, and the preliminary data to support the development of a series of compounds to screen them for their pharmacokinetics, pharmacodynamics, toxicity and use in clinically-relevant large animal models.

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Goal 3: Advance Translational Research

Submitted by (@rjjones)

Allogeneic transplantation as a safe and universally available therapeutic strategy for treating non-malignant blood diseases

Can new advances in allogeneic blood or marrow transplantation (BMT) make the procedure a safe and universally available therapeutic strategy for treating non-malignant blood and immune disorders such as sickle cell anemia, thalassemia, aplastic anemia, and severe combined immune deficiency?

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