Gene Replacement Research and the Hope to Cure Rare Disease

Researchers are studying gene replacement therapy in rare diseases at UT Southwestern Medical Center. Many rare diseases are caused from a defect in a single gene, so replacing the defective gene with a working gene may ultimately lead to a cure. TribTalk recently share the story  of one family feeling hopeful about this area of research. The Hann family has son has Batten disease, which is caused by a defect in a single gene. In order to help their son and others with his disease, they’ve started Joseph’s Foundation for Batten Hope to raise awareness and funds for gene therapy research.

Want to learn more about this? Read the source article at TribTalk.

What are your thoughts on gene replacement therapy research? Share your stories, thoughts, and hopes with the Patient Worthy community!

Share this post

Share on facebook
Share on google
Share on twitter
Share on linkedin
Share on pinterest
Share on print
Share on email