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000276161 1001_ $$0P:(DE-2719)2812633$$aLudolph, Albert$$b0$$eFirst author$$udzne
000276161 245__ $$aTofersen and other antisense oligonucleotides in ALS.
000276161 260__ $$aLondon [u.a.]$$bSage$$c2025
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000276161 520__ $$aThe advent of antisense oligonucleotide (ASO) therapies in neurodegenerative disorders is associated with enormous hope. Nusinersen treatment was a breakthrough intervention in the recessive disease spinal muscular atrophy, and superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS) seems to be the paradigm disease in dominant degenerative diseases. The results of treatment with the ASO tofersen in SOD1-ALS show that the drug has a convincing beneficial effect on ALS caused by SOD1 mutations, that preclinical studies in rodents predicted the therapeutic effect in the human disease, and that clinical efficacy is associated with a specific sequence of effects of the drug on mechanistic and degenerative biomarkers and, subsequently, functional outcomes such as weight stabilization and ALSFRS-R. Therefore, the enthusiasm seems to be justified; but this should be followed by an attempt to obtain further insights with the goal to improve this therapy. In particular, the following issues are only partially resolved: Which mechanisms are responsible for the clinical effect following the downregulation of SOD1 protein by ASOs? Is long-term downregulation of SOD1 function associated with side effects? Is there an autoimmune response caused by this and other ASO? Is prevention of SOD1-associated ALS possible?
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000276161 650_7 $$2Other$$aamyotrophic lateral sclerosis
000276161 650_7 $$2Other$$aantisense oligonucleotides
000276161 650_7 $$2Other$$asuperoxide dismutase
000276161 650_7 $$2Other$$atofersen
000276161 7001_ $$aWiesenfarth, Maximilian$$b1
000276161 773__ $$0PERI:(DE-600)2442245-9$$a10.1177/17562864251313915$$gVol. 18, p. 17562864251313915$$p17562864251313915$$tTherapeutic advances in neurological disorders$$v18$$x1756-2856$$y2025
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