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Gene Therapy May Have Cured Four Boys

April 22, 2002|DAVID BROWN | WASHINGTON POST

French researchers say that four boys stricken with a usually fatal type of immune deficiency are living normal lives following gene therapy procedures done, in one case, as long ago as 2 1/2 years ago.

A paper in Thursday's New England Journal of Medicine expands on a report two years ago showing benefit in the months immediately following the replacement of a defective gene with a functional one. The new study, which includes the experience of two more patients and a much longer period of observation, raises the possibility the boys may be the first human beings cured by gene therapy.

Ernest Beutler, a gene-therapy expert at Scripps Research Institute in La Jolla, said of the procedure's effect: "I would guess that very likely it's going to be permanent. That's a rather comforting length of time."

All four boys have a rare condition called X-linked severe combined immunodeficiency, or SCID, which has numerous causes. In the X-linked form, immune system cells lack a receptor for substances, called interleukins, on their surfaces. It is the ailment that afflicted David, the "bubble boy" in Texas who spent most of his life in a sterile tent before dying in 1984 at age 12.

All four children now live at home. Before they were treated, all had suffered severe infections or skin conditions caused by immune deficiency. These subsided and haven't recurred, the researchers reported.

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