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

Moazami MP., Wood MJA.

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.

DOI

10.1016/j.medj.2022.10.004

Type

Journal

Med (N Y)

Publication Date

11/11/2022

Volume

3

Pages

733 - 734

Keywords

Humans, Amyotrophic Lateral Sclerosis, Ribonuclease H, Neurodegenerative Diseases, Superoxide Dismutase-1, Superoxide Dismutase, Oligonucleotides, Antisense

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