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