CRISPR Gene-Editing Alternative Corrects Duchenne Muscular Dystrophy Mutations in Human Cells in Vitro & in Mice in Lab

Using the new gene-editing enzyme CRISPR-Cpf1, researchers at the University of Texas (UT) Southwestern Medical Center have successfully corrected Duchenne muscular dystrophy in human cells in vitro and mice in the lab. The UT Southwestern group had previously used CRISPR-Cas9, the original gene-editing system, to correct the Duchenne defect in a mouse model of the disease and in human cells. In the current work, they used a new variation of the gene-editing system to repair the defect in both a mouse model and in human cells. “We took patient-derived cells that had the most common mutation responsible for Duchenne muscular dystrophy and we corrected them in vitro to restore production of the missing dystrophin protein in the cells. This work provides us with a promising new tool in the CRISPR toolbox,” said author Dr. Eric Olson (phpto), Chairman of Molecular Biology, Co-Director of the UT Southwestern Wellstone Muscular Dystrophy Cooperative Research Center, and Director of the Hamon Center for Regenerative Science and Medicine, all at UT Southwestern. The research appeared in the April 12, 2017 issue of Science Advances. The article is titled “CRISPR-Cpf1 Correction of Muscular Dystrophy Mutations in Human Cardiomyocytes and Mice.” CRISPR-Cpf1 differs from CRISPR-Cas9 in a number of key ways. Cpf1 is much smaller than the Cas9 enzyme, which makes it easier to package inside a virus and therefore easier to deliver to muscle cells. Also, Cpf1 recognizes a different sequence of DNA than Cas9 does, which provides greater flexibility in terms of use. “There will be some genes that may be difficult to edit with Cas9, but may be easier to modify with Cpf1, or vice versa.
Login Or Register To Read Full Story