A new approach to gene therapy may revolutionize how inherited blood diseases like sickle cell disease and Fanconi anemia are treated—by editing blood stem cells directly inside the body. Researchers from the IRCCS San Raffaele Scientific Institute in Italy have successfully edited genes in infant mice using a single injection, bypassing the need to extract, modify, and reintroduce stem cells—a process that is currently complex, costly, and physically demanding for patients.
The new treatment leverages a critical time window shortly after birth, when blood stem cells naturally circulate from the liver to the bone marrow. During this phase, stem cells are more accessible in the bloodstream, making them an ideal target for in vivo gene editing. In adult patients, these cells are typically hidden deep in the bone marrow, making them harder to reach.
Continue reading… “Gene Therapy Breakthrough Targets Blood Disorders in Newborns With a Single Injection”
