Precision Antisense Oligonucleotide Therapy Amenability for Infantile Genetic Epilepsies - Takeaways - MDSpire

Precision Antisense Oligonucleotide Therapy Amenability for Infantile Genetic Epilepsies

  • By

  • Emma Sherrill

  • David Cheerie

  • Cara J. Beck

  • Ella F. Whittle

  • Yasin Shafi

  • Natalie J. Chandler

  • John Christodoulou

  • Jerusalem Daniel

  • Jane Hassell

  • Maria Lachgar-Ruiz

  • Sarah Mulhern

  • Elizabeth Scotchman

  • 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 R Sheidley

  • Lacey Smith

  • Zornitza Stark

  • Susan M White

  • June 1, 2026

  • 0 min

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  • 1

    Infantile-onset epilepsies affect approximately 1 in 1200 infants and are linked to significant morbidity and mortality.

  • 2

    Antisense oligonucleotides (ASOs) are customizable therapies that bind RNA and may be applicable to genetic conditions.

  • 3

    In a cohort study of 160 infants, 16% had genetic variants amenable to ASO therapies based on established guidelines.

  • 4

    Of the infants with amenable variants, 68% could be considered for ASO therapies currently or at seizure onset.

  • 5

    The study highlights the need for rapid genome sequencing and variant assessment to identify candidates for precision therapies.

Original Source(s)

Precision Antisense Oligonucleotide Therapy Amenability for Infantile Genetic Epilepsies

  • by Emma Sherrill, David Cheerie, Cara J. Beck, Ella F. Whittle, Yasin Shafi, Natalie J. Chandler, John Christodoulou, Jerusalem Daniel, Jane Hassell, Maria Lachgar-Ruiz, Sarah Mulhern, Elizabeth Scotchman, 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 R Sheidley, Lacey Smith, Zornitza Stark, Susan M White

  • June 1, 2026

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