Molecular BiologyCellPeer-reviewed
Towards a combined therapy for spinal muscular atrophy based on opposing effects of an antisense oligonucleotide on chromatin and splicing
Luciano E. Marasco, Gwendal Dujardin, Rui Sousa‐Luís…·Alberto Rodolfo Kornblihtt
The paper shows that combining an ASO with HDAC inhibitors enhances SMN2 exon 7 inclusion in SMA mice, improving SMN expression and function.
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