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Development of tailored splice-switching oligonucleotides for progressive brain disorders in Europe: development, regulation, and implementation considerations.
Aartsma-Rus, Annemieke; van Roon-Mom, Willeke; Lauffer, Marlen; Siezen, Christine; Duijndam, Britt; Coenen-de Roo, Tineke; Schüle, Rebecca; Synofzik, Matthis; Graessner, Holm.
Afiliación
  • Aartsma-Rus A; Dutch Center for RNA Therapeutics and Department of Human Genetics, Leiden University Medical Center, 2333 ZA Leiden, The Netherlands a.m.rus@lumc.nl.
  • van Roon-Mom W; 1 Mutation 1 Medicine, 72076 Tübingen, Germany.
  • Lauffer M; Dutch Center for RNA Therapeutics and Department of Human Genetics, Leiden University Medical Center, 2333 ZA Leiden, The Netherlands.
  • Siezen C; 1 Mutation 1 Medicine, 72076 Tübingen, Germany.
  • Duijndam B; Dutch Center for RNA Therapeutics and Department of Human Genetics, Leiden University Medical Center, 2333 ZA Leiden, The Netherlands.
  • Coenen-de Roo T; Medicines Evaluation Board, 3531 AH Utrecht, The Netherlands.
  • Schüle R; Medicines Evaluation Board, 3531 AH Utrecht, The Netherlands.
  • Synofzik M; Dutch Center for RNA Therapeutics and Department of Human Genetics, Leiden University Medical Center, 2333 ZA Leiden, The Netherlands.
  • Graessner H; Animal and Transgenesis Facility, Leiden University Medical Center, 2333 ZA Leiden, The Netherlands.
RNA ; 29(4): 446-454, 2023 04.
Article en En | MEDLINE | ID: mdl-36669889
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Oligonucleótidos / Encefalopatías Límite: Humans País/Región como asunto: Europa Idioma: En Revista: RNA Asunto de la revista: BIOLOGIA MOLECULAR Año: 2023 Tipo del documento: Article País de afiliación: Países Bajos
Texto completo
Añadir a Mi BVS
Imprimir
XML
PubMed Links
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Oligonucleótidos / Encefalopatías Límite: Humans País/Región como asunto: Europa Idioma: En Revista: RNA Asunto de la revista: BIOLOGIA MOLECULAR Año: 2023 Tipo del documento: Article País de afiliación: Países Bajos