Gene therapy proposal gains acceptance.Gene therapy proposal gains acceptance On June 1, a National Institutes of Health (NIH "Not invented here." See digispeak. NIH - The United States National Institutes of Health. ) 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 adenosine deaminase deficiency ADA deficiency A uniformly fatal AD disease, which consitutes 40% of Pts with SCID Clinical Cellular immune dysfunction, oral candidiasis, intractable diarrhea, FTT, severe diaper rash, pseudoachondrodysplasia, death by age 2 Lab as early as this fall. Children afflicted af·flict tr.v. af·flict·ed, af·flict·ing, af·flicts To inflict grievous physical or mental suffering on. [Middle English afflighten, from afflight, with this disorder lack the enzyme adenosine deaminase adenosine deaminase /aden·o·sine de·am·i·nase/ (ADA) (de-am´i-nas) an enzyme that catalyzes the hydrolytic deamination of adenosine to form inosine, a reaction of purine metabolism. , 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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