Gene-editing alternative corrects Duchenne muscular dystrophy

University of Texas| Thu Apr 13 11:01:39 EDT 2017
Gene-editing alternative corrects Duchenne muscular dystrophy

Using the new gene-editing enzyme CRISPR-Cpf1, researchers at UT Southwestern Medical Center have successfully corrected Duchenne muscular dystrophy in human cells 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 an....

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