Overview of alternative oligonucleotide chemistries for exon skipping.

  title={Overview of alternative oligonucleotide chemistries for exon skipping.},
  author={Amer F. Alhaj Saleh and Andrey A. Arzumanov and Michael J. Gait},
  journal={Methods in molecular biology},
The chemistry of the oligonucleotide backbone is crucial to obtaining high activity in vivo in exon skipping applications. Apart from the ability to bind strongly and sequence-specifically to pre-mRNA targets, the type of backbone also influences cell delivery, in vivo pharmacology, bio-distribution, toxicology, and ultimately the therapeutic use in humans. Reviewed here are classes of oligonucleotide commonly used for exon skipping applications, namely negatively charged backbones typified by… CONTINUE READING