Advances in gene therapy for neuromuscular diseases

Gene therapy has shown significant potential in the treatment of neuromuscular diseases such as Duchenne muscular dystrophy and spinal muscular atrophy. Advances in viral vector technology and gene editing techniques such as CRISPR-Cas9 have opened up new therapeutic possibilities. This paper explores the main advances, clinical outcomes and challenges associated with the application of this therapeutic approach. The literature review is based on recent studies on innovations in the field of gene therapy.

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Publication Details

Published
2024-09-13
DOI
https://doi.org/10.56238/iiicongressmedicalnursing-041
Primary Topic
Neurogenetic and Muscular Disorders Research
Type
article
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article

Advances in gene therapy for neuromuscular diseases

Gabriela Capalbo Garrote, Heike Felipe Rangel Dias, José Henrique Amaral dos Santos, Fernanda Melo Oliveira et al.
Neurogenetic and Muscular Disorders Research
article

Advances in gene therapy for neuromuscular diseases

Gabriela Capalbo Garrote, Heike Felipe Rangel Dias, José Henrique Amaral dos Santos, Fernanda Melo Oliveira, Marina Rosan Costa, Rennyson Siqueira do Amaral
article en

Abstract

Gene therapy has shown significant potential in the treatment of neuromuscular diseases such as Duchenne muscular dystrophy and spinal muscular atrophy. Advances in viral vector technology and gene editing techniques such as CRISPR-Cas9 have opened up new therapeutic possibilities. This paper explores the main advances, clinical outcomes and challenges associated with the application of this therapeutic approach. The literature review is based on recent studies on innovations in the field of gene therapy.

Openalex Percentile: Top 75%
Neurogenetic and Muscular Disorders Research
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Advances in gene therapy for neuromuscular diseases — Gabriela Capalbo Garrote, Heike Felipe Rangel Dias, et al. · (2024) | TGRS Research Map | TGRS