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Neonatal AAV-SIL1 therapy prevents Marinesco-Sjögren syndrome in mice

A bioRxiv preprint reports that neonatal AAV-SIL1 gene therapy prevented neurological and muscle disease in a mouse model of Marinesco-Sjögren syndrome. The treatment preserved cerebellar Purkinje cells and maintained its effects through 26 weeks.

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Preprint in mice links Angelman syndrome to seizure-prone circuits

A bioRxiv preprint reports that parvalbumin interneurons and dentate-gyrus changes shape seizure susceptibility in Angelman syndrome model mice. The findings point to altered inhibitory control and homeostatic plasticity as possible drivers of epileptogenesis.