Gene-Based Therapeutics for Rare Genetic Neurodevelopmental Psychiatric Disorders

Authors

Beverly L Davidson
Guangping Gao
Elizabeth Berry-Kravis
Allison M Bradbury
Carsten Bönnemann
Joseph D Buxbaum
Gavin R Corcoran
Steven J Gray
Heather Gray-Edwards
Robin J Kleiman
Adam J Shaywitz
Dan Wang
Huda Y Zoghbi
Terence R Flotte
Sitra Tauscher-Wisniewski
Cynthia J Tifft
Mustafa Sahin

Publication Date

7-6-2022

Journal

Molecular Therapy

DOI

10.1016/j.ymthe.2022.05.014

PMID

35585789

PMCID

PMC9263284

PubMedCentral® Posted Date

5-17-2022

PubMedCentral® Full Text Version

Post-print

Published Open-Access

yes

Keywords

Humans, Mental Disorders, Precision Medicine, Rare Diseases, United States, United States Food and Drug Administration

Abstract

We are in an emerging era of gene-based therapeutics with significant promise for rare genetic disorders. The potential is particularly significant for genetic central nervous system disorders that have begun to achieve Food and Drug Administration approval for select patient populations. This review summarizes the discussions and presentations of the National Institute of Mental Health-sponsored workshop "Gene-Based Therapeutics for Rare Genetic Neurodevelopmental Psychiatric Disorders," which was held in January 2021. Here, we distill the points raised regarding various precision medicine approaches related to neurodevelopmental and psychiatric disorders that may be amenable to gene-based therapies.