Columbia University and Broad Institute Researchers Develop New Gene Editor for Safer, More Precise Gene Therapies

Researchers from Columbia University's Vagelos College of Physicians and Surgeons and the Broad Institute of MIT and Harvard have developed a new gene editor, called evoCAST, that inserts an entire gene into specific sites in the human genome. This breakthrough marks a significant step toward more efficient and safer gene therapies.

Gene therapy researchers have faced a challenge in inserting long stretches of DNA into targeted human genome locations without causing harmful side effects. While existing tools have enabled progress, they also come with limitations, such as random insertions and making small corrections. Samuel Sternberg, lead investigator from Columbia, said that instead of developing hundreds of gene editing drugs to address the many mutations that cause diseases like cystic fibrosis, evoCAST could insert a complete healthy gene into the patient's genome.

The research team built upon CASTs (CRISPR-associated transposases) to allow genes to "jump" to new genome locations. Unlike CRISPR-Cas9, CAST can insert large pieces of DNA without breaking the chromosome. After hundreds of evolutionary generations, evoCAST achieved successful edits of 30% to 40% of cells. In the future, the team plans to include testing the system in disease models and tackling the challenge of delivering the tool effectively to cells.

For more information, read the article from the Columbia University Irving Medical Center.