Podcast
Questions and Answers
What is the purpose of using intein-split Cas9 in the pig model of DMD?
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?
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?
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?
Which gene was modified in the germline editing experiment conducted by He Jiankui?
What was the consequence faced by He Jiankui after his germline modification experiment?
What was the consequence faced by He Jiankui after his germline modification experiment?
What is the significance of removing exon 51 in the context of DMD gene editing?
What is the significance of removing exon 51 in the context of DMD gene editing?
What is the role of gRNAs flanking exon 51 in the DMDΔ52 pigs experiment?
What is the role of gRNAs flanking exon 51 in the DMDΔ52 pigs experiment?
In what way did AAV9-Cas9-gE51 application contribute to improving muscle function in the pig model?
In what way did AAV9-Cas9-gE51 application contribute to improving muscle function in the pig model?
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