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...

Descripció completa

Dades bibliogràfiques

Autors principals:	Francesco Errico, Carmen Marino, Manuela Grimaldi, Tommaso Nuzzo, Valentina Bassareo, Valeria Valsecchi, Chiara Panicucci, Elia Di Schiavi, Tommaso Mazza, Claudio Bruno, Adele D’Amico, Manolo Carta, Anna Maria D’Ursi, Enrico Bertini, Livio Pellizzoni, Alessandro Usiello
Format:	Artigo
Idioma:	Inglês
Publicat:	MDPI AG 2022-10-01
Col·lecció:	Biomolecules
Matèries:	spinal muscular atrophy (SMA) survival motor neuron (SMN) nusinersen cerebrospinal fluid (CSF) nuclear magnetic resonance (NMR)
Accés en línia:	https://www.mdpi.com/2218-273X/12/10/1431
Etiquetes:	Afegir etiqueta Sense etiquetes, Sigues el primer a etiquetar aquest registre!