For Patient Advocacy Groups
GENE TARGET Framework
We developed the GENE TARGET framework to identify gaps in the translational pipeline for monogenic neurodevelopmental disorders (NDDs). The model was first published in 2022 in the journal Molecular Therapy: Methods & Clinical Development.
This method allows investigators to calculate a GENE TARGET Suitability (GTS) score (values up to 40), based pre-clinical, clinical, ethical, and genetic considerations.
The framework informs prioritization of translational research efforts for both academic research centers and patient advocacy groups.
