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’...
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.
Episode Topics Include:
- What it’s like to receive a diagnosis of Duchenne Muscular Dystrophy
- How William’s rare mutation excludes him from all existing treatments
- The role of Cure Rare Disease in accelerating gene therapy development
- How CRISPR could eliminate William’s genetic duplication
- The emotional moment Joe saw rodent models with William’s mutation fully recover after CRISPR
- Why personalized gene therapies like this one can cost $2.2 million+
- What comes next once the fundraising goal is met
- The ripple effect: how William’s treatment could pave the way for other forms of DMD
- How Joe raised awareness by rowing 157 miles of the Rogue River in just 24 hours
- What the recent breakthroughs in personalized CRISPR therapies mean for the future of rare disease
Resources:
- At the beginning of the episode, the Host Kira Dineen couldn’t remember what percentage of cases of DMD are random/de novo, it’s 33% according to this study.
- Two DNA Today episodes were referenced:
- #156 Rich Horgan on Duchenne Muscular Dystrophy (Cure Rare Diseases Founder)
- #342 $10 Million for a Cure: Donating Mr. Beast’s “Beast Games” Winnings For Son’s Creatine Transporter Deficiency
- Towards the end of the interview Joe mentioned a brand new 8-minute video sharing about the “We Row For William” adventure, watch it here.
About the Guest:
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.
How You Can Help:
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.
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