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Number of items: 3.

Journal article

Hammond, Suzan M.; Aartsma-Rus, Annemieke; Alves, Sandra; Borgos, Sven E.; Buijsen, Ronald A. M.; Collin, Rob W. J.; Covello, Giuseppina; Denti, Michela A.; Desviat, Lourdes R.; Echevarria, Lucia; Foged, Camilla; Gaina, Gisela; Garanto, Alejandro; Goyenvalle, Aurelie T.; Guzowska, Magdalena; Holodnuka, Irina; Jones, David R.; Krause, Sabine; Lehto, Taavi; Montolio, Marisol; Roon-Mom, Willeke van und Arechavala-Gomeza, Virginia (2021): Delivery of oligonucleotide-based therapeutics: challenges and opportunities. In: EMBO Molecular Medicine, Vol. 13, No. 4, e13243

Synofzik, Matthis; Roon-Mom, Willeke M. C. van; Marckmann, Georg; Duyvenvoorde, Hermine A. van; Graessner, Holm; Schule, Rebecca und Aartsma-Rus, Annemieke (2021): Preparing n-of-1 Antisense Oligonucleotide Treatments for Rare Neurological Diseases in Europe: Genetic, Regulatory, and Ethical Perspectives. In: Nucleic Acid Therapeutics, Vol. 32, No. 2: pp. 83-94

Verwey, Nisha; Gazzoli, Isabella; Krause, Sabine; Mamchaoui, Kamel; Mouly, Vincent und Aartsma-Rus, Annemieke (2019): Antisense-Mediated Skipping of Dysferlin Exons in Control and Dysferlinopathy Patient-Derived Cells. In: Nucleic Acid Therapeutics, Vol. 30, No. 2: pp. 71-79

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