Revolutionary Gene Therapy: A Fairytale Cure for Children's Blood Disease
Two girls with beta thalassemia, a deadly blood disease, have become the first patients on the West Coast to receive a pioneering gene therapy offered by UCSF Benioff Children's Hospital Oakland. The therapy, called Zynteglo, uses an engineered virus to deliver a healthy gene into patient cells, aiming to permanently cure the disease. The treatment has shown promising results, with one of the girls already feeling better after 10 weeks. The therapy offers hope for an estimated 1,300 to 1,500 patients with beta thalassemia and opens up possibilities for treating other inherited disorders caused by single gene mutations. However, the treatment is expensive, costing $2.8 million for a single-use vial.
