Question

In theory, a genome editing method like CRISPR could be paired with repair by homologous recombination to cure DMD. However, repair by homologous recombination is rather inefficient. An alternative method is to use genome editing, paired with non-homologous end joining (NHEJ). Briefly explain how this strategy would work for DMD, and if it were 100% efficient, what you would expect it to do patient symptoms.

Answer 1

For the repair of DNA damage in our body,there is well known repair system is present

1. Homologous recombination repair system in which repair the segment using complementary strand as template for source of information.
2. Non homologous end joining in this mechanism no use of guide segment

Duchenne muscular dystrophy is a recessive lethal disease caused due to the mutation in dystrophin gene. NHEJ is the best method to cure the DMD disease because it repair the gene without loosing its regulation process. It is an efficient method to repair the genes.

With this approach there is formation of normal dystrophin mRNA. Normal ORF is used to generate the protein. This gives the functional dystrophin protein.