8 Questions
What is the purpose of using intein-split Cas9 in the pig model of DMD?
To induce expression of a shortened dystrophin
What type of gene editing was performed on DMDΔ52 pigs using adeno-associated viral vectors?
Non-homologous end joining (NHEJ)
What was the outcome of the intramuscular application of AAV9-Cas9-gE51 in the pig model of DMD?
High local rates of gene editing
Which gene was modified in the germline editing experiment conducted by He Jiankui?
CCR5 gene
What was the consequence faced by He Jiankui after his germline modification experiment?
Censured by the Guangdong health ministry
What is the significance of removing exon 51 in the context of DMD gene editing?
Restoring DMD reading frame
What is the role of gRNAs flanking exon 51 in the DMDΔ52 pigs experiment?
Targeting sequences for NHEJ repair
In what way did AAV9-Cas9-gE51 application contribute to improving muscle function in the pig model?
Induced expression of shortened dystrophin
Test your knowledge on the rAAV transduction pathway interaction with specific receptors, such as Clathrin mediated endocytosis, and the process of gene therapy using rAAV vectors. Explore topics like episome formation, gene integration, and the application of RPE65 gene therapy for Leber congenital amaurosis, a common cause of blindness.
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