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Updated: Aug 22, 2025

Application of RNAi and Heat-shock-induced Transcription Factor Expression to Reprogram Germ Cells to Neurons in C. elegans
Published on: January 1, 2018
RNase-H-mediated silencing in the CNS proves predictably nontrivial
Michael P Moazami1, Matthew J A Wood1
1Department of Paediatrics, University of Oxford, UK.
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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