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
Acceso en línea:https://doaj.org/article/36cb1abb0d714d319b1a54ec5f25a48f
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