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ESID Grand Rounds 16: Where is gene therapy for PID?

Topic: Where is gene therapy for PID? Panel: Moderator: Siobhán Burns Sung-Yun Pai Carsten Speckmann Thomas Fox Claire Booth Arjan Lankester Krzysztof Kalwak Description: The webinar covers various topics related to gene therapy for PID. After participating in the webinar, participants should be able to: Understand the diagnostic evaluation for X-SCID after positive TREC newborn screening. Recognize the curative treatment options for X-SCID. Understand the impact of pre-gene therapy conditioning on the immune outcome. Understand the underlying genetic basis of X-linked severe combined immunodeficiency. Gain knowledge on how gene editing technologies work and how they can be applied to PID. Understand the advantages and disadvantages of gene addition compared to gene editing in a clinical context. Have an overview of how other advances such as non-genotoxic conditioning and in vivo correction may make gene therapy an even more attractive therapeutic approach.