Comparative Analysis of Antisense Oligonucleotide Sequences for Targeted Skipping of Exon 51 During Dystrophin Pre-mRNA Splicing in Human Muscle

Cultured normal human skeletal muscle cells, cultured muscle cells from DMD patients (deletions 48-50 and 50), muscle explants from a DMD patient, and transgenic mice harboring the entire human DMD locus (hDMD mice).

Antisense oligonucleotides (AOs) targeting dystrophin exon 51 (8 sequences tested in 2-OMe and PMO chemistries, including AO B30).

Alternative AO sequences and mock-transfected/untreated controls.

Exon 51 skipping efficiency (measured by RT-PCR) and dystrophin protein restoration (measured by Western blot).surrogate