📋 QUICK READ: This Issue's Impact on You
✅ Texas Children's performs first AADC gene therapy — historic brain surgery offers cure for ultra-rare disease
✅ UT Southwestern awarded $23M NIH grant for Alzheimer’s research — AI and digital twins advancing dementia studies
✅ Quick Hits-Texas Measles, Gene Therapy Pipeline

⏱ Estimated read time: 5 minutes

Editor's Note

In a world heavy with strife, and daily uncertainty, it's easy to forget that miracles still happen. But they do—right here in Texas.

This month, a 3-year-old received gene therapy that didn't exist when they were born. A disease that once meant five years of life now has hope written into its DNA. Literally.

I think about that child going home with "increased energy and happiness." About parents who can finally breathe. About the 350 families worldwide watching Texas lead the way.

We're living through hard times. But we're also living through the era when we learned to rewrite genes, to heal brains, to turn impossible into possible.

I’m old, well pretty old. Gene therapy? A miracle not long ago.The words didn’t even make sense together. Who knows what the next miracle will be.

Here’s to miracles!

In August 2025, Texas Children’s Hospital performed the first-ever FDA-approved gene therapy for AADC deficiency, a disorder impacting only about 350 people worldwide.

The six-hour procedure involved delivering gene therapy directly into the putamen region of a 3-year-old patient’s brain, targeting an area that controls movement and learning. Prior to this treatment (approved November 2024), AADC deficiency meant a life expectancy of just 5 to 7 years with no available cure.

Texas Children’s was the largest U.S. contributor to the clinical trial that secured FDA approval. The hospital’s Intracerebral Gene Therapy Program also explores treatments for rare diseases including Rett syndrome, NGLY-1 deficiency, and multiple Batten disease forms.

After two weeks of monitoring, the patient returned home showing signs of “increased energy and happiness.” Full therapeutic effects are expected within 2 to 6 months.

🎯 Action for you: If your child has unexplained developmental delays with poor muscle control, ask your neurologist about genetic testing for rare metabolic disorders.

📞 Texas Children's Gene Therapy Program: 832-824-4000
🔗 Full story: Texas Children's breakthrough treatment

A federally funded five-year, $23 million grant has established the North Texas Alzheimer’s Disease Research Center — one of only 37 nationwide and the second in Texas.

Texas ranks third nationwide for Alzheimer’s patients and second for related deaths, with the highest “dementia burden score” measuring caregiver impact. Unlike traditional Alzheimer’s studies, this center focuses on cardiometabolic factors, especially high blood pressure affecting over 120 million Americans, as key triggers for dementia.

Innovations include:

The center’s leadership states this designation offers a unique chance to advance understanding and treatment of Alzheimer’s disease and related dementias drastically.

🎯 For you: If you or a loved one has high blood pressure, this research could translate into new ways to prevent dementia.

📞 UT Southwestern clinical trials: 214-645-8300
🔗 utsouthwestern.edu

🦠 Texas Leads Nation in Measles Cases — Neurological Risk Rising
Texas reported measles cases in the outbreak of 2025, mostly concentrated in rural West Texas areas with low vaccination rates.

Neurological danger: Subacute sclerosing panencephalitis (SSPE), a degenerative and usually fatal condition, develops 7 to 10 years post-measles infection. Measles also wipes immune system memory for 2 to 3 years, increasing vulnerability to other infections.

🎯 Action: Confirm vaccination status if you are in or traveling to outbreak areas, and follow public health guidelines carefully.

🔬 Gene Therapy Pipeline — What’s Coming to Texas
Beyond AADC deficiency, Texas Children's active gene therapy programs include ongoing trials for:

Additionally, a local collaboration raised $6 million for rare epilepsy gene therapy, now seeking $750,000 to complete toxicology studies before human trials.

“The AADC gene therapy news made me cry. My nephew has a different rare disease, but seeing any breakthrough gives us hope that our day is coming too.” — Jennifer M., Austin

“Thank you for explaining the Alzheimer’s research in plain English. My mom has early dementia and high blood pressure. I’m calling UT Southwestern tomorrow.” — David L., Dallas

💬 Share your story (kept anonymous unless you specify): [email protected]

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NEIGHBORLY NOTE & MEDICAL DISCLAIMER

This newsletter provides educational information reviewed by a medical advisory board. Content is not intended as medical advice. Always consult your healthcare provider before making treatment decisions. Clinical trial information is provided for educational purposes only.

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