Article

Assessment of CSF biomarkers and microRNA-mediated disease mechanisms in SMA

2021-05-21

Abstract excerpt

<h4>Objective</h4> Cerebral spinal fluid (CSF) is a promising biospecimen for the detection of central nervous system (CNS) biomarkers to monitor therapeutic efficacy at the cellular level in neurological diseases. Spinal muscular atrophy (SMA) patients receiving intrathecal antisense oligonucleotide (nusinersen) therapy tend to show improved motor function, but the treatment effect on cellular function remains u...

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Literature Corpus work: a05c8c03-fd50-5b7e-9ffa-07e71933028b
DOI: 10.1101/2021.05.19.444816

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