In effort to save the life of a baby born with a devastating neurological disease, Israeli team uses experimental gene therapy directly deliver missing gene to the child’s brain
Doctors at Schneider Children’s Medical Center in Israel have treated an eight-month-old baby with an experimental gene therapy designed to replace a missing gene linked to a fatal neurological disorder, in what the hospital described as the first procedure of its kind in the world.
The baby, from an ultra-Orthodox family in Israel, was hospitalized after suffering seizures and severe developmental decline. Genetic testing identified a rare disorder caused by the absence of a functioning gene needed for normal brain function.
