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

Schule, Rebecca; Graessner, Holm; Aartsma-Rus, Annemieke; van Roon-Mom, Willeke M. C.; Synofzik, Matthis

dc.contributor.author	Graessner, Holm
dc.contributor.author	Synofzik, Matthis
dc.contributor.author	Schüle, Rebecca
dc.date.accessioned	2026-06-02T06:34:51Z
dc.date.available	2026-06-02T06:34:51Z
dc.date.issued	2025
dc.identifier.issn	2162-2531
dc.identifier.uri	http://hdl.handle.net/10900/180172
dc.language.iso	en	de_DE
dc.publisher	Cambridge : Cell Press	de_DE
dc.relation.uri	http://dx.doi.org/10.1016/j.omtn.2025.102615	de_DE
dc.subject.ddc	610	de_DE
dc.title	Tailored antisense oligonucleotides for ultrarare CNS diseases: An experience-based best practice framework for individual patient evaluation	de_DE
dc.type	Article	de_DE
utue.quellen.id	20251118000000_00750
utue.personen.roh	Schule, Rebecca
utue.personen.roh	Graessner, Holm
utue.personen.roh	Aartsma-Rus, Annemieke
utue.personen.roh	van Roon-Mom, Willeke M. C.
utue.personen.roh	Synofzik, Matthis
dcterms.isPartOf.ZSTitelID	Molecular Therapy Nucleic Acids	de_DE
dcterms.isPartOf.ZS-Issue	Article 102615	de_DE
dcterms.isPartOf.ZS-Volume	36 (3)	de_DE
utue.fakultaet	04 Medizinische Fakultät	de_DE