rAAV Transduction Pathway and Gene Therapy Quiz
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Questions and Answers

What is the purpose of using intein-split Cas9 in the pig model of DMD?

  • To prevent malignant arrhythmias
  • To induce expression of a shortened dystrophin (correct)
  • To prolong lifespan
  • To enhance skeletal muscle function

    • What type of gene editing was performed on DMDΔ52 pigs using adeno-associated viral vectors?

  • Homology-directed repair (HDR)
  • Non-homologous end joining (NHEJ) (correct)
  • Transposon-mediated editing
  • CRISPR-Cas9 editing

    • What was the outcome of the intramuscular application of AAV9-Cas9-gE51 in the pig model of DMD?

  • Prolonged lifespan
  • Restored the CCR5 gene
  • High local rates of gene editing (correct)
  • Prevented malignant arrhythmias

    • Which gene was modified in the germline editing experiment conducted by He Jiankui?

    <p>CCR5 gene</p> Signup and view all the answers

    What was the consequence faced by He Jiankui after his germline modification experiment?

    <p>Censured by the Guangdong health ministry</p> Signup and view all the answers

    What is the significance of removing exon 51 in the context of DMD gene editing?

    <p>Restoring DMD reading frame</p> Signup and view all the answers

    What is the role of gRNAs flanking exon 51 in the DMDΔ52 pigs experiment?

    <p>Targeting sequences for NHEJ repair</p> Signup and view all the answers

    In what way did AAV9-Cas9-gE51 application contribute to improving muscle function in the pig model?

    <p>Induced expression of shortened dystrophin</p> Signup and view all the answers

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