Question

When utilizing a KO mouse model, you want to see if introducing the dystrophin gene can rescue the DMD mouse model. Propose a mechanism to (re)introduce dystrophin back into the KO mouse.

Answer 1

CRISPR/Cas9

Explanation:

CRISPR can be used to reintroduce dystrophin back into the KO mouse

• CRISPR stands for Clustered Regularly Interspaced Short Palindromic Repeats and is used to for gene editing
• CRISPR/Cas-mediated genome editing has been shown to permanently correct DMD mutations and restore dystrophin function in mouse models
• Germline editing by injecting zygotes with CRISPR/Cas9 editing component was first done in mdx mice by correcting the mutated exon 23
• Postnatal editing of mdx mice was then achieved using recombinant adeno-associated virus to deliver CRISPR/Cas9 genome editing components and correct the dystrophin gene by skipping or deleting the mutated exon 23 in vivo
• Germline and postnatal CRISPR/Cas9 editing approaches both successfully restored dystrophin function in the mice and same technique can be used for KO mouse model