Skip to main content

MINI REVIEW article

Front. Mol. Neurosci.
Sec. Neuroplasticity and Development
Volume 17 - 2024 | doi: 10.3389/fnmol.2024.1412964
This article is part of the Research Topic Come as You R(NA): Post-transcriptional Regulation Will Do the Rest View all 4 articles

Splice-switching antisense oligonucleotides for pediatric neurological disorders

Provisionally accepted
Xiaochang Zhang Xiaochang Zhang *
  • Department of Human Genetics, The Neuroscience Institute, University of Chicago, Chicago, IL 60637, USA, Chicago, United States

The final, formatted version of the article will be published soon.

    Pediatric neurological disorders are frequently devastating and present unmet needs for effective medicine. The successful treatment of spinal muscular atrophy with antisense splice-switching oligonucleotides (SSO) indicates a feasible path to targeting neurological disorders by redirecting pre-mRNA splicing. One direct outcome is the development of SSOs to treat haploinsufficient disorders by targeting naturally occurring non-productive splice isoforms. The recent development of personalized SSO treatment further inspired the therapeutic exploration of rare diseases. This review will discuss the recent advances that utilize SSOs to treat pediatric neurological disorders.

    Keywords: ASO, sso, neurodevelopmental disorder, Epilepsy, autism, Alternative Splicing, nonsense-mediated mRNA decay (NMD), Syngap1

    Received: 06 Apr 2024; Accepted: 12 Jul 2024.

    Copyright: © 2024 Zhang. This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) or licensor are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.

    * Correspondence: Xiaochang Zhang, Department of Human Genetics, The Neuroscience Institute, University of Chicago, Chicago, IL 60637, USA, Chicago, United States

    Disclaimer: All claims expressed in this article are solely those of the authors and do not necessarily represent those of their affiliated organizations, or those of the publisher, the editors and the reviewers. Any product that may be evaluated in this article or claim that may be made by its manufacturer is not guaranteed or endorsed by the publisher.