Gene-based therapies for neuromuscular disorders
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 regulatio...
Saved in:
| Main Authors: | , , |
|---|---|
| Format: | Article |
| Language: | English |
| Published: |
Thieme Revinter Publicações
2024-06-01
|
| Series: | Arquivos de Neuro-Psiquiatria |
| Subjects: | |
| Online Access: | http://www.thieme-connect.de/DOI/DOI?10.1055/s-0043-1777755 |
| Tags: |
Add Tag
No Tags, Be the first to tag this record!
|
| _version_ | 1849470114401878016 |
|---|---|
| author | Edmar Zanoteli Marcondes Cavalcante França Wilson Marques |
| author_facet | Edmar Zanoteli Marcondes Cavalcante França Wilson Marques |
| author_sort | Edmar Zanoteli |
| collection | DOAJ |
| description | 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. |
| format | Article |
| id | doaj-art-e28f903fa4774e919921309e93bc562f |
| institution | Kabale University |
| issn | 0004-282X 1678-4227 |
| language | English |
| publishDate | 2024-06-01 |
| publisher | Thieme Revinter Publicações |
| record_format | Article |
| series | Arquivos de Neuro-Psiquiatria |
| spelling | doaj-art-e28f903fa4774e919921309e93bc562f2025-08-20T03:25:15ZengThieme Revinter PublicaçõesArquivos de Neuro-Psiquiatria0004-282X1678-42272024-06-01820600101010.1055/s-0043-1777755Gene-based therapies for neuromuscular disordersEdmar Zanoteli0https://orcid.org/0000-0002-4991-6760Marcondes Cavalcante França1https://orcid.org/0000-0003-0898-2419Wilson Marques2https://orcid.org/0000-0002-4589-2749Universidade de São Paulo, Faculdade de Medicina, Departamento de Neurologia, São Paulo SP, Brazil.Universidade Estadual de Campinas, Faculdade de Ciências Médicas, Departamento de Neurologia, Campinas SP, Brazil.Universidade de São Paulo, Faculdade de Medicina de Ribeirão Preto, Departamento de Neurociências e Ciências do Comportamento, Ribeirão Preto SP, Brazil.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.http://www.thieme-connect.de/DOI/DOI?10.1055/s-0043-1777755Gene TherapyMuscular Atrophy, SpinalAmyotrophic Lateral SclerosisAmyloid NeuropathiesMuscular Dystrophy, DuchenneTerapia GenéticaAtrofia Muscular EspinhalEsclerose Amiotrófica LateralNeuropatias AmiloidesDistrofia Muscular de Duchenne |
| spellingShingle | Edmar Zanoteli Marcondes Cavalcante França Wilson Marques Gene-based therapies for neuromuscular disorders Arquivos de Neuro-Psiquiatria Gene Therapy Muscular Atrophy, Spinal Amyotrophic Lateral Sclerosis Amyloid Neuropathies Muscular Dystrophy, Duchenne Terapia Genética Atrofia Muscular Espinhal Esclerose Amiotrófica Lateral Neuropatias Amiloides Distrofia Muscular de Duchenne |
| title | Gene-based therapies for neuromuscular disorders |
| title_full | Gene-based therapies for neuromuscular disorders |
| title_fullStr | Gene-based therapies for neuromuscular disorders |
| title_full_unstemmed | Gene-based therapies for neuromuscular disorders |
| title_short | Gene-based therapies for neuromuscular disorders |
| title_sort | gene based therapies for neuromuscular disorders |
| topic | Gene Therapy Muscular Atrophy, Spinal Amyotrophic Lateral Sclerosis Amyloid Neuropathies Muscular Dystrophy, Duchenne Terapia Genética Atrofia Muscular Espinhal Esclerose Amiotrófica Lateral Neuropatias Amiloides Distrofia Muscular de Duchenne |
| url | http://www.thieme-connect.de/DOI/DOI?10.1055/s-0043-1777755 |
| work_keys_str_mv | AT edmarzanoteli genebasedtherapiesforneuromusculardisorders AT marcondescavalcantefranca genebasedtherapiesforneuromusculardisorders AT wilsonmarques genebasedtherapiesforneuromusculardisorders |