Be Better Award in honor of Victor Pauca

Corey Lab at UT Southwestern

The root cause of Pitt Hopkins syndrome is insufficient production of a critical protein called TCF4. Correcting this deficit at the genetic level remains one of the most promising paths toward a meaningful treatment.

Dr. Corey’s lab is pioneering the use of small activating RNAs (saRNAs)—short RNA molecules designed to precisely target and “turn on” genes that aren’t making enough protein. In this case, the therapy will focus on increasing expression of TCF4 by binding to regulatory regions of the gene, effectively boosting its activity.

The Corey Lab has spent years refining this approach and will now test its potential in Pitt Hopkins. Their immediate goal is to determine whether sbRNA can reliably elevate TCF4 protein levels—a critical proof-of-concept milestone expected within six months.

This work is part of the Foundation’s larger strategy to invest in multiple, complementary approaches to increase TCF4 and drive progress toward a cure.

We are honored to support this cutting-edge work and grateful to Dr. Corey and his team for their dedication to our children.

Read more about the Corey Lab’s work at coreyutswlab.com