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Amy Wagers: Repairing Genes That Cause Muscular Dystrophy

Summary

Thousands of diseases are rooted in our genes, occurring when something goes wrong during cell multiplication and causes a mutation in the gene’s DNA sequence. This is why researchers the world over heralded the 2012 revelation of the CRISPR-Cas9 system, a groundbreaking tool for editing faulty genes. CRISPR-Cas9 allows scientists with relative ease and precision to snip out a segment of mutated or damaged DNA, correcting genes that are disease-causing and opening the door to potential treatments for diseases where there currently are none. Duchenne muscular dystrophy (DMD) is one of those diseases, and Dr. Amy Wagers of the Harvard Stem Cell Institute is leading an effort to use edited stem cells to treat Duchenne.