Antisense oligonucleotides: treatment strategies and cellular internalization

Authors

Colton M. Miller, University of Nebraska- LincolnFollow
Ed Harris, University of Nebraska - LincolnFollow

Document Type

Article

Date of this Version

2016

Citation

Colton M. Miller, et al. Antisense oligonucleotides: treatment strategies and cellular internalization. RNA Dis 2016; 3: e1393. doi: 10.14800/rd.1393.

Comments

© 2016 The Authors. Licensed under a Creative Commons Attribution 4.0 International License

Abstract

The clinical application of antisense oligonucleotides (ASOs) is becoming more of a reality as several drugs have been approved for the treatment of human disorders and many others are in various phases in development and clinical trials. ASOs are short DNA/RNA oligos which are heavily modified to increase their stability in biological fluids and retain the properties of creating RNA-RNA and DNA-RNA duplexes that knock-down or correct genetic expression. This review outlines several strategies that ASOs utilize for the treatment of various congenital diseases and syndromes that develop with aging. In addition, we discuss some of the mechanisms for specific non-targeted ASO internalization within cells.