A gene therapy is being developed to permanently inactivate
a dominant gain-of-function mutation that causes a severe neurological
disorder. The therapeutic strategy involves introducing a targeted
double-strand break within the mutant gene, without providing a repair
template.

Which DNA repair pathway is being deliberately exploited in
this therapeutic design, and why?

Question

A gene therapy is being developed to permanently inactivate
a dominant gain-of-function mutation that causes a severe neurological
disorder. The therapeutic strategy involves introducing a targeted
double-strand break within the mutant gene, without providing a repair
template.

Which DNA repair pathway is being deliberately exploited in
this therapeutic design, and why?

Accepted Answer

Non-homologous
end joining, because it frequently introduces insertions or deletions that
disrupt gene function

Answer

DNA
replication, to accurately copy the correct gene sequence from the intact
allele

Answer

A
high-fidelity repair pathway that restores the original DNA sequence without
mutations

Answer

Homology-directed
repair, to precisely replace the mutant sequence with a healthy one

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A gene therapy is being developed to permanently inactivate a dominant gain-of-...

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