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.
Authors
- Gabriela Capalbo Garrote
- Heike Felipe Rangel Dias (ORCID: https://orcid.org/0000-0001-7784-4904)
- José Henrique Amaral dos Santos
- Fernanda Melo Oliveira
- Marina Rosan Costa
- Rennyson Siqueira do Amaral
Publication Details
- Published
- 2024-09-13
- DOI
- https://doi.org/10.56238/iiicongressmedicalnursing-041
- Primary Topic
- Neurogenetic and Muscular Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 0.00