Goal 3: Advance Translational Research

Lipid and lipoprotein metabolism in the CNS

The analysis of lipoprotein metabolism has traditionally been restricted to the easily accessible circulation and peripheral tissues. Very little work has been done behind the blood brain barrier, where many of the lipid carrying or metabolizing genes are also expressed. Yet we know very little about their functions there, although for instance ApoE4 is THE primary risk factor for late-onset Alzheimer's disease. Conventional ...more »

Submitted by (@joachim.herz)

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Goal 2: Reduce Human Disease

Mitigating risks due to the RBC storage lesion and vulnerable patients

What are the underlying dependencies (genomic, metabolic, disease) in individual donors that either accelerate or delay the changes to red blood cells during refrigerated storage? What methods of preparation might protect patients from the risks posed by the accelerated degradation of RBCs provided by "poor storers"? What characteristics of individual patients make them particularly vulnerable to transfusion of red ...more »

Submitted by (@andrew.dunham)

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Goal 2: Reduce Human Disease

How can we increase the pharmaceutical clinical research of targeted therapies in pediatric PAH patients, including encouraging

Clinical research, especially randomized pharmaceutical clinical trials, poses many unique challenges compared to research in adult subjects. In pulmonary arterial hypertension, a disease characterized by high blood pressure of the lungs with increased pulmonary vascular resistance leading to right ventricular failure, there are 12 FDA-approved PAH-targeted therapies for adults. None of these medications are currently ...more »

Submitted by (@katherinek)

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Goal 3: Advance Translational Research

Improving blood supply by giving a pig a human bone marrow transplant from a universal donor

What do you think of giving a pig a human bone marrow transplant from a universal donor? The pig could produce blood for human use! Although the pig will eventual die of an autoimmune disease its life can be prolonged with all the new autoimmune medicines we have and/or the pig can be engineered to overexpress PDL1 (or a similar autoimmune suppressor). Additionally even if each pint of blood made this way might be ...more »

Submitted by (@nhlbiforumadministrator1)

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Goal 3: Advance Translational Research

Tools to facilitate availability and safe use of innovative blood products and their analogs

Novel blood products are being developed based on innovative science (e.g., ex vivo manufactured RBC and platelets, and platelet and plasma derived hemostatic products). However, there is a significant lag in the development of appropriate tools and model systems, which poses a challenge when evaluating such products for regulatory approval.

Submitted by (@chintamani.atreya)

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Goal 2: Reduce Human Disease

Anemia, oxygen delivery, and red blood cell transfusion

In neonatal, pediatric, and adult patients with critical illness, what is the best means to identify: (1) the degree to which anemia contributes to insufficient oxygen (O2) delivery and (2) the likelihood that O2 delivery will be improved by red blood cell (RBC) transfusion? These questions are most relevant to critically ill populations that exhibit unique physiology, including those with low cardiac output (cardiac ...more »

Submitted by (@nareg.roubinian)

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Goal 2: Reduce Human Disease

In pulmonary arterial hypertension (PAH), how can right ventricular function be improved in the setting of increased afterload

Pulmonary arterial hypertension (PAH) is a complex, progressive condition characterized by high blood pressure in the lungs and restriction of flow through the pulmonary arterial system. Significant improvements have been made in medical management with through approved pulmonary vasodilator therapies. However, long-term right ventricular afterload reductions have still not yet been achieved. The process by which the ...more »

Submitted by (@katherinek)

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Goal 3: Advance Translational Research

To find specific medical therapies to treat the wide array of human vascular malformations and vascular tumors.

Vascular malformations and vascular tumors, together referred to as vascular anomalies, comprise a complex and wide array of diseases in which there is a fundamental disruption in blood and lymphatic vasculature. The lesions disrupt organ function, destroy tissue, cause bleeding, increase infections and can threaten life. At present, there are some medical therapies but none are specifically targeted to an underlying ...more »

Submitted by (@joyce.bischoff)

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Goal 2: Reduce Human Disease

Devices to Enhance AVF Eligibility and Maturation

There are about 400,000 patients with end-stage renal disease in the U.S. who depend on hemodialysis (HD) for their survival. Creating effective and reliable vascular access sites that can connect patients to HD machines remains a major unmet clinical need. HD patients who use an arteriovenous fistula (AVF) for vascular access live longer, healthier lives and cost less to care for. However, only about 55% of HD patients ...more »

Submitted by (@hloree)

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