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...
Enregistré dans:
| Auteurs principaux: | , , |
|---|---|
| Format: | article |
| Langue: | EN |
| Publié: |
MDPI AG
2021
|
| Sujets: | |
| Accès en ligne: | https://doaj.org/article/36cb1abb0d714d319b1a54ec5f25a48f |
| Tags: |
Ajouter un tag
Pas de tags, Soyez le premier à ajouter un tag!
|
| Résumé: | 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 models, and reviewed current gene therapies for each syndrome. Given the wide variety and novelty of some forms of gene therapy, the best possible option must be decided based on the gene and mutation. |
|---|