Counteracting chromatin effects of a splicing-correcting antisense oligonucleotide improves its therapeutic efficacy in spinal muscular atrophy
Luciano E. Marasco, Gwendal Dujardin, Rui Sousa‐Luís, Ying Hsiu Liu, José N. Stigliano, Tomoki Nomakuchi, Nick J. Proudfoot, Adrian R. Krainer, Alberto R. Kornblihtt
Topics & Concepts
SMN1Spinal muscular atrophyBiologyExonsnRNPRNA splicingChromatinSMA*Histone deacetylaseMotor neuronMolecular biologyGene silencingHistoneCancer researchCell biologyRNAGeneGeneticsNeuroscienceSpinal cordMathematicsCombinatoricsNeurogenetic and Muscular Disorders ResearchRNA Research and SplicingRNA modifications and cancer