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Children's Hospital of Philadelphia collaborates in gene therapy success in an immune disorder
Date:11/15/2010

e defense against viruses. Over the past decade he has defined the field of human natural killer cell deficiencies in various genetic disorders.

At Children's Hospital, where he treats patients, Orange is investigating a treatment for WAS distinct from gene therapy. In a current clinical trial in WAS, his immunotherapy approach bypasses the gene defect in the disease, instead using the cytokine IL-2 to boost immune function. He expects to publish initial results shortly.

"If our immunotherapy approach is safe and effective, it may represent a treatment for the full spectrum of WAS patients," he said, noting that the gene therapy trial is currently limited to the most severe cases. "Even patients with less severe WAS are at risk for lymphoma and other blood cell cancers, so broadening treatment options would be an important advance in treatment."


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Contact: John Ascenzi
Ascenzi@email.chop.edu
267-426-6055
Children's Hospital of Philadelphia
Source:Eurekalert  

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Children's Hospital of Philadelphia collaborates in gene therapy success in an immune disorder
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