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September 26, 2025Journal of Translational Genetics and GenomicsOpen Access

SMN2 as a therapeutic target in spinal muscular atrophy: advances in gene expression modulation

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Authors

MVMichael J. VirataRARichard AbelloEBEdroico Mari B. Brillante

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Overview

This review discusses gene therapy and splicing modulation in spinal muscular atrophy, highlighting novel strategies and implications.

Key Points

  • SMN2 gene plays a crucial role in modifying disease severity in spinal muscular atrophy.
  • Advances in understanding splicing regulation have sparked new therapeutic strategies like gene therapy and antisense oligonucleotides.
  • Emerging approaches using CRISPR/Cas9 genome editing show potential for enhanced treatment options for spinal muscular atrophy.
  • Integrating therapies targeting SMN-dependent and -independent pathways could significantly improve treatment outcomes.

Cite This Study

Virata et al. (2025) studied this question.

synapsesocial.com/papers/68d6c68eb1249cec298b2ecehttps://doi.org/10.20517/jtgg.2025.56
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