Gene-based therapeutics for rare genetic neurodevelopmental psychiatric disorders

Gene Therapy Workshop Faculty

doi:10.1016/j.ymthe.2022.05.014

Gene-based therapeutics for rare genetic neurodevelopmental psychiatric disorders

Gene Therapy Workshop Faculty

Research output: Contribution to journal › Review article › peer-review

8 Scopus citations

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.

Original language	English (US)
Pages (from-to)	2416-2428
Number of pages	13
Journal	Molecular Therapy
Volume	30
Issue number	7
DOIs	https://doi.org/10.1016/j.ymthe.2022.05.014
State	Published - Jul 6 2022
Externally published	Yes

Keywords

gene therapy
neurodevelopment
psychiatric disorders

ASJC Scopus subject areas

Molecular Medicine
Molecular Biology
Genetics
Pharmacology
Drug Discovery

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10.1016/j.ymthe.2022.05.014

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title = "Gene-based therapeutics for rare genetic neurodevelopmental psychiatric disorders",

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.",

keywords = "gene therapy, neurodevelopment, psychiatric disorders",

author = "{Gene Therapy Workshop Faculty} and Davidson, {Beverly L.} and Guangping Gao and Elizabeth Berry-Kravis and Bradbury, {Allison M.} and Carsten B{\"o}nnemann and Buxbaum, {Joseph D.} and Corcoran, {Gavin R.} and Gray, {Steven J.} and Heather Gray-Edwards and Kleiman, {Robin J.} and Shaywitz, {Adam J.} and Dan Wang and Zoghbi, {Huda Y.} and Flotte, {Terence R.} and Sitra Tauscher-Wisniewski and Tifft, {Cynthia J.} and Mustafa Sahin",

note = "Publisher Copyright: {\textcopyright} 2022 The Author(s)",

year = "2022",

month = jul,

day = "6",

doi = "10.1016/j.ymthe.2022.05.014",

language = "English (US)",

volume = "30",

pages = "2416--2428",

journal = "Molecular Therapy",

issn = "1525-0016",

publisher = "Nature Publishing Group",

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TY - JOUR

T1 - Gene-based therapeutics for rare genetic neurodevelopmental psychiatric disorders

AU - Gene Therapy Workshop Faculty

AU - Davidson, Beverly L.

AU - Gao, Guangping

AU - Berry-Kravis, Elizabeth

AU - Bradbury, Allison M.

AU - Bönnemann, Carsten

AU - Buxbaum, Joseph D.

AU - Corcoran, Gavin R.

AU - Gray, Steven J.

AU - Gray-Edwards, Heather

AU - Kleiman, Robin J.

AU - Shaywitz, Adam J.

AU - Wang, Dan

AU - Zoghbi, Huda Y.

AU - Flotte, Terence R.

AU - Tauscher-Wisniewski, Sitra

AU - Tifft, Cynthia J.

AU - Sahin, Mustafa

PY - 2022/7/6

Y1 - 2022/7/6

N2 - 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.

AB - 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.

KW - gene therapy

KW - neurodevelopment

KW - psychiatric disorders

UR - http://www.scopus.com/inward/record.url?scp=85131377539&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=85131377539&partnerID=8YFLogxK

U2 - 10.1016/j.ymthe.2022.05.014

DO - 10.1016/j.ymthe.2022.05.014

M3 - Review article

C2 - 35585789

AN - SCOPUS:85131377539

SN - 1525-0016

VL - 30

SP - 2416

EP - 2428

JO - Molecular Therapy

JF - Molecular Therapy

IS - 7

ER -

Gene-based therapeutics for rare genetic neurodevelopmental psychiatric disorders

Abstract

Keywords

ASJC Scopus subject areas

Access to Document

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