Gene-based Therapies for Neuromuscular Disorders

Overview

Journal Arq Neuropsiquiatr

Publisher Thieme

Specialties Neurology
Psychiatry

Date 2024 Feb 7

PMID 38325390

Authors

Edmar Zanoteli

Marcondes Cavalcante Franca Jr

Wilson Marques Jr

Affiliations

Soon will be listed here.

Abstract

Neuromuscular diseases (NMD) include a broad group of medical conditions with both acquired and genetic causes. In recent years, important advances have been made in the treatment of genetically caused NMD, and most of these advances are due to the implementation of therapies aimed at gene regulation. Among these therapies, gene replacement, small interfering RNA (siRNA), and antisense antinucleotides are the most promising approaches. More importantly, some of these therapies have already gained regulatory approval or are in the final stages of approval. The review focuses on motor neuron diseases, neuropathies, and Duchenne muscular dystrophy, summarizing the most recent developments in gene-based therapies for these conditions.

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