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Article

Gene Therapies for Monogenic Autism Spectrum Disorders.

Genes - 22 Oct 2021

Weuring Wout, Geerligs Jeroen, Koeleman Bobby P C

Abstract excerpt

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...

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Identifiers

PMID: 34828273
DOI: 10.3390/genes12111667
PMCID: PMC8617899

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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