Researchers have found a promising new method for gene therapy. They successfully restarted inactive genes by bringing them ...

Beta-thalassemia: Everyone has two beta globin genes (one on each chromosome 11). The severity depends on how many of these two genes are defective: One gene mutation: Beta-thalassemia minor ...

Gene therapy has always held enormous promise to correct genetic diseases, but turning that potential into treatments has ...

CRSP bets on Casgevy to drive growth, but its extremely high price tag and slow rollout test investor patience in a crowded ...

A new gene therapy developed at the Children's ... Nabeel was born with an inherited blood disorder called beta thalassemia. "It was just, like, so painful," she said. "Like I was in constant ...

Editas Medicine reports successful gene editing levels in stem cells, supporting potential treatment for sickle cell disease and beta thalassemia. Editas Medicine announced promising new data on ...

KJ Muldoon, a 10-month-old baby, was diagnosed with the genetic disease carbamoyl-phosphate synthetase 1 deficiency after he ...

New gene therapy improves the life of 18-year-old with Sickle Cell Disease which affects 5,000 New Jersey residents living with the condition.