Goal 3: Advance Translational Research

Submitted by (@lungmatbio1)

Next Stage of COPD Discovery

1) Refinement of COPD subphenotypes for therapeutics, diagnostics and mechanistic interrogation. The NIH should encourage a strong focus on a) rigorous, mechanistically-reinforced definitions (chronic bronchitis, emphysema (with and without obstruction), frequent exacerbators, combined pulmonary fibrosis and emphysema) and 2) the development and optimization of animal model systems that replicate the different subphenotypes. ...more »

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Goal 4: Develop Workforce and Resources

Submitted by (@mmongrawchaffin)

Enhance funding for population and public health research

Many challenges posted here focus on increasing translation and development of interdisciplinary teams. Diverse backgrounds and epidemiological training makes population and public health scientists ideal candidates to connect teams in different areas of research around translation to human health. While funding exists specifically for career development of physician and pre-clinical scientists, none exists for epidemiologists. ...more »

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29 up votes
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Goal 2: Reduce Human Disease

Submitted by (@tcooper)

The relationship between genetic variation and disease mechanisms

What is the contribution of individual differences in RNA processing to disease causation, disease modification, disease susceptibility, and positive or negative responses to therapies? Studies using genome sequencing combined with RNA-seq have determined that genetic variation affects regulation of RNA processing as frequently as transcriptional regulation. While transcriptional networks are well defined in heart development ...more »

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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 (@klaus0)

Integrate mechanistic and translational research

Other entries have asked for support for translational research. To be successful, this must be integrated with mechanistic research. Ideally. MD-PhD teams would get together. Challenges in translation: find formulation, preclinical toxicology, phase I trials to establish safety, biomarkers. Challenges in mechanism: RNA-Seq, Chip-Seq of relevant cells, KO mice by Cas-CRISPR, deep expertise in the relevant discipline.

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Goal 3: Advance Translational Research

Submitted by (@hubel001)

Advancing the preservation of cellular therapies

Cell therapies are produced in specialized facilities and the viability/function of the cells must be retained in order to permit transportation to the site of use, coordination with patient care, etc. Current options for preserving cells are limited. Conventional methods of cryopreservation may result in poor post thaw function and are difficult to use at the point of care. Liquid storage of cells is typically limited ...more »

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Goal 1: Promote Human Health

Submitted by (@nhlbiforumadministrator1)

Transforming Transplantation with RISC

What is necessary to reprogram the immune system to improve transplant outcomes of hearts, lungs, and hematopoietic cells? While NIAID is a major funder of immunology research, we are a major contributor to stem cell research. Our resources could be combined, where NIAID would support this approach for autoimmune diseases, and we would support work in tolerance for transplants. If the NCI also wants to collaborate on ...more »

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