[HTML][HTML] Antisense oligonucleotides: treatment strategies and cellular internalization

CM Miller, EN Harris - RNA & disease (Houston, Tex.), 2016 - ncbi.nlm.nih.gov
CM Miller, EN Harris
RNA & disease (Houston, Tex.), 2016ncbi.nlm.nih.gov
The clinical applicaton 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 …
Abstract
The clinical applicaton 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.
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