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

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 efforts are u...

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Detalles Bibliográficos
Autores principales: Kordala, Anna J, Stoodley, Jessica, Ahlskog, Nina, Hanifi, Muhammad, Garcia Guerra, Antonio, Bhomra, Amarjit, Lim, Wooi Fang, Murray, Lyndsay M, Talbot, Kevin, Hammond, Suzan M, Wood, Matthew JA, Rinaldi, Carlo
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10630883/
https://www.ncbi.nlm.nih.gov/pubmed/37724723
http://dx.doi.org/10.15252/emmm.202317683