The 50 families stretch from the Netherlands and the United Kingdom to the United States and China. Each family has a child who is paralyzed from a mutation in a single gene named Contactin-Associated Protein 1 (Cntnap1).
The children are locked inside their bodies, unable to move. The families feed them and change them, and someone monitors them 24/7.
Manzoor Bhat, MS, PhD, vice dean for research in the health science center's Joe R. and Teresa Lozano Long School of Medicine "We can control when the normal gene is turned on," Bhat said. "And it turns out that we can use the normal gene to rescue the mice from their neurological deficits." Mice were placed on a beam to measure their movement. After the normal copy of the gene is turned on, and with time, the mice start passing the beam with ease. This is because the normal gene is producing protein that improves nerve signal conduction.
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