The UT Southwestern gene therapy center should be up and running by the end of this year, and it may be an answer for the family of a young boy with a rare genetic neurological disorder who is running out of time. D CEO contributor Jason Heid shares the story in Texas Monthly.
When Joseph Hann was three, he began to lose his balance and vision, and after he experienced a grand mal seizure was diagnosed with a rare form Batten disease, which is almost always fatal in childhood. The disease attacks the nervous system, taking away a person’s ability to see, walk, and talk, the story says.
Hann’s family moved from Arizona to Dallas to be closer to the gene therapy center at UT Southwestern, where head of pediatric neurology Dr. Berge Minassian and others are working on a way to use healthy genes to replace diseased ones, which could slow or reverse Joseph’s deterioration.
The story walks through the families journey to raise 1.5 million for a clinical trial, (for which Joseph may not even be a candidate), as well as the development of the center and the future of gene therapy. Read the hopeful and heartbreaking piece here.