Researchers are developing an antisense oligonucleotide (ASO) therapy
for a patient with Duchenne muscular dystrophy (DMD) who has a deletion of
exon 52 in the dystrophin gene. This deletion disrupts the reading frame,
preventing production of a functional dystrophin protein.

To address this, the therapy is designed to
cause exons 52 & 53 to be skipped during mRNA splicing .

Why could this strategy restore dystrophin function?

Question

Researchers are developing an  antisense oligonucleotide (ASO) therapy 
for a patient with  Duchenne muscular dystrophy (DMD)  who has a deletion of
 exon 52  in the  dystrophin  gene. This deletion disrupts the reading frame,
preventing production of a functional dystrophin protein.

To address this, the therapy is designed to
cause  exons 52 & 53  to be skipped during mRNA splicing .

Why could this strategy restore dystrophin function?

Accepted Answer

Skipping
exons 52 & 53 restores the correct reading frame of the mRNA, allowing
production of a shorter but functional dystrophin protein

Answer

Skipping
exon 52 & 53 increases the stability of dystrophin by removing an unstable
protein domain

Answer

Skipping
exons 52 & 53 restores dystrophin to its original wildtype length

Answer

Skipping
exons 52 & 53 removes all mutations present in the dystrophin gene

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Researchers are developing an antisense oligonucleotide (ASO) therapy for ...

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