Current Strategies of Muscular Dystrophy Therapeutics: An Overview

Overview

Journal Methods Mol Biol

Specialty Molecular Biology

Date 2022 Nov 18

PMID 36401021

Authors

Kenji Rowel Q Lim

Toshifumi Yokota

Affiliations

Soon will be listed here.

Abstract

Muscular dystrophies are a group of genetic disorders characterized by varying degrees of progressive muscle weakness and degeneration. They are clinically and genetically heterogeneous but share the common histological features of dystrophic muscle. There is currently no cure for muscular dystrophies, which is of particular concern for the more disabling and/or lethal forms of the disease. Through the years, several therapies have encouragingly been developed for muscular dystrophies and include genetic, cellular, and pharmacological approaches. In this chapter, we undertake a comprehensive exploration of muscular dystrophy therapeutics under current development. Our review includes antisense therapy, CRISPR, gene replacement, cell therapy, nonsense suppression, and disease-modifying small molecule compounds.

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