PRMT inhibitor promotes SMN2 exon 7 inclusion and synergizes with nusinersen to rescue SMA mice

PRMT inhibitor promotes SMN2 exon 7 inclusion and synergizes with nusinersen to rescue SMA mice

Abstract Spinal muscular atrophy (SMA) is a leading genetic cause of infant mortality. The advent of approved treatments for this devastating condition has significantly changed SMA patients' life expectancy and quality of life. Nevertheless, these are not without limitations, and research effo...

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Bibliographic Details
Main Authors: Anna J Kordala, Jessica Stoodley, Nina Ahlskog, Muhammad Hanifi, Antonio Garcia Guerra, Amarjit Bhomra, Wooi Fang Lim, Lyndsay M Murray, Kevin Talbot, Suzan M Hammond, Matthew JA Wood, Carlo Rinaldi
Format: Article
Language:English
Published: Springer Nature 2023-11-01
Series:EMBO Molecular Medicine
Subjects:
nusinersen
PRMT inhibitor
small molecule
spinal muscular atrophy
Online Access:https://doi.org/10.15252/emmm.202317683

Internet

https://doi.org/10.15252/emmm.202317683

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