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Article

Moxifloxacin rescues SMA phenotypes in patient-derived cells and animal model.

Cellular and molecular life sciences : CMLS - 22 Jul 2022

Januel Camille, Menduti Giovanna, Mamchaoui Kamel, Martinat Cecile, Artero Ruben, Konieczny Piotr, Boido Marina

Abstract excerpt

Spinal muscular atrophy (SMA) is a genetic disease resulting in the loss of α-motoneurons followed by muscle atrophy. It is caused by knock-out mutations in the survival of motor neuron 1 (SMN1) gene, which has an unaffected, but due to preferential exon 7 skipping, only partially functional human-specific SMN2 copy. We previously described a Drosophila-based screening of FDA-approved drugs that led us to...

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Identifiers

PMID: 35864358
DOI: 10.1007/s00018-022-04450-8
PMCID: PMC9304069

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