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.

Original languageEnglish
Pages (from-to)2416-2428
Number of pages13
JournalMolecular Therapy
Issue number7
StatePublished - 6 Jul 2022


  • gene therapy
  • neurodevelopment
  • psychiatric disorders


Dive into the research topics of 'Gene-based therapeutics for rare genetic neurodevelopmental psychiatric disorders'. Together they form a unique fingerprint.

Cite this