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X-SCIDX-Linked Severe Combined Immunodeficiency
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References in periodicals archive ?
Infants with X-SCID, caused by mutations in the IL2RG gene, are highly susceptible to severe infections.
Rhys became the first in the UK and only the second in the world to receive gene therapy for x-Scid.
However, a recent incident in which two children participating in an X-SCID gene therapy trial developed leukemia highlights the need to proceed cautiously.
Rhys was born with severe combined immuno-deficiency (X-Scid) which meant the slightest contact with an infectious bug would have been fatal.
The FDA had asked the committee for direction following the report in mid-January that a second child in a clinical trial in France had developed leukemia, allegedly the result of otherwise successful treatment with gene therapy for X-linked severe combined immunodeficiency syndrome (x-SCID).
Immune measures improved with gene therapy in two patients with the X-linked severe combined immunodeficiency (X-SCID).
She had pre-natal tests at 10 weeks to see if her second child would also have severe combined immuno-deficiency (x-Scid).
The agency stressed that both children had been successfully treated by gene therapy for X-SCID and would have died without treatment.