Duchenne Muscular dystrophy (DMD) can be caused by a mutation leading to the exclusion of exons 49 and 50 of the dystrophin gene (exon pattern illustrated below).
Which of the following proposed antisense oligonucleotide (ASO) treatment would likely be most effective for patients with this mutation, to allow them to produce some functional dystrophin protein?

Question

Duchenne Muscular dystrophy (DMD) can be caused by a mutation leading to the exclusion of exons 49 and 50 of the dystrophin gene (exon pattern illustrated below).

Which of the following proposed antisense oligonucleotide (ASO) treatment would likely be most effective for patients with this mutation, to allow them to produce some functional dystrophin protein?

DMD patient exon pattern

Accepted Answer

A antisense oligonucleotide therapy that results in skipping of exon 51

Answer

A antisense oligonucleotide therapy that results in skipping of exon 51 & 52

Answer

A antisense oligonucleotide therapy that results in skipping of exon 47 & 48

Answer

A antisense oligonucleotide therapy that results in skipping of exon 48, 51 & 52

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Duchenne Muscular dystrophy (DMD) can be caused by a mutation leading to the exc...

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