Nusinersen Induces Disease-Severity-Specific Neurometabolic Effects in Spinal Muscular Atrophy

Intrathecal delivery of Nusinersen–an antisense oligonucleotide that promotes survival motor neuron (SMN) protein induction–is an approved therapy for spinal muscular atrophy (SMA). Here, we employed nuclear magnetic resonance (NMR) spectroscopy to longitudinally characterize the unknown metabolic e...

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Detalles Bibliográficos
Autores principales: Errico, Francesco, Marino, Carmen, Grimaldi, Manuela, Nuzzo, Tommaso, Bassareo, Valentina, Valsecchi, Valeria, Panicucci, Chiara, Di Schiavi, Elia, Mazza, Tommaso, Bruno, Claudio, D’Amico, Adele, Carta, Manolo, D’Ursi, Anna Maria, Bertini, Enrico, Pellizzoni, Livio, Usiello, Alessandro
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2022
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9599672/
https://www.ncbi.nlm.nih.gov/pubmed/36291640
http://dx.doi.org/10.3390/biom12101431

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9599672/
https://www.ncbi.nlm.nih.gov/pubmed/36291640
http://dx.doi.org/10.3390/biom12101431

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