RNase-H-mediated silencing in the CNS proves predictably nontrivial.
| Autor: | Moazami MP; Department of Paediatrics, University of Oxford, UK. Electronic address: michael.moazami@paediatrics.ox.ac.uk., Wood MJA; Department of Paediatrics, University of Oxford, UK. |
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| Jazyk: | angličtina |
| Zdroj: | Med (New York, N.Y.) [Med] 2022 Nov 11; Vol. 3 (11), pp. 733-734. |
| DOI: | 10.1016/j.medj.2022.10.004 |
| Abstrakt: | Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease primarily affecting motor neurons. Recently, 1 Miller et al. presented phase 3 data for the RNase-H-recruiting antisense oligonucleotide (ASO) targeting superoxide dismutase 1 (SOD1) in ALS, which represents a step forward in the deployment of ASO therapeutics against CNS targets. Competing Interests: Declaration of interests The authors declare no competing interests. (Copyright © 2022 Elsevier Inc. All rights reserved.) |
| Databáze: | MEDLINE |
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