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Article

Slow Intrathecal Injection of rAAVrh10 Enhances its Transduction of Spinal Cord and Therapeutic Efficacy in a Mutant SOD1 Model of ALS.

Neuroscience - 4 Dec 2017

Li Dongxiao, Liu Chong, Yang Chunxing, Wang Dan, Wu Dongxia, Qi Yinkuang, Su Qin, Gao Guangping, Xu Zuoshang, Guo Yansu

Abstract excerpt

Mutant SOD1 causes amyotrophic lateral sclerosis (ALS) by a dominant gain of toxicity. Previous studies have demonstrated therapeutic potential of mutant SOD1-RNAi delivered by intrathecal (IT) injection of recombinant adeno-associated virus (rAAV). However, optimization of delivery is needed to overcome the high degree of variation in the transduction efficiency and therapeutic efficacy. Here, on the basis of...

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Identifiers

PMID: 29024785
DOI: 10.1016/j.neuroscience.2017.10.001
PMCID: PMC5757625

Genes

SOD1

Variants

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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