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

Aartsma‐Rus, Annemieke; van Roon‐Mom, Willeke; C. Lauffer, Marlen; L. E. Siezen, Christine; Duijndam, Britt; Coenen-de Roo, Tineke; Schüle, Rebecca; Synofzik, Matthis; Graeßner, Holm

doi:10.1261/rna.079540.122

Public

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

Published in RNA • Jan 20, 2023

Authors:

Annemieke Aartsma‐Rus

Willeke van Roon‐Mom

Marlen C. Lauffer

Abstract

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...

Subject

Biology

Oligonucleotide

Drug development

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