Small Molecule Corrective Treatment in a Preclinical Mouse Model of CTNNB1 Syndrome

Time: 2:40 pm
day: Day One


• CTNNB1 syndrome is caused by CTNNB1/ beta-catenin heterozygous disruptive
gene mutations
• It is characterized by learning and motor disabilities
• Identification of a small molecule in vivo treatment that normalizes beta-catenin
levels and significantly improves learning and motor disabilities in our preclinical
mouse model