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Cell-mediated cytotoxicity within CSF and brain parenchyma in spinal muscular atrophy unaltered by nusinersen treatment

Abstract 5q-associated spinal muscular atrophy (SMA) is a motoneuron disease caused by mutations in the survival motor neuron 1 (SMN1) gene. Adaptive immunity may contribute to SMA as described in other motoneuron diseases, yet mechanisms remain elusive. Nusinersen, an antisense treatment, enhances...

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Principais autores: I-Na Lu, Phyllis Fung-Yi Cheung, Michael Heming, Christian Thomas, Giovanni Giglio, Markus Leo, Merve Erdemir, Timo Wirth, Simone König, Christine A. Dambietz, Christina B. Schroeter, Christopher Nelke, Jens T. Siveke, Tobias Ruck, Luisa Klotz, Carmen Haider, Romana Höftberger, Christoph Kleinschnitz, Heinz Wiendl, Tim Hagenacker, Gerd Meyer zu Horste
Format: Artigo
Sprog:Inglês
Udgivet: Nature Portfolio 2024-05-01
Serier:Nature Communications
Online adgang:https://doi.org/10.1038/s41467-024-48195-3
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