(@jinsongzhang8012)

Goal 2: Reduce Human Disease

Develop and apply single-cell-based assays to verify/modify existing biological theories

Many biological models developed to date are based on studies using heterogeneous cell populations. For example, ChIP-Seq studies have shown that RNA Pol II is frequently paused near promotes and that in stem cells, developmentally poised genes are controlled by bivalent enhancers. These studies are largely based on statistical analyses of average ChIP-Seq reads from millions of cells. Earlier biochemical analysis of... more »

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0 net votes
2 up votes
2 down votes
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(@nhlbiforumadministrator1)

Goal 3: Advance Translational Research

Arrhythmia Therapies Based on Understanding Mechanisms

There is a need to translate these new insights of genetic, molecular, cellular, and tissue arrhythmia mechanisms into the development of novel, safe, and new therapeutic interventions for the treatment and prevention of cardiac arrhythmias.

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51 net votes
86 up votes
35 down votes
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(@akuaban)

Goal 2: Reduce Human Disease

Predictors of Sickle Cell Disease Severity

Can better predictors of disease severity such as specific biomarkers and/or genetic polymorphisms be identified so as to help understand the course and progression of sickle cell disease in various patients?

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58 net votes
76 up votes
18 down votes
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(@sophpacz)

Goal 3: Advance Translational Research

Maximizing anti-tumor immunity following allogeneic HCT with biomarkers

Allogeneic hematopoietic cell transplantation (allo-HCT) is one of the most effective forms of tumor immunotherapy available to date. Allo-HCT can be life-saving for patients with aggressive malignancies that cannot be cured through other strategies. The immunotherapeutic efficacy of allo-HCT depends on donor T cell recognition of alloantigens on leukemic cells, which is known as the graft-versus-tumor effect (GVT). No... more »

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32 net votes
52 up votes
20 down votes
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(@coretta.jenerette)

Goal 4: Develop Workforce and Resources

Sickle cell education for healthcare providers

Although sickle cell was first described more than 100 years ago and more than 100,000 individuals in the US are living with sickle cell disease, healthcare providers still lack basic knowledge of the key components in providing care for individuals with sickle cell. This often leads to poor health outcomes including stigmatization of patients with sickle cell seeking care. Evidenced-based curriculum should be available... more »

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18 net votes
21 up votes
3 down votes
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(@xuejunparsons)

Goal 3: Advance Translational Research

Deriving Cardiac Elements from Pluripotent Human embryonic Stem Cells for Heart Reconstitution

to date, the existing markets lack a clinically-suitable human cardiomyocyte source with adequate myocardium regenerative potential, which has been the major setback in developing safe and effective cell-based therapies for regenerating the damaged human heart in cardiovascular disease.

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-33 net votes
10 up votes
43 down votes
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(@xuejunparsons)

Goal 3: Advance Translational Research

Embedding the future of regenerative medicine into the open epigenomic landscape of pluripotent human embryonic stem cells

Large-scale profiling of developmental regulators and histone modifications by genome-wide approaches have provided powerful genome-wide, high-throughput, and high resolution techniques that lead to great advances in our understanding of the global phenomena of human developmental processes. However, without a practical strategy to convert pluripotent cells direct into a specific lineage, previous studies are limited... more »

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-24 net votes
9 up votes
33 down votes
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(@tosinola)

Goal 2: Reduce Human Disease

Impact of each VOC Crisis in patients with sickle cell disease

While the long term cumulative effects of frequency, duration and severity of VOC on mortality is known in SCD, there is little known about the impact of each individual crisis or the amount of damage during crisis versus background smoldering ischemia from the disease. Any effort in quantifying this for SCD in the absence of interventional agents initially, and then as a potential measurement of the benefit of drug... more »

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3 net votes
3 up votes
0 down votes
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(@nhlbiforumadministrator)

Goal 3: Advance Translational Research

Next generation of cellular blood products for clinical use

What advancements in our understanding of hematopoietic stem cell differentiation can be combined with practical developments in novel processing and storage technologies to develop the next generation of transfused blood therapeutics? There is a dearth of tools to evaluate the efficacy of transfused blood products, although this is the most commonly employed therapeutic procedure in the US. There is also no financial... more »

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-3 net votes
24 up votes
27 down votes
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