Scientists use gene therapy to cure immune deficient childBMJ 2002; 325 doi: https://doi.org/10.1136/bmj.325.7354.10 (Published 06 July 2002) Cite this as: BMJ 2002;325:10
- Judy Siegel-Itzkovich
An international team of scientists has managed to cure a child with severe combined immunodeficiency (SCID) using gene therapy.
Patients with SCID are usually treated by replacing an essential enzyme, adenosine deaminase, which is lacking in such people. But this treatment is effective for only a couple of days at a time and is expensive. Some patients can have a bone marrow transplant, but only if a good match can be found.
The team was headed by Professor Shimon Slavin, of the Hadassah University Hospital in Jerusalem, and his colleagues Dr Shoshana Morecki and Dr Memet Aker. They collaborated with Dr Allesandro Aiuti and other staff from the San Raffaele Institute for …