In the past three years, gene therapy has reshaped what's possible in hemophilia treatment for patients 18 and older. But a ...

Corresponding author Akshay Sharma, MBBS, MSc, St. Jude Department of Bone Marrow Transplantation and Cellular Therapy talks to St. Jude patient enrolled on the reported clinical trial, Corey Luellen.

While gene therapy for cystic fibrosis is still in the research phase, researchers aim to correct the defective gene responsible for the disease. Current research is promising. Cystic fibrosis (CF) is ...