Details

Autor(en) / Beteiligte

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

Titel

Gene-based therapeutics for rare genetic neurodevelopmental psychiatric disorders

Ist Teil von

• Molecular therapy, 2022-07, Vol.30 (7), p.2416-2428

Ort / Verlag

United States: Elsevier Inc

Erscheinungsjahr

2022

Quelle

MEDLINE

Beschreibungen/Notizen

• 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. [Display omitted] Davidson, Gao, Sahin, et al. summarize the National Institute of Mental Health-sponsored workshop “Gene-Based Therapeutics for Rare Genetic Neurodevelopmental Psychiatric Disorders,” held in January 2021. They distill the points raised regarding precision medicine approaches for neurodevelopmental and psychiatric disorders that may be amenable to gene-based therapies.