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9085 ISTH NHF 2021 Enduring

Recent Advances in Gene Therapy for Hemophilia Clinical Trials

Which of the following gene therapy methods involves integration of genetically modified DNA into the host genome? *This question is required.
Phase 3 results based on at least 52 weeks of post-infusion data for gene therapy trials have been reported for: *This question is required.
A patient with moderately-severe hemophilia B is 18 years of age, with no history of inhibitors, and low titer AAV antibodies, has been treated on-demand for the past 5 years. Is this patient a candidate for gene therapy currently in phase 3 trials? *This question is required.
The most common adverse event reported for clinical trials of gene therapy for hemophilia is: *This question is required.