Gene therapy proposal gains acceptance
On June 1, a National Institutes of Health (NIH) subcommittee approved use of an experimental gene therapy to treat children with a severe, inherited immune disorder. The project must clear other regulatory hurdles, but federal scientists say they may begin treating three or four children with adenosine deaminase deficiency as early as this fall.
Children afflicted with this disorder lack the enzyme adenosine deaminase, a problem that results in destruction of white cells that help the body fight infection. Under the plan, scientists would engineer white cells to carry healthy genes that code for the missing enzyme (SN: 4/7/90, p.213).
NIH researchers R. Michael Blaese and W. French Anderson note that their proposal must first win the approval of another NIH review panel and the Food and Drug Administration.
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