The Institute for Genomic Medicine (IGM) at Columbia University in New York is taking a precision medicine approach to study the GNB1 neurodevelopmental disorder. By using the latest genome engineering techniques (CRISPR/Cas9), they have generated human induced pluripotent stem cell (hiPSC) and genetic mouse models that replicate specific patient variants. The goal is to study the effect of these variants on neuronal activity and protein function as well as testing drugs that could correct or ameliorate adverse effects of these variants.
Please refer to the GNB1 foundation website (http://www.gnb1.org/) and www.clinicaltrials.gov for additional research opportunities.