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Examples
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After a single gene-therapy treatment, four of the men started making enough of their own clotting factor that they no longer needed the regular protein injections that are currently used to prevent bleeding episodes.
Gene Therapy Used to Treat Hemophilia B Amy Docker Marcus 2011
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"The idea of treating hemophilia with gene therapy has been around for 25 years, but the problem was how to do it right," says Ronald G. Crystal , chairman of the department of genetic medicine at Weill Cornell Medical College, who is working on gene-therapy research but wasn't involved in the trial.
Gene Therapy Used to Treat Hemophilia B Amy Docker Marcus 2011
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There are now scores of gene-therapy trials under way world-wide.
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Researchers have launched a new gene-therapy trial for children with a rare disease known as "bubble boy syndrome," reflecting fresh hopes that the strategy of delivering working genes can be used to treat many intractable ailments.
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Food and Drug Administration to place a temporary halt on certain gene-therapy trials.
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A few years later, several children in two separate gene-therapy trials in Europe came down with leukemia.
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Susan Robinson, chief executive officer of Seattle-based Targeted Genetics Corp., which is involved in an ongoing gene-therapy trial in Britain for Leber's congenital amaurosis.
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Food and Drug Administration to put a hold on certain gene-therapy studies.
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Meantime, St. Jude Children's Research Hospital, Memphis, Tenn., is planning a separate gene-therapy trial for infants with SCID-X1.
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While gene-therapy studies in other diseases have gone forward, the new trial is the first involving SCID-X1 to take place in the U.S. since the hold.
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