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Comment
. 2022 Nov 11;3(11):733-734.
doi: 10.1016/j.medj.2022.10.004.

RNase-H-mediated silencing in the CNS proves predictably nontrivial

Michael P Moazami  1 , Matthew J A Wood  2
Affiliations
Free article
Comment

RNase-H-mediated silencing in the CNS proves predictably nontrivial

Michael P Moazami et al. Med. .
Free article

Abstract

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.

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Conflict of interest statement

Declaration of interests The authors declare no competing interests.

Comment on

  • Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS.
    Miller TM, Cudkowicz ME, Genge A, Shaw PJ, Sobue G, Bucelli RC, Chiò A, Van Damme P, Ludolph AC, Glass JD, Andrews JA, Babu S, Benatar M, McDermott CJ, Cochrane T, Chary S, Chew S, Zhu H, Wu F, Nestorov I, Graham D, Sun P, McNeill M, Fanning L, Ferguson TA, Fradette S; VALOR and OLE Working Group. Miller TM, et al. N Engl J Med. 2022 Sep 22;387(12):1099-1110. doi: 10.1056/NEJMoa2204705. N Engl J Med. 2022. PMID: 36129998 Clinical Trial.

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