Work

Advances in therapeutic use of a drug-stimulated translational readthrough of premature termination codons

Year: 2018

Type: review

Abstract: Premature termination codons (PTCs) in the coding regions of mRNA lead to the incorrect termination of translation and generation of non-functional, truncated proteins. Translational readthrough of PTCs induced by pharmaceutical compounds is a promising way of restoring functional protein expression and reducing disease symptoms, without affecting the genome or transcriptome of the patient. While in some cases proven effective, the clinical use of readthrough-inducing compounds is still associated with many risks and difficulties. This review focuses on problems directly associated with compounds used to stimulate PTC readthrough, such as their interactions with the cell and organism, their toxicity and bioavailability (cell permeability; tissue deposition etc.). Various strategies designed to overcome these problems are presented.

Source: Molecular Medicine

DOI: 10.1186/s10020-018-0024-7

Authors: Maciej Dabrowski, Zuzanna Bukowy-Bieryllo, Ewa Zietkiewicz

Institutions: Institute of Human Genetics, Polish Academy of Sciences

Language: English

Topic: RNA Research and Splicing

Subfield: Molecular Biology

Field: Biochemistry, Genetics and Molecular Biology

Domain: Life Sciences

SDG: Good health and well-being

Open Access status: gold

Estimated APC paid: $3,190