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?

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.

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 Cure Rare Disease 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.

In this conversation, Joe opens up about the emotional toll of a devastating diagnosis, the scientific promise of genome editing, and the urgent, grassroots efforts it takes to fund a first-in-human therapy when time is running out.

Joe Jackson is a father, rare disease advocate, and fundraiser whose youngest son, William, is battling Duchenne Muscular Dystrophy. With support from Cure Rare Disease, Joe and his wife are working to develop a custom CRISPR-based gene editing therapy tailored to William’s exact mutation. His story was recently featured on CNN’s website and Instagram here. Joe continues to inspire families around the world with his passion, creativity, and determination to save his son’s life.

To support William’s custom gene therapy and learn more about Duchenne visit WeWillForWilliam.org. Every donation brings William one step closer to a groundbreaking treatment, and helps advance the future of genetic medicine.

Get ready, genetic nerds—another brand-new episode of DNA Today drops this Friday! You can always count on us to deliver fresh content every Friday. 

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DNA Today is hosted and produced by Kira Dineen, MS, LCGC, CG(ASCP)CM . Our Video Lead is Amanda Andreoli. Our Social Media Lead is Kajal Patel. Our Outreach Intern is Liv Davidson. And our logo Graphic Designer is Ashlyn Enokian, MS, CGC. 

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