Gene-based therapies for neuromuscular disorders

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Citações na Scopus
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Tipo de produção
article
Data de publicação
2024
DOI
Scopus
Web of Science
PubMed
Dimensions
Título da Revista
ISSN da Revista
Título do Volume
Editora
ASSOC ARQUIVOS NEURO- PSIQUIATRIA
Autores
JR, Marcondes Cavalcante Franca
JR, Wilson Marques
Citação
ARQUIVOS DE NEURO-PSIQUIATRIA, v.82, n.6, 2024
Projetos de Pesquisa
Unidades Organizacionais
Fascículo
Resumo
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.
Palavras-chave
Gene Therapy, Muscular Atrophy, Spinal, Amyotrophic Lateral Sclerosis, Amyloid Neuropathies, Muscular Dystrophy, Duchenne
URI
https://observatorio.fm.usp.br/handle/OPI/58915
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Artigos e Materiais de Revistas Científicas - FM/MNE