ADAM10 INHIBITION TO TREAT FRAGILE X SYNDROME

The present application relates to Fragile X syndrome and the treatment thereof. It was found that ADAM10 (A Disintegrin And Metalloproteinase domain10), the most likely candidate for ?-secretase, involved in proteolytic cleavage of APP at the plasma membrane, was upregulated in Fmr1 KO mice, a model for Fragile X disease. Moreover, it could be shown that reducing ADAM10 activityin vitro and in vivo, improves the Fragile X phenotype, i.a. by rescuing spine dysmorphogenesis and exaggerated mGluR-dependent LTD.

Octrooi-publicatienummer: WO2015086742

Jaar aanvraag: 2014

Jaar toekenning: 2016

Jaar van publicatie: 2015

Status: Aangevraagd

Technologiedomeinen: Farmaceutische producten

Gevalideerd voor IOF-sleutel: Ja

Toegewezen aan: Associatie KULeuven