Alternative to CRISPR/Cas9 May Cause Fewer Undesired Changes
Tuesday, March 5, 2019
IRP researchers have always worked on the cutting edge of biomedical science, from testing the first successful treatment for childhood schizophrenia to pioneering the first screening technique for HIV. In a new study, an IRP team recently achieved yet another first: simultaneously editing two genetic sites in mice using a brand-new approach called base editing that may prove to be more precise – and therefore safer – than other gene editing methods.
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, February 27, 2018
Between 25 and 30 million Americans have a rare disease, defined as a condition affecting fewer than 200,000 people. On March 1, the NIH will host its annual Rare Disease Day to increase awareness of these under-recognized and often undiagnosed illnesses and highlight the efforts of scientists, patients, and advocates to produce treatments.
In anticipation of the occasion, on February 23, NIH organized a Twitter chat with NIH Director Francis Collins, M.D., Ph.D., and Sharon Terry, President and CEO of Genetic Alliance and a member of the Research Program Advisory Panel for NIH’s All of US project. Check out some of the more noteworthy exchanges below or look at the full Twitter chat by searching for #NIHchat on Twitter.
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.