Amazing Things Podcast
Meet the leaders of today’s scientific revolution, and discover the new technologies allowing scientists to understand and treat disease, improve health, and overcome impossible odds. Today, research funded by the National Institutes of Health is making Amazing Things possible. The Amazing Things Podcast hosted by Adam Belmar is presented by United for Medical Research, a coalition of the nation’s leading scientific research institutions and industries, and health and patient advocates seeking steady, long-term growth in the NIH budget.
Amazing Things Podcast
Amy Wagers: Repairing Genes That Cause Muscular Dystrophy
•
United For Medical Research
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.