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Screening rare genetic diagnoses for amenability to bespoke antisense oligonucleotide therapy development: a retrospective cohort study

2025-02-12 | Preprint
Contributors: David Cheerie; Marlen C. Lauffer; Logan Newton; Kimberly Amburgey; Danique Beijer; Bushra Haque; Brian T. Kalish; Margaret Meserve; Rachel Y. Oh; Amy Y. Pan et al.
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Consensus guidelines for eligibility assessment of pathogenic variants to antisense oligonucleotide treatments

2024-09-28 | Preprint
Contributors: David Cheerie; Margaret Meserve; Danique Beijer; Charu Kaiwar; Logan Newton; Ana Lisa Taylor Tavares; Aubrie Soucy Verran; Emma Sherrill; Stefanie Leonard; Stephan J. Sanders et al.
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