Gene Therapies for Monogenic Autism Spectrum Disorders

Novel genome editing and transient gene therapies have been developed the past ten years, resulting in the first in-human clinical trials for monogenic disorders. Syndromic autism spectrum disorders can be caused by mutations in a single gene. Given the monogenic aspect and severity of syndromic ASD...

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Autores principales:	Wout Weuring, Jeroen Geerligs, Bobby P. C. Koeleman
Formato:	article
Lenguaje:	EN
Publicado:	MDPI AG 2021
Materias:	ASD gene therapy gene edit RNA therapy epilepsy gene replacement Genetics QH426-470
Acceso en línea:	https://doaj.org/article/36cb1abb0d714d319b1a54ec5f25a48f
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spelling	oai:doaj.org-article:36cb1abb0d714d319b1a54ec5f25a48f2021-11-25T17:40:30ZGene Therapies for Monogenic Autism Spectrum Disorders10.3390/genes121116672073-4425https://doaj.org/article/36cb1abb0d714d319b1a54ec5f25a48f2021-10-01T00:00:00Zhttps://www.mdpi.com/2073-4425/12/11/1667https://doaj.org/toc/2073-4425Novel genome editing and transient gene therapies have been developed the past ten years, resulting in the first in-human clinical trials for monogenic disorders. Syndromic autism spectrum disorders can be caused by mutations in a single gene. Given the monogenic aspect and severity of syndromic ASD, it is an ideal candidate for gene therapies. Here, we selected 11 monogenic ASD syndromes, validated by animal models, and reviewed current gene therapies for each syndrome. Given the wide variety and novelty of some forms of gene therapy, the best possible option must be decided based on the gene and mutation.Wout WeuringJeroen GeerligsBobby P. C. KoelemanMDPI AGarticleASDgene therapygene editRNA therapyepilepsygene replacementGeneticsQH426-470ENGenes, Vol 12, Iss 1667, p 1667 (2021)
institution	DOAJ
collection	DOAJ
language	EN
topic	ASD gene therapy gene edit RNA therapy epilepsy gene replacement Genetics QH426-470
spellingShingle	ASD gene therapy gene edit RNA therapy epilepsy gene replacement Genetics QH426-470 Wout Weuring Jeroen Geerligs Bobby P. C. Koeleman Gene Therapies for Monogenic Autism Spectrum Disorders
description	Novel genome editing and transient gene therapies have been developed the past ten years, resulting in the first in-human clinical trials for monogenic disorders. Syndromic autism spectrum disorders can be caused by mutations in a single gene. Given the monogenic aspect and severity of syndromic ASD, it is an ideal candidate for gene therapies. Here, we selected 11 monogenic ASD syndromes, validated by animal models, and reviewed current gene therapies for each syndrome. Given the wide variety and novelty of some forms of gene therapy, the best possible option must be decided based on the gene and mutation.
format	article
author	Wout Weuring Jeroen Geerligs Bobby P. C. Koeleman
author_facet	Wout Weuring Jeroen Geerligs Bobby P. C. Koeleman
author_sort	Wout Weuring
title	Gene Therapies for Monogenic Autism Spectrum Disorders
title_short	Gene Therapies for Monogenic Autism Spectrum Disorders
title_full	Gene Therapies for Monogenic Autism Spectrum Disorders
title_fullStr	Gene Therapies for Monogenic Autism Spectrum Disorders
title_full_unstemmed	Gene Therapies for Monogenic Autism Spectrum Disorders
title_sort	gene therapies for monogenic autism spectrum disorders
publisher	MDPI AG
publishDate	2021
url	https://doaj.org/article/36cb1abb0d714d319b1a54ec5f25a48f
work_keys_str_mv	AT woutweuring genetherapiesformonogenicautismspectrumdisorders AT jeroengeerligs genetherapiesformonogenicautismspectrumdisorders AT bobbypckoeleman genetherapiesformonogenicautismspectrumdisorders
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Gene Therapies for Monogenic Autism Spectrum Disorders

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