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9937 ISTH APSTH Evaluation-Enduring

Gene Therapy in Hemophilia: ASPTH 2021 Enduring

1. Which of the following factors is not one of the advantages of using AAV vectors for gene transfer and expression? *This question is required.
2. In the schematic of the adeno-associated virus genome below, which element(s) is/are replaced to make an AAV therapeutic vector for gene therapy?   *This question is required.
3. Of the current clinical trials for hemophilia gene therapy, what is the longest duration of post-infusion follow-up? *This question is required.
4. Which of the following statements about liver transaminase elevations in gene therapy trials is NOT correct? *This question is required.
5. Gene therapy involving ex vivo transduction and re-infusion of autologous haematopoietic stem cells potentially can overcome which of the disadvantages of AAV vector transgene infusions? *This question is required.
6. Which of the following gene replacement/editing methods are currently being used in clinical trials to study potential treatments for hemophilia? *This question is required.
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