Antisense oligonucleotide treatment following viral delivery of artificial SOD1-targeting miRNA shows improved efficacy in SOD1-G93A mice

Zachary C. E. Hawley; Amanda J. Guise; Ingrid D. Pardo; Twinkle Chowdhury; Sam Hana; Maria I. Zavodszky; L. Engle; Kelly Wiltberger; Daniel Bartlett; Denitza Raitcheva; Santhosh Bommegowda; Wan-Hung Lee; Shukkwan K. Chen; Adam Sheehy; Rachelle Driscoll; Michael Peterson; Jennifer Sebalusky; Stefan Hamann; Galina Marsh; Jessica Doherty; Kay-ellen Chicoine; Paige Cundiff; Sean Dwyer; Pete Clarner; Weike Zeng; Daniel Ferretti; Taras Tuczkewycz; Isabel Isaza; Melissa Patrovanie; Collin McGladrigan; J Chu; James P. Morrison; H. Moore Arnold; Shen Shen; Jessica Hurt; Patrick Trapa; Danielle Graham; Edward D. Plowey; Shih‐Ching Lo

doi:10.1016/j.omta.2026.201818

Ask about this paper

Answers are grounded in this paper and cite their sources.

Abstract

-targeting antisense oligonucleotide, provided an additive therapeutic benefit in SOD1-G93A mice compared with AAV9-amiR-SOD1 alone. This combinatorial approach lowered the viral load needed to reach efficacious endpoints, which could mitigate AAV-related adverse events alongside IMS.

Discussion

No takes yet. Share an insight, caveat, or question.

Cite This Study

Hawley et al. (2026) studied this question.

synapsesocial.com/papers/6aa91850f031ab46a4d2a97c https://doi.org/10.1016/j.omta.2026.201818

View Full Paper

Ask AI

Bookmark

Share

Antisense oligonucleotide treatment following viral delivery of artificial SOD1-targeting miRNA shows improved efficacy in SOD1-G93A mice

Ask about this paper

Abstract

Discussion

Cite This Study

Antisense oligonucleotide treatment following viral delivery of artificial SOD1-targeting miRNA shows improved efficacy in SOD1-G93A mice

Ask about this paper

Abstract

Discussion

Cite This Study

Keep reading

Keep reading