PMID: 16629057Apr 25, 2006Paper

Antisense oligonucleotides, exon skipping and the dystrophin gene transcript

Acta Myologica : Myopathies and Cardiomyopathies : Official Journal of the Mediterranean Society of Myology
Steve D Wilton, S Fletcher

Abstract

Antisense oligonucleotide induced exon skipping has recently emerged as a potential therapy to by-pass the consequences of many, but not all dystrophin mutations that lead to Duchenne muscular dystrophy. Targeted removal of one or more exons, to restore a disrupted reading frame, or omit a nonsense mutation, could lessen the consequences of an estimated 80% of dystrophin gene mutations. Promising in vitro and in vivo experiments in animal models of dystrophinopathies, as well as demonstration of induced exon skipping in cultured human myogenic cells have prompted considerable enthusiasm. Furthermore, advances in antisense oligonucleotide chemistries have resulted in the development of more stable and less toxic compounds, some of which are currently in Phase III clinical trials for selected antiviral applications. This review will summarize developments in induced exon skipping that have paved the way to clinical trials and some of the challenges and possible limitations.

Related Concepts

Related Feeds

Antivirals

Antivirals are medications that are used specifically for treating viral infections. Discover the latest research on antivirals here.

CREs: Gene & Cell Therapy

Gene and cell therapy advances have shown promising outcomes for several diseases. The role of cis-regulatory elements (CREs) is crucial in the design of gene therapy vectors. Here is the latest research on CREs in gene and cell therapy.

Antisense Oligonucleotides: ND

This feed focuses on antisense oligonucleotide therapies such as Inotersen, Nusinursen, and Patisiran, in neurodegenerative diseases including amyotrophic lateral sclerosis.

Antivirals (ASM)

Antivirals are medications that are used specifically for treating viral infections. Discover the latest research on antivirals here.

© 2022 Meta ULC. All rights reserved