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Advancements in Gene Therapy for Primary Immunodeficiencies
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Overview
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The article delves into gene therapy advancements for treating Primary Immunodeficiencies, focusing on conditions like SCID and hereditary angioedema. It highlights successful trials for ADA-SCID with Strimvelis approval in the EU and ongoing U.S. trials. However, it notes limitations such as specificity and genetic variant transmission risks.
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How can gene therapy advancements in treating PIs revolutionize healthcare for other genetic disorders?
How might the success in ADA-SCID trials impact future gene therapy developments?
What ethical considerations surround the transmission of genetic variants through gene therapy?
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