By Fondazione Telethon – SciTechDaily –
Researchers have identified an early postnatal window that allows gene transfer to circulating blood stem cells, advancing the development of potential treatments for genetic disorders.
A team of scientists from the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy, has discovered a specific period shortly after birth when circulating blood stem cells can be effectively targeted with gene therapy directly inside the body. Published in Nature, the study opens new possibilities for treating certain genetic blood disorders without requiring stem cell transplantation or chemotherapy.
