New Approach Could Enhance Existing Treatments for Debilitating Genetic Disease
Tuesday, April 6, 2021
The prospect of editing our DNA to treat genetic diseases may have captured the imaginations of scientists and the public in recent years, but that doesn’t mean there aren’t other ways of combating these illnesses. Many promising therapies act not on DNA itself but rather on DNA’s often overlooked cousin, RNA. For instance, experiments in cells performed by IRP researchers have shown promising results or a RNA-targeting therapeutic developed to treat the debilitating genetic disease spinal muscular atrophy.
Biomarker Discovery Could Aid Diagnosis and Therapeutic Development
Tuesday, February 16, 2021
Our cells can’t afford to be wasteful, so they prefer to recycle broken components. However, when the mitochondria that provide their energy are damaged beyond repair, cells may have no choice but to throw them out. New IRP research suggests that more of this mitochondrial debris floats in the blood of patients with Alzheimer’s disease, potentially providing an easy, cost-effective way to diagnose or even possibly predict the illness.
First-Trimester Blood Analysis Could Enable Earlier, More Effective Intervention
Tuesday, October 20, 2020
Imagine a world in which pregnant women routinely travel to places of healing and meet with wise sages who examine a bit of their blood to divine when their babies will be born. While this may sound like something out of Greek mythology, it may soon become a reality, as IRP researchers have developed a test that was able to use blood samples taken early in pregnancy to identify women who would later deliver their babies prematurely.
Program Boosts Initiatives Supporting Researchers Across NIH
Tuesday, September 1, 2020
From Superbowl-winning football teams to comic book cohorts like The Avengers, combining the efforts of multiple talented individuals is a proven strategy for achieving remarkable results. It may come as no surprise, then, that the NIH’s Intramural Research Program (IRP) strongly encourages collaborations that breach the boundaries of its 24 Institutes and Centers. One example of these efforts is the Director’s Challenge Innovation Awards Program, which since 2009 has funded high-impact scientific projects that bring together researchers from across the IRP.
Tuesday, October 9, 2018
It seems like every day there is a new story in a prominent news outlet about the revolutionary gene-editing approach known as CRISPR/Cas9. What these reports often fail to mention is all the scientific discoveries that paved the way for that groundbreaking technology, including the key contributions of government scientists working in the Intramural Research Program of NIH’s National Human Genome Research Institute (NHGRI). Last week, the NHGRI IRP celebrated its 25th anniversary with a day-long symposium headlined by a keynote from the co-discoverer of CRISPR/Cas9, University of California, Berkeley professor Dr. Jennifer Doudna.
Tuesday, September 4, 2018
With summer winding down, it's about time we took another dive into some NIH history! These new additions to the NIH Stetten Museum collection feature some of the most prominent investigators ever to walk the NIH campus, including a Nobel prize winner and a scientist who made important discoveries about how electricity travels between neurons.
Tuesday, October 24, 2017
There are many ways to categorize the research performed at the NIH Intramural Research Program: biomedical or behavioral; computational, basic, translational, or clinical; excellent or outstanding; wow or double-wow; and so on. When we launched the irp.nih.gov website, we utilized the concept of scientific focus areas, or SFAs, and identified 21 such SFAs at the IRP, from biomedical engineering & biophysics to virology.
We thought the 21 SFAs did a rather nice job of summing up all the diverse science in the IRP. Then along comes RNA biology. It's not as if the field is new; some 30 Nobel Prizes have been won involving RNA over the decades. But the field has had a renaissance in recent years with discoveries such as that of noncoding RNA (ncRNA) functioning in genome defense and chromosome inactivation. Newly revealed classes of RNAs and their remarkable functions are poised to revolutionize molecular biology, with profound implications for clinical sciences.
Tuesday, April 4, 2017
Last month I moderated our annual retreat with the NIH Scientific Directors, those individuals tasked with leading their Institute or Center (IC)-based intramural research program. We were joined by many of the IC Clinical Directors. And this year we decided to do something a little different: listen to a series of talks about exciting, new IRP research.
Thursday, January 26, 2017
For gene therapy research, the perennial challenge has been devising a reliable way to insert safely a working copy of a gene into relevant cells that can take over for a faulty one. But with the recent discovery of powerful gene editing tools, the landscape of opportunity is starting to change. Instead of threading the needle through the cell membrane with a bulky gene, researchers are starting to design ways to apply these tools in the nucleus—to edit out the disease-causing error in a gene and allow it to work correctly.
Tuesday, February 17, 2015
You mix everything together that’s necessary for the reaction, and half the time it works, half the time it doesn’t. One day you get great PCR results, you’re on cloud nine, everything worked, and then you go repeat it to verify the result (because n never equals 1 in science), and it doesn’t work. You begin to feel like maybe you just got lucky with the first experiment.