What question did this study set out to answer?

The aim is to discuss nusinersen's effectiveness and the implications of its off-target effects for future therapies.

May 21, 2026Open Access

Nusinersen: the antisense oligonucleotide at the forefront of spinal muscular atrophy treatment

Key Points

The aim is to discuss nusinersen's effectiveness and the implications of its off-target effects for future therapies.
Review of existing literature on nusinersen and antisense oligonucleotides.
Analysis of off-target effects and their implications for therapy development.
Nusinersen has significantly improved outcomes for spinal muscular atrophy patients.
Off-target effects present challenges for current ASO therapies, indicating areas for improvement.
Insights from nusinersen's application may inform next-generation oligonucleotide treatments.

Abstract

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.

Nusinersen: the antisense oligonucleotide at the forefront of spinal muscular atrophy treatment

Key Points

Abstract

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