Goal 3: Advance Translational Research

Current State of Regenerative Medicine: Moving Stem Cell Research from Animals into Humans for Clinical Trials

Realizing the developmental and therapeutic potential of pluripotent human embryonic stem cell (hESC) derivatives has been hindered by the inefficiency and instability of generating clinically-relevant functional cells from pluripotent cells through conventional uncontrollable and incomplete multi-lineage differentiation.

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Goal 3: Advance Translational Research

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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Goal 1: Promote Human Health

What is the role of the environment in HLB disease etiology

It is clear that genetic sequence variation does not account for a substantial portion of disease burden. It is likely that the broad environment contributes to HLB disease via epigenomic alterations and interaction with genetic variants. There is an urgent need to understand the environmental contribution to disease as most exposures are modifiable and are targets for prevention interventions.

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Goal 3: Advance Translational Research

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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Goal 2: Reduce Human Disease

10. What biological variables are most influential in the development and clinical outcomes of heart disease and what can be don

Given that approximately 64 percent of women who died suddenly of CHD had no previous symptoms4 and that traditional risk factors and scores underestimate CHD risk in women, there is a need to identify unique markers for women at risk for CHD60.

 

( from 10 Report)

Submitted by (@scampbell)

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Goal 1: Promote Human Health

The CRISPER-Cas challenge: Need to re-phenotype KO animals?

Because traditional knock out models and CRISP/Cas models often show different phenotypes for the same gene deletion, do we need to re-phenotype hundreds/thousands of knock out animal models and revisit the conclusions of many studies based on using these animal models? This research may not appear very innovative but may be very important for drawing correct conclusions about gene functions and interactions - should ...more »

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Goal 3: Advance Translational Research

Point of care detection of rare cells in blood

Laboratory analyses at the bedside or in the hinterlands can reduce the cost and increase the capture of disease states in underserved populations. The injection of a blood draw directly into a portable device that requires no further operator interventions is a Critical Challenge. With today’s automated chemistry and a miniaturized flow cytometer this challenge could be met.

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Goal 2: Reduce Human Disease

Prevention of rheumatic heart disease

Global health system outcomes studies are needed to identify impediments to successful primary and secondary prevention of rheumatic heart disease.

Other approaches, such as development of a vaccine for group A streptococcus and delaying disease progression once valve damage is present, could also be explored.

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