More than 4000 patients have been enrolled in gene transfer experiments over the last decade, but until now the research has produced few unambiguous results. Last month, a French research team announced the first clear success. Marina Cavazzana-Calvo and Alain Fischer of the Necker Hospital for Children in Paris reported that they had put a healthy gene into the bone marrow of two children with a rare, lethal immune disorder (SCID-X1), enabling the children to leave a protective bubble for the first time (Science, 28 April, p. 669). It was welcome news, adding substance to the promise that gene therapy will be used to cure genetic diseases. At the same time, however, it was a reminder of how difficult it has been to find ways of transferring genes into patients. The method used by the French team is one of the oldest in the tool kit, a “vector” based on a mouse virus. It is just one of many being developed, each with its own risks and advantages:
No takes yet. Share an insight, caveat, or question.
Eliot Marshall (2000) studied this question.