The Revell family hoped a gene therapy could buy time for their sons, who have a rare and fatal disease. After two patients ...

Ardeshir states, "This new hypothesis could help protect newborns in high-risk areas during the most vulnerable period of ...

For nearly three decades, Mayo Clinic researcher Christopher Evans, Ph.D., has pushed to expand gene therapy beyond its ...

A man born with Usher syndrome type 1b, a rare genetic disease that causes congenital deafness and progressive blindness, has ...

Parents of boys with Duchenne muscular dystrophy weigh in on drug innovation and medical regulation.

A groundbreaking study titled "An Engineered Intravitreal Injection Retinal-Pigment-Epithelium-Tropic Adeno-Associated Virus ...

An expert discusses how clinics can successfully implement gene therapy workflows by establishing strong institutional ...

A pivotal medical milestone has been reached. For the first time ever, researchers have used a personalized CRISPR-based gene therapy to treat an infant’s rare and life-threatening illness.

KJ Muldoon, a 10-month-old baby who sparked nationwide headlines after receiving a first-of-its kind gene-editing treatment, was released from the hospital this week. KJ has spent the majority of ...