Tailored antisense oligonucleotides for ultrarare CNS diseases: An experience-based best practice framework for individual patient evaluation

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Tailored antisense oligonucleotides for ultrarare CNS diseases: An experience-based best practice framework for individual patient evaluation

Author: Schule, Rebecca; Graessner, Holm; Aartsma-Rus, Annemieke; van Roon-Mom, Willeke M. C.; Synofzik, Matthis
Tübinger Autor(en):
Graessner, Holm
Synofzik, Matthis
Schüle, Rebecca
Published in: Molecular Therapy Nucleic Acids (2025), Bd. 36 (3), Article 102615
Verlagsangabe: Cambridge : Cell Press
Language: English
Full text: http://dx.doi.org/10.1016/j.omtn.2025.102615
ISSN: 2162-2531
DDC Classifikation: 610 - Medicine and health
Dokumentart: Article
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