Goal 2: Reduce Human Disease

To extend our knowledge of the pathobiology of heart, lung, blood, and sleep disorders and enable clinical investigations that advance the prediction, prevention, preemption, treatment, and cures of human disease.

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 »

Submitted by (@jinsongzhang8012)

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Goal 2: Reduce Human Disease

Contribution of non-lipid factors to atherosclerotic vascular disease

Despite the generally accepted thesis linking lipids to atherosclerosis, a large number of patients fail to benefit from statins, the current lipid-lowering drugs of choice. Moreover statins are reported to cause diabetes and severe muscle weakness in some patients thus adding to the general conundrum associated with statin therapy. This prompts an important, yet unresolved question related to non-lipid factors contributing ...more »

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Goal 2: Reduce Human Disease

National cardiac arrest registry

Cardiac arrest registries are needed to measure and improve the process and outcome of resuscitation care and provide a platform for exploring insight into risk factors, prognosis, and the effectiveness of interventions for out-of-hospital (OHCA) and in-hospital cardiac arrest (IHCA).

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Goal 2: Reduce Human Disease

Clinical Trials and Rare Diseases

Strategies and infrastructure to support clinical trials in rare diseases must go further in the development of critical partnerships with advocacy organizations. These partnerships must be formalized and based on models that help to accelerate the research.

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Goal 2: Reduce Human Disease

How gene mutations contribute to defects in vascular development

How do gene mutations in endoglin and alk 1 create arteriovenous malformations leading to disease. Alk 1 and endoglin are receptors in TGFB/BMP family signaling. TGFB/BMP have roles in vascular development, remodeling and maintenance in vascular integrity. Understanding the downstream effect will lead to advancements in reducing genetic diseases such as HHT as well as vascular malformations in general

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