A new class of life-saving drugs is helping children who once had no hope. But some carry a price tag of millions for a ...

The treatment uses a modified, harmless virus to deliver healthy genes to a patient’s cells to treat genetic diseases. Read ...

For more than a decade, the strongest AIDS drugs could not fully control Matt Chappell's HIV infection. Now his body controls it by itself, and researchers are trying to perfect the gene editing that ...

A cutting-edge base-edited CAR T-cell therapy is opening a revolutionary new frontier for patients battling aggressive T-cell ...

Scientists at UCL and GOSH have used groundbreaking base-edited CAR‑T cell therapy — BE‑CAR7 — to treat aggressive T‑cell ...

A new gene therapy can reverse the effects of heart failure and restore heart function in a large animal model. The therapy increases the amount of blood the heart can pump and dramatically improves ...

Moci and Kael were adopted from an orphanage in China by their parents Mandy and Chris. Both have beta thalassemia major, a genetic blood disorder in which the body does not make enough hemoglobin, ...

T therapy has helped previously untreatable for T-cell acute lymphoblastic leukemia patients achieve remission, with 82% ...

The year 2023 marked a significant milestone for the treatment of sickle cell disease (SCD), with the approval of two gene therapies shown to help reduce the pain experienced by people with this ...

Menopausal hormone therapy (MHT) does not raise breast cancer risk in women with breast cancer gene mutations, while estrogen only MHT leads to reduced risk.

Duchenne muscular dystrophy (DMD) is a neuromuscular disorder that results from mutations in the DMD gene. Gene therapies for DMD change genetic material in a person’s body to treat this condition.