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9039 Gene Therapy ASH Enduring Evaluation

Highlights of Key Considerations: Advances in Gene Therapy for Hemophilia

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. Which of the following statements most accurately reflects findings from long-term expression of FVIII after gene therapy in canines? *This question is required.
3. When discussing gene therapy for hemophilia B, the term “Padua variant” refers to what? *This question is required.
4. Which of the following is a safety concern that has been associated with gene therapy for hemophilia in clinical trials? *This question is required.
5. Early studies involving gene therapy for hemophilia B showed: *This question is required.
6. Which of the following findings does not apply to the high dose group (6E13 vg/kg) in clinical trials for AAV5-FVIII?
  *This question is required.