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000163947 0247_ $$2doi$$a10.1089/nat.2021.0039
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000163947 037__ $$aDZNE-2022-00621
000163947 041__ $$aEnglish
000163947 082__ $$a610
000163947 1001_ $$0P:(DE-2719)2811275$$aSynofzik, Matthis$$b0$$eFirst author$$udzne
000163947 245__ $$aPreparing n-of-1 Antisense Oligonucleotide Treatments for Rare Neurological Diseases in Europe: Genetic, Regulatory, and Ethical Perspectives.
000163947 260__ $$aNew Rochelle, NY$$bLiebert$$c2022
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000163947 520__ $$aAntisense oligonucleotide (ASO) therapies present a promising disease-modifying treatment approach for rare neurological diseases (RNDs). However, the current focus is on 'more common' RNDs, leaving a large share of RND patients still without prospect of disease-modifying treatments. In response to this gap, n-of-1 ASO treatment approaches are targeting ultrarare or even private variants. While highly attractive, this emerging, academia-driven field of ultimately individualized precision medicine is in need of systematic guidance and standards, which will allow global scaling of this approach. We provide here genetic, regulatory, and ethical perspectives for preparing n-of-1 ASO treatments and research programs, with a specific focus on the European context. By example of splice modulating ASOs, we outline genetic criteria for variant prioritization, chart the regulatory field of n-of-1 ASO treatment development in Europe, and propose an ethically informed classification for n-of-1 ASO treatment strategies and level of outcome assessments. To accommodate the ethical requirements of both individual patient benefit and knowledge gain, we propose a stronger integration of patient care and clinical research when developing novel n-of-1 ASO treatments: each single trial of therapy should inherently be driven to generate generalizable knowledge, be registered in a ASO treatment registry, and include assessment of generic outcomes, which allow aggregated analysis across n-of-1 trials of therapy.
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000163947 650_7 $$2Other$$aantisense oligonucleotides
000163947 650_7 $$2Other$$aethics
000163947 650_7 $$2Other$$an-of-1
000163947 650_7 $$2Other$$apolicy
000163947 650_7 $$2Other$$arare diseases
000163947 650_7 $$2Other$$arare neurological diseases
000163947 650_7 $$2Other$$aregulatory
000163947 650_7 $$2NLM Chemicals$$aOligonucleotides
000163947 650_7 $$2NLM Chemicals$$aOligonucleotides, Antisense
000163947 650_2 $$2MeSH$$aEurope
000163947 650_2 $$2MeSH$$aHumans
000163947 650_2 $$2MeSH$$aOligonucleotides
000163947 650_2 $$2MeSH$$aOligonucleotides, Antisense: genetics
000163947 650_2 $$2MeSH$$aOligonucleotides, Antisense: therapeutic use
000163947 7001_ $$avan Roon-Mom, Willeke M C$$b1
000163947 7001_ $$aMarckmann, Georg$$b2
000163947 7001_ $$avan Duyvenvoorde, Hermine A$$b3
000163947 7001_ $$aGraessner, Holm$$b4
000163947 7001_ $$0P:(DE-2719)2812018$$aSchüle, Rebecca$$b5$$udzne
000163947 7001_ $$00000-0003-1565-654X$$aAartsma-Rus, Annemieke$$b6
000163947 773__ $$0PERI:(DE-600)2639888-6$$a10.1089/nat.2021.0039$$gVol. 32, no. 2, p. 83 - 94$$n2$$p83 - 94$$tNucleic acid therapeutics$$v32$$x1050-5261$$y2022
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