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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Detaylı Bibliyografya
Asıl Yazarlar:	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
Materyal Türü:	Artigo
Dil:	Inglês
Baskı/Yayın Bilgisi:	MDPI AG 2022-10-01
Seri Bilgileri:	Biomolecules
Konular:	spinal muscular atrophy (SMA) survival motor neuron (SMN) nusinersen cerebrospinal fluid (CSF) nuclear magnetic resonance (NMR)
Online Erişim:	https://www.mdpi.com/2218-273X/12/10/1431
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Nusinersen Induces Disease-Severity-Specific Neurometabolic Effects in Spinal Muscular Atrophy

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