2026-10-08T08:30:01Z https://api.figshare.com/v2/oai

oai:figshare.com:article/33685129 2026-09-13T09:26:14Z category_24733 item_type_3 month_year_09_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." Michisuke Yuzaki (16837671) Wataru Kakegawa (577209) Tokiwa Yamasaki (19001576) Central nervous system Cbln1 AAV gene therapy cerebellar ataxia secretion defect <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. Astrocyte-targeted AAV delivery restores synaptic CBLN1 and rescues circuit and motor dysfunction, establishing extracellular synaptic organizer replacement as a therapeutic strategy.</p> 2026-09-13T09:26:14Z Dataset Dataset 10.6084/m9.figshare.33685129.v1 https://figshare.com/articles/dataset/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_/33685129 CC BY 4.0