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

Beta thalassemia is treatable. You can manage it with blood transfusions, a treatment called iron chelation therapy, and by staying healthy. A stem cell transplant and gene therapy may also be ...

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 ...

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 ...

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.