Cas9 alternative Cpf1 decreases DMD pathology

In a paper published Wednesday in Science Advances, researchers from the University of Texas Southwestern Medical Center showed that CRISPR gene editing using the Cpf1 endonuclease can correct mutations in the dystrophin gene that cause Duchenne muscular dystrophy. The study is the first to show that CRISPR from Prevotella and Francisella 1 (Cpf1), an alternative to the CRISPR-associated protein 9 (Cas9) enzyme typically used for CRISPR gene editing, can correct a disease-associated mutation in human cells and an animal model.

First identified as a CRISPR-associated endonuclease by Feng Zhang's group at the Broad Institute of MIT and Harvard, Cpf1 is smaller than Cas9, making it easier to package into delivery vehicles, and has the potential to target different gene sequences. ...