The NIMH is supporting research to develop and test technologies and methods for creating gene therapies aimed at treating rare neurodevelopmental disorders. This involves different gene therapy approaches like replacing, adding, editing, or silencing genes. The goal is to gather preclinical data using cell or animal experiments to evaluate the safety and effectiveness of these potential treatments.

The NIMH is encouraging research to establish, optimize, and evaluate technologies and assays needed for the clinical development of somatic cell gene therapies to treat rare neurodevelopmental disorders. This includes gene replacement, addition, editing, and silencing. The focus is on obtaining… Show More