GENE TARGET: A framework for evaluating Mendelian neurodevelopmental disorders for gene therapy.

ABSTRACT: Interest in gene-based therapies for neurodevelopmental disorders is increasing exponentially, driven by the rise in recognition of underlying genetic etiology, progress in genomic technology, and recent proof of concept in several disorders. The current prioritization of one genetic disorder over another for development of therapies is driven by competing interests of pharmaceutical companies, advocacy groups, and academic scientists. Although these are all valid perspectives, a consolidated framework will facilitate more efficient and rational gene therapy development. Here we outline features of Mendelian neurodevelopmental disorders that warrant consideration when determining suitability for gene therapy. These features fit into four broad domains: genetics, preclinical validation, cli

SUBMITTER: Chopra M 

PROVIDER: S-EPMC9478871 | biostudies-literature | 2022 Dec

REPOSITORIES: biostudies-literature