Episode 26: Gene Therapy Research with Dr. Steven Gray and Caroline Claflin

In this episode, we dive into the promising world of gene therapy with Dr. Steven Gray from the University of Texas Southwestern and Caroline Claflin, co-founder of the MEF2C Family Foundation.

Dr. Gray shares his two-decade journey in gene therapy—beginning at the University of North Carolina—where his focus on virus-based treatments has led to pioneering work in neurodegenerative and neurodevelopmental diseases such as Rett syndrome and MEF2C-related disorders. Caroline discusses the foundation’s new partnership with Dr. Gray’s lab, uniting science and community advocacy to explore AAV9 vector technology as a potential treatment path for MEF2C haploinsufficiency syndrome (MCHS).

Together, they unpack what makes AAV9 so powerful for reaching the brain, the hurdles of dose optimization and safe gene expression, and how open-source research and collaboration are accelerating discovery. Dr. Gray and Caroline also highlight the critical role of families, registries, and advocacy groups in driving research from concept to clinic.

As gene therapy moves closer to reality, this conversation offers a hopeful, behind-the-scenes look at how community and science come together to push progress forward.

Subjects covered include:

• Dr. Gray’s path into gene therapy and the evolution of AAV research

• How the AAV9 vector targets the brain and what makes it promising for MCHS

• The challenges of dose sensitivity and ensuring safe gene delivery

• The MEF2C Family Foundation’s collaboration with Dr. Gray’s lab and collaboration with Dr. Cowan's lab

• The importance of patient advocacy and open-source research

• The decreasing cost of AAV manufacturing and future accessibility

• How community participation fuels discovery and therapeutic progress

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