Novel genome-editing-based approaches to treat motor neuron diseases: Promises and challenges

Motor neuron diseases are untreatable with common pharmacological approaches. Spinal muscular atrophy (SMA) is caused by SMN1 gene mutations leading to lowered SMN expression. Symptoms are alleviated in infants with a higher copy number of the SMN2 gene, which, however, displays a splicing defect re...

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Detalles Bibliográficos
Autores principales: Miccio, Annarita, Antoniou, Panagiotis, Ciura, Sorana, Kabashi, Edor
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2022
Materias:
Review
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8753272/
https://www.ncbi.nlm.nih.gov/pubmed/33823304
http://dx.doi.org/10.1016/j.ymthe.2021.04.003

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8753272/
https://www.ncbi.nlm.nih.gov/pubmed/33823304
http://dx.doi.org/10.1016/j.ymthe.2021.04.003

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