GUNTER PUSCHResearchers have reported promising results from two gene therapy trials for the treatment of two different rare diseases. The team used lentiviral vectors to swap out faulty genes for working copies in cultures of the patients’ own hematopoietic stem cells (HSCs)—cells that mature into all different types of blood cells—before transfusing the corrected cells back into the patients. In the both trials, 18–32 months after treatment a high precentage of the patients’ blood cells were genetically corrected, and disease progression was arrested.
Moreover, there was no evidence that the vector has delivered its genetic payload into the host genome in a way that can cause leukemia—an outcome that has shuddered previous human gene therapy trials using retroviral vectors.
Although the researchers recognize that longer-term follow-ups are required, the two studies—both of which were published today (July 11) in Science—indicate that lentiviral gene therapy is a safe and effective approach to treat certain diseases.
“We now have extensive engineering of hematopoiesis in humans with what appears to be safe integration [of therapeutic genes],” said Luigi Naldini, director of the San Raffaele-Telethon Institute for Gene Therapy (TIGET) in Milan, Italy, and a coauthor on both papers.
“I was very happy to see these two papers, as they are very ...
