Nature study reveals novel strategy for in vivo blood stem cell gene therapy
A team of scientists from the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy, has identified a unique window shortly after birth in which circulating blood stem cells can be effectively targeted with gene therapy directly in the body. The study, published in Nature, opens new avenues for treating some genetic blood disorders without the need for stem cell transplantation or chemotherapy.
SR-Tiget is internationally recognized as a leading center for lentiviral vector–based ...