
#343 The First Child to Receive CRISPR for Duchenne? A Father’s Fight for a Cure
Published on May 30
2423
0:000:00
<p>What would you do if your child was diagnosed with a terminal genetic condition, and the only hope for survival required raising $2.2 million?</p>
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<p>In this deeply moving episode of DNA Today, we’re joined by Joe Jackson, a rare disease advocate and father of 7-year-old William, who lives with Duchenne Muscular Dystrophy (DMD), a rare, progressive genetic disorder that leads to muscle degeneration and shortened life expectancy.</p>
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<p>But William’s story stands apart. Due to a rare duplication mutation in the DMD gene, existing treatments aren’t an option. So Joe and his wife Kati are doing something extraordinary: working with <a href='https://www.cureraredisease.org/'>Cure Rare Disease</a> to develop a personalized CRISPR gene-editing therapy that could become the first of its kind in the United States; and possibly save William’s life.</p>
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<p>In this conversation, Joe opens up about the emotional toll of a devastating diagnosis, the scientific promise ...