Article

Expression of a miRNA targeting mutated SOD1 in astrocytes induces motoneuron plasticity and improves neuromuscular function in ALS mice

2021-01-09

Abstract excerpt

In amyotrophic lateral sclerosis (ALS) caused by SOD1 gene mutations, both cell-autonomous and non-cell-autonomous mechanisms lead to the selective degeneration of motoneurons. Here, we evaluate the therapeutic potential of gene therapy targeting mutated SOD1 in mature astrocytes using mice expressing the mutated SOD1 G93A protein. An AAV-gfaABC 1 D vector encoding an artificial microRNA is used to deliver RNA...

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Literature Corpus work: ca34d044-93cf-5380-babc-5c4893847387
DOI: 10.1101/2021.01.08.425706

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