Gene Therapy Progress Against X-linked SCID
David Vetter was the “bubble boy’s” name and he may have been the most well known patient in medical history before his death in 1984 due to X-linked Server Combined Immune Deficiency. Besides news stories about him and a movie, his case became the public’s background for the first human gene therapy experiment in 1990. The first two gene therapy trials succeeded, but later attempts in Europe were shut down because the patients, who had SCID, developed leukemia. Much of concern over gene therapy has centered on the engineered viruses which are used to introduce transgenes. This piece reports success at Boston Children’s Hospital against X-SCID using a modified gammaretrovirus, though it does not say which gammaretrovirus was the vector or how it was modified to prevent subsequent leukemia. MORE