AUTHOR=Zhang Xiaochang TITLE=Splice-switching antisense oligonucleotides for pediatric neurological disorders JOURNAL=Frontiers in Molecular Neuroscience VOLUME=Volume 17 - 2024 YEAR=2024 URL=https://www.frontiersin.org/journals/molecular-neuroscience/articles/10.3389/fnmol.2024.1412964 DOI=10.3389/fnmol.2024.1412964 ISSN=1662-5099 ABSTRACT=Pediatric neurological disorders are frequently devastating and present unmet needs for effective medicine. The successful treatment of spinal muscular atrophy with splice-switching antisense 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 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.