Gene Therapies for Monogenic Autism Spectrum Disorders

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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Bibliographic Details
Main Authors: Wout Weuring, Jeroen Geerligs, Bobby P. C. Koeleman
Format: Article
Language:English
Published: MDPI AG 2021-10-01
Series:Genes
Subjects:
ASD
gene therapy
gene edit
RNA therapy
epilepsy
gene replacement
Online Access:https://www.mdpi.com/2073-4425/12/11/1667

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