Joining forces to develop individualized antisense oligonucleotides for patients with brain or eye diseases: the example of the Dutch Center for RNA Therapeutics
Therapeutic Advances in Rare Disease, vol. 5, pp. 26330040241273465
Abstract
Antisense oligonucleotides (ASOs) offer versatile tools to modify the processing and expression levels of gene transcripts. As such, they have a high therapeutic potential for rare genetic diseases, where applicability of each ASO ranges from thousands of patients worldwide to single individuals based on the prevalence of the causative pathogenic variant. It was shown that development of individualized ASOs was feasible within an academic setting, starting with Milasen for the treatment of a patient with CLN7 Batten's disease in the USA. Inspired by this, the Dutch Center for RNA Therapeutics (DCRT) was established by three academic medical centers in the Netherlands with a track record in ASO development for progressive, genetic neurodegenerative, neurodevelopmental, and retinal disorders. The goal of the DCRT is to bundle expertise and address national ethical, regulatory, and financial issues related to ASO treatment, and ultimately to develop individualized ASOs for eligible patients with genetic diseases affecting the central nervous system in an academic, not-for-profit setting. In this perspective, we describe the establishment of the DCRT in 2020 and the achievements so far, with a specific focus on lessons learned: the need for processes and procedures, the need for global collaboration, the need to raise awareness, and the fact that N-of-1 is N-of-a-few.
Authors 5
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Annemieke M Aartsma-Rus corresponding Aachen Dutch Center for RNA Therapeutic Department of Human Genetics
Leiden University Medical Center
Affiliation as printed
Dutch Center for RNA Therapeutic, Department of Human Genetics, Leiden University Medical Center, Leiden, The Netherlands
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Radboud University Nijmegen · Radboud University Medical Center
Affiliation as printed
Dutch Center for RNA Therapeutics, Department of Human Genetics, Radboud University Medical Center, Nijmegen, The Netherlands
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Affiliation as printed
Dutch Center for RNA Therapeutics, Department of Clinical Genetics, Erasmus MC, Rotterdam, The Netherlands
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Leiden University Medical Center
Affiliation as printed
Dutch Center for RNA Therapeutics, Department of Human Genetics, Leiden University Medical Center, Leiden, The Netherlands
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Leiden University Medical Center
Affiliation as printed
Dutch Center for RNA Therapeutics, Department of Human Genetics, Leiden University Medical Center, Leiden, The Netherlands
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