Counteracting chromatin effects of a splicing-correcting antisense oligonucleotide improves its therapeutic efficacy in spinal muscular atrophy.
Marasco LE, Dujardin G, Sousa-Luis R, Liu YH, Stigliano JN, Nomakuchi T, Proudfoot NJ, Krainer AR, Kornblihtt AR
Cell. 2022 Jun 9;185(12):2057-2070.e15. doi: 10.1016/j.cell.2022.04.031.
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