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Development of tailored splice-switching oligonucleotides for progressive brain disorders in Europe: development, regulation, and implementation considerations

Splice-modulating antisense oligonucleotides (ASOs) offer treatment options for rare neurological diseases, including those with very rare mutations, where patient-specific, individualized ASOs have to be developed. Inspired by the development of milasen, the 1 Mutation 1 Medicine (1M1M) and Dutch C...

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Detalles Bibliográficos
Autores principales:	Aartsma-Rus, Annemieke, van Roon-Mom, Willeke, Lauffer, Marlen, Siezen, Christine, Duijndam, Britt, Coenen-de Roo, Tineke, Schüle, Rebecca, Synofzik, Matthis, Graessner, Holm
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Cold Spring Harbor Laboratory Press 2023
Materias:	Perspectives
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10019362/ https://www.ncbi.nlm.nih.gov/pubmed/36669889 http://dx.doi.org/10.1261/rna.079540.122

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