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<p>Recent progress in the development of genetic therapies promises that impactful treatments for single-gene neurodevelopmental disorders are imminent. But can derailed neurodevelopmental processes be mended after broken genes are replaced or otherwise restored? The results of ongoing clinical trials for Angelman syndrome will soon yield answers to this pressing question, yet the trials face significant obstacles. Here we identify insights needed to aid the quest for a disease-modifying Angelman syndrome therapy, which could serve as a roadmap for the expeditious development of genetic therapies for other single-gene neurodevelopmental disorders.</p>

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Crossing the finish line towards a disease-modifying treatment for Angelman syndrome

  • 期刊论文

  • 出版模式：

    开放获取

  • 发表日期：

    2026年3月7日

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  • Matthew C. Judson,
  • Luis Pereira de Almeida,
  • Rebecca D. Burdine,
  • Stormy J. Chamberlain,
  • Benjamin E. Deverman,
  • Ben Distel,
  • Michael D. Ehlers,
  • Elizabeth Jalazo,
  • Steven A. Kushner,
  • Mark Nespeca,
  • Stephan J. Sanders,
  • Martin Scheffner,
  • Jason J. Yi,
  • Mark J. Zylka,
  • Ype Elgersma,
  • Benjamin D. Philpot

摘要

Recent progress in the development of genetic therapies promises that impactful treatments for single-gene neurodevelopmental disorders are imminent. But can derailed neurodevelopmental processes be mended after broken genes are replaced or otherwise restored? The results of ongoing clinical trials for Angelman syndrome will soon yield answers to this pressing question, yet the trials face significant obstacles. Here we identify insights needed to aid the quest for a disease-modifying Angelman syndrome therapy, which could serve as a roadmap for the expeditious development of genetic therapies for other single-gene neurodevelopmental disorders.