Multi-omics profiling of CSF from spinal muscular atrophy type 3 patients after nusinersen treatment: a 2-year follow-up multicenter retrospective study

Spinal muscular atrophy (SMA) is a neurodegenerative disorder caused by mutations in the SMN1 gene resulting in reduced levels of the SMN protein. Nusinersen, the first antisense oligonucleotide (ASO) approved for SMA treatment, binds to the SMN2 gene, paralogue to SMN1, and mediates the translation...

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Detalles Bibliográficos
Autores principales: Faravelli, Irene, Gagliardi, Delia, Abati, Elena, Meneri, Megi, Ongaro, Jessica, Magri, Francesca, Parente, Valeria, Petrozzi, Lucia, Ricci, Giulia, Farè, Fiorenza, Garrone, Giulia, Fontana, Manuela, Caruso, Donatella, Siciliano, Gabriele, Comi, Giacomo Pietro, Govoni, Alessandra, Corti, Stefania, Ottoboni, Linda
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer International Publishing 2023
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10404194/
https://www.ncbi.nlm.nih.gov/pubmed/37543540
http://dx.doi.org/10.1007/s00018-023-04885-7

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10404194/
https://www.ncbi.nlm.nih.gov/pubmed/37543540
http://dx.doi.org/10.1007/s00018-023-04885-7

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