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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Main Authors: | Wout Weuring, Jeroen Geerligs, Bobby P. C. Koeleman |
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Format: | article |
Language: | EN |
Published: |
MDPI AG
2021
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Online Access: | https://doaj.org/article/36cb1abb0d714d319b1a54ec5f25a48f |
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