A CRISPR-Cas9–engineered mouse model for GPI-anchor deficiency mirrors human phenotypes and exhibits hippocampal synaptic dysfunctions

Significance Inherited GPI-anchor biosynthesis deficiencies (IGDs) explain many cases of syndromic intellectual disability. Although diagnostic methods are improving, the pathophysiology underlying disease remains unclear. Furthermore, we lack rodent models suitable for characterizing cognitive and social disabilities. To address this issue, generated a viable mouse model an IGD that mirrors condition in human patients with behavioral phenotype susceptibility to epilepsy. Using model, obtained neurological insights such as deficits synaptic transmission will facilitate understanding IGDs.