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NIH Marks $275M for Rare Disease and Extracellular RNA Studies

NEW YORK (GenomeWeb News) – The National Institutes of Health has pledged $275 million to fund two multi-year research programs, including studies to discover the causes and cures for rare diseases and how extracellular RNA is involved in disease, NIH said today.

The new strategic funding from NIH's Common Fund includes $145 million for the Undiagnosed Diseases Program, or UDP, and $130 million for the Extracellular RNA Communication program.

The seven-year UDP is a trans-NIH program that will link up medical research centers into a network that will seek to capitalize on genomics-based advances to discover, diagnose, and ultimately develop treatments for patients who are undiagnosed.

Unveiled in 2008, the UDP seeks to spur the use of genomic data in disease diagnosis, to fund studies to identify underlying disease mechanisms, and to teach clinicians how to use genomic approaches to understand and combat undiagnosed diseases.

"This program will spawn new medical discoveries and accelerate clinical investigations that will ultimately improve the lives of thousands of patients living with undiagnosed diseases," NIH Director Francis Collins said in a statement. "Establishing a national network of clinical research centers is a critical first step towards addressing the need for more rapid and coordinated approaches to diagnose and manage rare undiagnosed diseases."

NIH estimates that around 6 percent of the US population suffers from an undiagnosed rare disorder.

The five-year Extracellular RNA Communications program will fund research projects that will seek to understand the synthesis, distribution, uptake, and function of RNAs present outside of cells, and how they are involved in health.

The research will aim to discover how both coding and non-coding RNA are secreted from cells, how they may be taken into cells, and how they affect or influence cells. The researchers also will seek information about the number of RNA molecules that are present in blood and other human fluids and will explore the use of RNAs as diagnostic or therapeutic tools in the clinic.

"Because cell-to-cell communication is critical to so many different diseases, the program will open up new avenues for therapeutic delivery of RNAs that could transform basic science and clinical practice," said James M. Anderson, director of the Division of Program Coordination, Planning, and Strategic Initiatives that guides the NIH Common Fund's programs.

“Through development of new technologies, research tools, and data, the program will lay the groundwork for new investigations into the role of RNAs in health and disease.”

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