CRISPR/Cas9-generated mouse model with humanizing single-base substitution in the Gnao1 for safety studies of RNA therapeutics

The development of personalized medicine for genetic diseases requires preclinical testing in the appropriate animal models. GNAO1 encephalopathy is a severe neurodevelopmental disorder caused by heterozygous de novo mutations in the GNAO1 gene. GNAO1 c.607 G>A is one of the most common pathogeni...

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Detalles Bibliográficos
Autores principales: Polikarpova, Anna V., Egorova, Tatiana V., Lunev, Evgenii A., Tsitrina, Alexandra A., Vassilieva, Svetlana G., Savchenko, Irina M., Silaeva, Yuliya Y., Deykin, Alexey V., Bardina, Maryana V.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2023
Materias:
Genome Editing
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10106585/
https://www.ncbi.nlm.nih.gov/pubmed/37077890
http://dx.doi.org/10.3389/fgeed.2023.1034720

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