Precision Antisense Oligonucleotide Therapy Amenability for Infantile Genetic Epilepsies

JAMA Neurology 2026 Open access

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Authors: Emma Sherrill, David Cheerie, C S Beck, Ella F. Whittle, Yasin Shafi, Natalie J. Chandler, John Christodoulou, Jerusalem Daniel, J Hassell, Maria Lachgar-Ruiz, Sarah Mulhern, Elizabeth Scotchman
+30 more Jashanpreet Sidhu, Celine Florentia Tedja, Lyn S. Chitty, J. Helen Cross, Ingrid E. Scheffer, Haiyan Zhou, Timothy W. Yu, Vann Chau, Sarah E. M. Stephenson, Annapurna Poduri, Katherine B. Howell, Amy McTague, Gregory Costain, Alissa M. D’Gama, Gene-STEPS Study Group, Joanna Cobb, Anna J S Griffiths, Edward J Higgenbotham, Puneet Jain, Nicole S Y Liang, Sebastian Lunke, Christian R Marshall, Catherine Marx, Lyndsey McRae, Jimmy N H Nguyen, Wanqing Shao, Beth Rosen Sheidley, Lacey Smith, Zornitza Stark, Susan M. White
DOI: 10.1001/jamaneurol.2026.1021
Cited by: 1
References: 6

Abstract

This study evaluates the proportion of established antisense oligonucleotides (ASO) assessment guidelines that are amenable to ASO therapy approaches for genetically diagnosed infants.

Fields of Study & Subjects

Genomics and Rare Diseases Neurogenetic and Muscular Disorders Research DNA and Nucleic Acid Chemistry

Publication details

Journal / source
JAMA Neurology
Publisher
American Medical Association (AMA)
Volume
83
Issue
6
Pages
598–598
Publication date
4 May 2026
Language
EN