Author

Joseph G. Gleeson

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Open access Sep 2026

AAV-mediated CBLN1 replacement rescues hereditary ataxia caused by biallelic CBLN1 variants.

These findings establish CBLN1 deficiency as a cause of hereditary ataxia and identify extracellular CBLN1 replacement as a therapeutic strategy for a reversible cerebellar synaptopathy.

Tokiwa Yamasak, W. Kakegawa, Ayumi Hayashi et al. · Molecular Therapy · 0 citations

Open access Jul 2026

Individualized antisense oligonucleotides for SCN2A-related developmental epileptic encephalopathy

In two patients with SCN2A epileptic encephalopathy, treatment with personalized allele-selective antisense oligonucleotides led to a decrease in seizure frequency with a positive safety profile, and a pathway from n = 1 to n of more patients with SCN2A-RD and other monogenic disorders is provided.

Olivia Kim-Mcmanus, L. Mignon, J. Douville et al. · Nature Medicine · 2 citations