In 1990, three men--W. French Anderson, R. Michael Blaese, and Kenneth Culver--led a trial in which the genetically corrected adenosine deaminase (ADA) T cells, belonging to a 4-year-old girl, were returned to her.1 Today, the 16-year-old teenager is alive and well.
It took another decade or so for any accomplishments as dramatic as that first trial to be reported, due in part to a relatively empty toolkit. In April, following a trial of gene therapy that occurred two years prior, French researchers announced that the immune systems of several children severely affected with X-linked severe combined immunodeficiency (SCID) were nearly normal, and that no supplementary therapies were involved.2 Other, less headline-grabbing reports also occurred, including work on curing fatal congenital diseases,3 reversing infertility in mice,4 treating patients with hemophilia,5 and combining different therapies with gene therapy.6
The short history of gene therapy is like a roller coaster, with quick, adrenaline-creating ...
