Gene-based therapeutics for rare genetic neurodevelopmental psychiatric disorders

Mol Ther. 2022 Jul 6;30(7):2416-2428. doi: 10.1016/j.ymthe.2022.05.014. Epub 2022 May 17.


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.

Keywords: gene therapy; neurodevelopment; psychiatric disorders.

Publication types

  • Review
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Humans
  • Mental Disorders* / genetics
  • Mental Disorders* / psychology
  • Mental Disorders* / therapy
  • Precision Medicine*
  • Rare Diseases
  • United States
  • United States Food and Drug Administration