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Table · dataset · 2026

Values for all data points in graphs in main and supplementary figures in "AAV-mediated CBLN1 replacement rescues hereditary ataxia caused by biallelic CBLN1 variants."

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<p dir="ltr">Yuzaki and colleagues identify biallelic CBLN1 variants as a cause of early-onset hereditary ataxia and show that loss of extracellular CBLN1 disrupts cerebellar synapses.

Description

Astrocyte-targeted AAV delivery restores synaptic CBLN1 and rescues circuit and motor dysfunction, establishing extracellular synaptic organizer replacement as a therapeutic strategy.</p>

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Provenance · 1 source records, 18 field assertions
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figshareoai:figshare.com:article/336851295 d agoJSON v1
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