lbh589

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Combined treatment with the histone deacetylase inhibitor LBH589 and a splice-switch antisense oligonucleotide enhances SMN2 splicing and SMN expression in Spinal Muscular Atrophy cells.

Pagliarini, Vittoria;Guerra, Marika;Di Rosa, Valentina;Compagnucci, Claudia;Sette, Claudio;

2019 Journal of Neurochemistry DOI: 10.1111/jnc.14935 292 views

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Journal of Neurochemistry

ISSN: 1471-4159 376 views

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spinal muscular atrophy nusinersen therapy smn2 splicing