NEUROMUSCULAR DISEASE

Alternative gene therapy target identified in spinal muscular atrophy mice

Virus-based delivery of the STMN1 gene, which encodes the tubulin depolymerizing protein stathmin, decreases disease severity in a mouse model of spinal muscular atrophy (SMA), say researchers. Villalón et al. induced overexpression of STMN1 in a mouse model of SMA by injecting the STMN1-carrying virus into the cerebral ventricles on postnatal day 2. When compared with untreated mice, those that received the gene therapy survived longer, had better motor performance and less pathology in neuromuscular junctions and motor neurons, and exhibited restoration of microtubule networks in the spinal cord. The findings demonstrate the potential for developing therapies for SMA with targets other than the survival motor neuron protein.

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  1. Villalón, E. et al. AAV9–Stathmin1 gene delivery improves disease phenotype in an intermediate mouse model of spinal muscular atrophy. Hum. Mol. Genet. https://doi.org/10.1093/hmg/ddz188 (2019)

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Correspondence to Sarah Lemprière.

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Lemprière, S. Alternative gene therapy target identified in spinal muscular atrophy mice. Nat Rev Neurol 15, 558 (2019). https://doi.org/10.1038/s41582-019-0262-5

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