City of Hope researchers may have found a way to sharpen the fastest, cheapest and most accurate gene editing technique, CRISPR-Cas9, so that it can more successfully cut out undesirable genetic information.

This improved cutting ability could one day fast-track potential therapies for HIV, sickle cell disease and, potentially, other immune conditions.

“Our CRISPR-Cas9 design may be the difference between trying to cut a ribeye steak with a butter knife versus slicing it with a steak knife,” said Tristan Scott, Ph.D., lead author of the study and a staff research scientist at City of Hope’s Center for Gene Therapy. “Other scientists have tried to improve CRISPR cutting through chemical modifications, but that’s an expensive process and is like diamond-coating a blade. Instead, we have designed a better pair of scissors you can buy at any convenience store.”

The first attempt in the United States to use a gene editing tool called CRISPR against cancer seems safe in the three patients who have had it so far, but it’s too soon to know if it will improve survival, doctors reported Wednesday.

The doctors were able to take immune system cells from the patients’ blood and alter them genetically to help them recognize and fight cancer, with minimal and manageable side effects. The treatment deletes three genes that might have been hindering these cells’ ability to attack the disease, and adds a new, fourth feature to help them do the job.

“It’s the most complicated genetic, cellular engineering that’s been attempted so far,” said the study leader, Dr. Edward Stadtmauer of the University of Pennsylvania in Philadelphia. “This is proof that we can safely do gene editing of these cells.”

Read more

Scientists have developed a new gene-editing technology that could potentially correct up to 89% of genetic defects, including those that cause diseases like sickle cell anemia.

The new technique is called “prime editing,” and was developed by researchers from the Broad Institute of MIT and Harvard, who published their findings Monday in the journal Nature.

Prime editing builds on powerful CRISPR gene editing, but is more precise and versatile — it “directly writes new genetic information into a specified DNA site,” according to the paper.