Success of Oligonucleotide Therapeutics Enabled by Modified DNA Backbone

The two oligonucleotide-based drugs on the market, that have recently been approved by U.S. FDA, have modified chemical backbone made of phosphorothioates.

The two oligonucleotide-based drugs on the market, that have recently been approved by U.S. FDA, have modified chemical backbone made of phosphorothioates. The therapeutic advantages of the phosphorothioate group and the new types of gene expression-regulation oligonucleotide drugs that it is enabling are detailed in a Review article in Nucleic Acid Therapeutics, a peer-reviewed journal from Mary Ann Liebert, Inc. publishers. The article is available free on the Nucleic Acid Therapeutics website until December 13, 2014.

In the article "Phosphorothioates, Essential Components of Therapeutic Oligonucleotides," Fritz Eckstein, Max-Planck-Institut für Experimentelle Medizin, Göttingen, Germany, describes how the chemical and biochemical properties of this significant nucleic acid modification have made DNA suitable for use as therapeutic agents. Initial applications focused on cleaving the mRNA product of genes to block protein production. Dr. Eckstein explores novel applications including microRNA and long non-coding RNA targets and the use of decoy oligonucleotides.

Quiz on DNA Fingerprinting
DNA fingerprinting, a technique that was brought forth by Sir Alec Jeffreys, has been a boon to forensic studies Test your knowledge on DNA fingerprinting by taking this quiz.

"We will advance the field of nucleic acid therapeutics by looking to key opinion leaders to educate and initiate the experienced and newcomers alike. In this review, Dr. Eckstein has produced a tour de force concerning the therapeutic application of phosphorothioates," says Executive Editor Graham C. Parker, PhD, The Carman and Ann Adams Department of Pediatrics, Wayne State University School of Medicine, Children's Hospital of Michigan, Detroit, MI.

Source-Eurekalert

About DNA Fingerprinting
DNA fingerprinting is a technique which helps forensic scientists and legal experts solve crimes, identity thefts, legal suits and terrorism cases.

Sequencing Fetal Genome With DNA from Mother's Blood
Entire genome of a fetus with the DNA obtained from the mother’s blood was sequenced.

Abrupt Shifts in Disease Linked to Steadily Rising Increases in Mitochondrial DNA Mutations
The way in which subtle changes in mitochondrial function may cause a broad range of common metabolic and degenerative diseases is highlighted.