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Severe Combined Immunodeficiency Disease

Gene Therapy of Human (SCID)-X1 Reaches Clinical Trial

Published in Gene Therapy Weekly, June 15th, 2000

A clinical trial of gene therapy for severe combined immunodeficiency disease (SCID) produced positive results, researchers in France reported.

"SCID-X1 is an X-linked inherited disorder characterized by an early block in T and natural killer (NK) lymphocyte differentiation," wrote M. Cavazzana-Calvo and colleagues, Hopital Necker Enfants Maladaies, France. "This block is caused by mutations of the gene encoding the gamma c cytokine receptor subunit of interleukin-2, -4, -7, -9, and -15 receptors, which participates in the delivery of growth, survival, and differentiation signals to early lymphoid progenitors."

After successful preclinical studies,...

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