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Success Story
The
first disease successfully treated by gene therapy was Severe Combined
Immune Deficiency ().
Researchers at the National Institutes of Health used gene therapy
to deliver functioning genes into the of a four-year-old girl. The girl's immune system functioning was
boosted to 25% of normal. She has to continue receiving injections
of the "repaired" T-cells, but lives an otherwise normal life. Thanks
to this pioneering experiment, others who are afflicted with SCID
can now receive treatment. |
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