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AAV-mediated FOXG1 gene editing in human Rett primary cells
Susanna Croci1, Miriam Lucia Carriero1, Katia Capitani1,2
1Medical Genetics, University of Siena, Siena, Italy.
Researchers used CRISPR/Cas9 gene editing delivered by adeno-associated viruses (AAVs) to correct mutations in the Forkhead Box G1 (FOXG1) gene. This approach shows promise for developing a therapy for FOXG1 syndrome and Rett syndrome.
Area of Science:
- Genetics and Genomics
- Gene Therapy
- Neuroscience
Background:
- Forkhead Box G1 (FOXG1) gene variations cause a spectrum of disorders, including a congenital variant of Rett syndrome.
- Current treatments for FOXG1 syndrome are limited due to poorly understood disease mechanisms.
- Both reduced and increased FOXG1 gene expression lead to human diseases, necessitating precise gene correction.
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