(@nhlbiforumadministrator)

Goal 1: Promote Human Health

Re-examining Antigenicity of Rare Blood Disease Proteins

What specific roles do protein glycosylation and protein complex formation on stable and activated cellular membranes play in inherent immunogenicity and antibody formation to life-saving therapies for rare blood disorders?

 

NHLBI leadership and collaboration with NIAID is needed to bring this combined expertise to bear on the understanding and prevention of this most significant complication of hemophilia therapy.

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-1 net votes
8 up votes
9 down votes
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(@nhlbiforumadministrator)

Goal 2: Reduce Human Disease

Detection of rupture prone small aortic aneurysms

Critical challenges in the assessment of aortic aneurysms are: (1) Availability of reliable animal models that simulate the human pathology, (2) Availability of molecular imaging resources – identification of biomarkers, development of targeted imaging probes and pre-clinical imaging methods, and plasma markers that predict whether an aneurysm is prone to rupture or dissection, (3) Bringing together a wide array of multi-disciplinary ...more »

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7 net votes
18 up votes
11 down votes
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(@nhlbiforumadministrator)

Goal 3: Advance Translational Research

Bone Marrow Stem Cell Transplant in Peds sibling matched SCD

There is a need to improve accessibility of Bone Marrow Stem Cell Transplantation (BMSCT) for Sickle Cell Disease patients who are most likely to benefit from this treatment option. 1. Building a culture of trust between and among primary care providers, specialists, patients/families, and other stakeholders 2. Consensus building around BMSCT as an acceptable treatment alternative (as opposed to another research endeavor) ...more »

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52 net votes
80 up votes
28 down votes
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(@jamesr.priestmd)

Goal 2: Reduce Human Disease

Understanding the Genetic & Epigenetic Basis of Congenital Heart Disease?

Over the last thirty years, our fundamental understanding of the genetics and pathogenesis of congenital heart disease has lagged the tremendous advances in the surgical and clinical care of infants with this group of disorders. We need to close this gap with investigation into the genetic basis of congenital heart malformations to develop new models of disease. The goall is translate an improved molecular genetic and ...more »

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22 net votes
37 up votes
15 down votes
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(@nhlbiforumadministrator)

Goal 3: Advance Translational Research

Implementation science research to reduce adverse effects of SCD

From various publications and reports, we have characterized the risks associated with sickle cell disease (SCD) and understand many of the barriers for treatment of SCD in LMICs. How can implementation science research be used to reduce the negative outcomes of SCD in low/middle income countries?

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18 net votes
31 up votes
13 down votes
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