RNA

CRISPR Pioneer Jennifer Doudna Headlines NHGRI 25th Anniversary Celebration

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.

Dr. Jennifer Doudna

A Summer Dive Into NIH History

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.

Dr. Marshall Nirenberg

RNA Biology Comes into Focus

mRNA

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.

Six Paths Forward in Biomedical Research

NIH Clinical Center

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.

Find and Replace: DNA Editing Tool Shows Gene Therapy Promise

Reblogged from the NIH Director’s Blog.

This image represents an infection-fighting cell called a neutrophil. In this artist’s rendering, the DNA of a cell is being “edited” with a pen-like tool to help restore its ability to fight bacterial invaders.

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.

For the Love of PCR

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.

PCR curve

Non-Coding RNAs Are Rising Stars in Gene Expression Regulation

During my Ph.D., I decided to pursue my thesis project in a lab working in the RNA field and, more specifically, on the mechanisms of alternative splicing regulation. Moving onto my post-doctoral training, I decided to stay in this field mainly because I found it fascinating to work with RNA. It is such a flexible and diverse molecule, but also largely unexplored. I believed that this relatively new area of research would attract more interest among scientists, and the last few years show that I was thinking in the right direction.

RNA Rising Stars