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. 2021 Oct 22;12(11):1667. doi: 10.3390/genes12111667

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© 2021 by the authors.

Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (https://creativecommons.org/licenses/by/4.0/).

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Gene therapies for syndromic autism spectrum disorders. Left; transient gene therapies such as antisense oligonucleotides (ASO), noncodingRNA (ncRNA), RNA editing and gene delivery. Right; Permanent gene therapies inlcude those that edit the host cell genome such as gene replacement (aimed at integration in the genome), CRISPR-KO (for targeted genomic disruptions) and Gene editing (for targeted repair of disease-causing mutations). Red and blue nucleotides display synthetic RNA or DNA, respectively. Green nucleotides display host-cell endogenous RNA or DNA. Created with BioRender.com.