Gene correction of the CLN3 c.175G>A variant in patient‐derived induced pluripotent stem cells prevents pathological changes in retinal organoids

BACKGROUND: Mutations in CLN3 cause Batten disease, however non‐syndromic CLN3 disease, characterized by retinal‐specific degeneration, has been also described. Here, we characterized an induced pluripotent stem cell (iPSC)‐derived disease model derived from a patient with non‐syndromic CLN3‐associa...

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Detalles Bibliográficos
Autores principales: Zhang, Xiao, Zhang, Dan, Thompson, Jennifer A., Chen, Shang‐Chih, Huang, Zhiqin, Jennings, Luke, McLaren, Terri L., Lamey, Tina M., De Roach, John N., Chen, Fred K., McLenachan, Samuel
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2021
Materias:
Original Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8104174/
https://www.ncbi.nlm.nih.gov/pubmed/33497524
http://dx.doi.org/10.1002/mgg3.1601

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8104174/
https://www.ncbi.nlm.nih.gov/pubmed/33497524
http://dx.doi.org/10.1002/mgg3.1601

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