Goal 3: Advance Translational Research

Genome Editing and Gene Therapy

There is a critical need for the establishment of strategies that will determine the efficacy, safety, and toxicity of genome editing techniques specifically in hematologic diseases.

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69 net votes
87 up votes
18 down votes
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(@klaus0)

Goal 3: Advance Translational Research

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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28 net votes
37 up votes
9 down votes
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(@nhlbiforumadministrator1)

Goal 4: Develop Workforce and Resources

Need to train and nurture more "translators"!

One of the major challenges in translating from bench-to-bedside and back is communication: the ability of basic and clinical scientists to understand each other's scientific language to be able to appreciate the importance of the other’s research questions and findings.

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22 net votes
39 up votes
17 down votes
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(@tcooper)

Goal 2: Reduce Human Disease

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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19 net votes
24 up votes
5 down votes
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(@mmongrawchaffin)

Goal 4: Develop Workforce and Resources

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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16 net votes
29 up votes
13 down votes
Active
(@lungmatbio1)

Goal 3: Advance Translational Research

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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16 net votes
26 up votes
10 down votes
Active
(@nhlbiforumadministrator)

Goal 1: Promote Human Health

Transforming Transplantation with Reprogramming Immune System Cells (RISC)

Can we "reprogram" the immune system to improve outcomes of heart, lung, and hematopoietic cell transplants? 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 co-stimulatory... more »

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15 net votes
27 up votes
12 down votes
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(@nhlbiforumadministrator)

Goal 3: Advance Translational Research

Implementation of T4 Translation Research Platforms and Networks

How can cost-effective implementation of late stage translation (T4) research protocols be facilitated for heart, lung, blood, sleep diseases and health inequities?

Can research platforms and networks be created and utilized to facilitate execution of multi-level interventions and approaches for the end user in collaboration with key stake holders?

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4 net votes
13 up votes
9 down votes
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(@nhlbiforumadministrator)

Goal 3: Advance Translational Research

T4 Translation Research Informing Early Stage Translational Research

There is a need to utilize insights gained from T4 translation research and implementation science to inform the design and execution of early-stage translational research and clinical trials.

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3 net votes
13 up votes
10 down votes
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(@nhlbiforumadministrator1)

Goal 3: Advance Translational Research

Translation Research Dissemination & Implementation Frameworks

We need to identify and test the proven effective dissemination and implementation frameworks that are relevant to heart, lung, and blood disorders in order to scale up evidence-based interventions in real world settings, ultimately improving health equity among minority populations, including low income minority residents living in public housing.

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4 net votes
13 up votes
9 down votes
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(@nhlbiforumadministrator)

Goal 3: Advance Translational Research

Leveraging PEPFAR infrastructure for CVDs

How do we go about leveraging existing infrastructure, such as PEPFAR, to reduce the risk of HLBS diseases among HIV patients and other vulnerable populations? • Common goals and deliverables between NHLBI and partners will need to be identified • The best return on investment of NHLBI funds will need to be determined • Feasible T4 translation interventions in PEPFAR funded studies utilizing HIV populations with HLBS... more »

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-1 net votes
7 up votes
8 down votes
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(@nhlbiforumadministrator1)

Goal 3: Advance Translational Research

Developing Methods and Metrics for T4 Outcomes and Impact

How can methods and metrics capable of conducting high quality T4 research be developed to accurately capture outcomes and the overall impact new T4 knowledge has on population health for heart, lung, blood, sleep diseases and disorders?

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-1 net votes
7 up votes
8 down votes
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