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'Bubble Boy' Kids Living Normally After Gene Therapy: Study
Date:8/24/2011

inked SCID (SCID-X1); and six with ADA-SCID, which involves a slightly different gene mutation. All were between 6 months and 39 months old.

Gene therapy successfully treated four of the six ADA-SCID patients.

All the SCID-X1 children recovered, although one developed leukemia. That boy is currently in remission, but leukemia has been a problem with previous gene therapy trials.

Last year, French researchers reported that eight of nine male infants born with SCID-X1 had recovered as a result of gene therapy. Unfortunately, almost half developed acute leukemia, one of whom died.

The virus vector used in this earlier trial inadvertently activated an oncogene, which led to the development of the leukemia, researchers said.

The latest research circumvented this problem by using a different virus vector.

"A cloud was thrown over the field several years ago and they've solved it nicely," said Dr. Darwin Prockop, director of the Texas A&M Health Science Center College of Medicine Institute for Regenerative Medicine at Scott & White in Temple, Texas. "Very probably this can be used for other genetic diseases."

"This field came on with huge promise, then hit a few bumps and now . . . we're starting to see more and more of these successes," added Bowers.

More information

The U.S. National Human Genome Research Institute has more on SCID.

SOURCES: Donald B. Kohn, M.D., professor, microbiology, immunology and molecular genetics and pediatrics, University of California, Los Angeles; William J. Bowers, Ph.D., associate professor, neurology, University of Rochester Medical Center, Rochester, N.Y.; Darwin J. Prockop, M.D., Ph.D., director, Texas A&M Health Science Center College of Medicine Institute for Regenerative Medicine at Scott & White, Temple, Texas; Aug. 24, 2011, Science Translational Medicine
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