Gene therapy holds the promise of preventing and curing disease by manipulating gene expression within a patient's cells. However, to be effective, the new gene must make it into a cell's nucleus. The ...

A blood stem cell gene therapy co-developed by UCLA’s Dr. Donald Kohn restored immune function in 59 of 62 children with ADA-SCID, a rare and fatal immune disorder, with no serious complications ...

This story is the first in an occasional series following Louisiana’s first patients to receive gene therapy for sickle cell disease. On the third floor of Manning Family Children’s Hospital in New ...