Review highlights lessons on nusinersen off-target effects to enhance future antisense oligonucleotide therapies.
exon 7. Nusinersen has saved thousands of lives affected by SMA. However, limitations of an ASO-based therapy continue to emerge. Here we describe lessons learned from ASO-mediated splicing corrections in general and nusinersen in particular. Specific focus of this review is to discuss how information gleaned from the off-target effects of nusinersen could be utilized to develop next generation of ASO-based therapies with improved efficacies.
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Singh et al. (2026) studied this question.
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