New era of gene therapy treats baby with ultrarare disease
Dr Kiran Musunuru and Dr Rebecca Ahrens-Nicklas with patient KJ. Credit: Children’s Hospital of Philadelphia In a major step forward...
Baby
A Pennsylvania baby is first to receive personalized gene therapy
A baby born with a rare and dangerous genetic disease is growing and thriving after getting an experimental gene editing treatment made just for...
A bespoke gene-editing treatment for a baby born with a life-threatening disorder : Shots
KJ Muldoon, who was born at Children's Hospital of Philadelphia in August, reaches out to doctors after being treated for...
Desperately ill baby healed with personalized gene therapy, doctors say
A baby born with a rare and dangerous genetic disease is growing and thriving after getting an experimental gene editing...
Baby born deaf can hear after breakthrough gene therapy
Opal Sandy from Oxfordshire is the first patient treated in a global gene therapy trial, which shows 'mind-blowing' results. She...
