Question

Is 2'-O-methyl PS still used in exon skipping for Duchenne muscular dystrophy?

Answer 1

Ans- Duchenne muscular dystrophy (DMD) is the most common inherited muscular disease in childhood mostly affected boys. DMD disease is caused by mutations in the dystrophin gene. dystrophin gene encodes a dystrophin protein of muscle cells, dystrophin proteins helps in strengthen muscle fibers and protect them from injury. Exon skipping therapy is useful to recover dystrophin expression in DMD. Antisense oligonucleotides (AOs) are used in exon skipping therapy. Initiation of exon skipping with antisense oligonucleotides (AOs) that convert out -of- frame dystrophin mRNA into in-frame sequences. Exon deletion mutations maintain the original reading frame in the mRNA. 2'-O-methyl Phosphorothioate is an example of antisense oligonucleotide (AOs) that are used in exon skipping for Duchenne muscular dystrophy. (DMD). It is important to understanding the similarities and differences in pharmacokinetics (PKs) among antisense oligonucleotises (AOs) classes. For the class 2'-O-methyl phosphorothioate (2'-O-methyl PS) most pharmacokinetics data available, are derived from development of exon skipping therapy for DMD. And, 2'-O-methyl PS also contains most of the properties for being prominent antisense oligonucleotide (AOs) for exon skipping therapy in DMD.