Characterization and AAV-mediated CRB gene augmentation in human-derived CRB1(KO) and CRB1(KO)CRB2(+/−) retinal organoids

The majority of patients with mutations in CRB1 develop either early-onset retinitis pigmentosa as young children or Leber congenital amaurosis as newborns. The cause for the phenotypic variability in CRB1-associated retinopathies is unknown, but might be linked to differences in CRB1 and CRB2 prote...

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Detalles Bibliográficos
Autores principales: Boon, Nanda, Lu, Xuefei, Andriessen, Charlotte A., Orlovà, Michaela, Quinn, Peter M.J., Boon, Camiel J.F., Wijnholds, Jan
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2023
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10597801/
https://www.ncbi.nlm.nih.gov/pubmed/37886604
http://dx.doi.org/10.1016/j.omtm.2023.101128

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10597801/
https://www.ncbi.nlm.nih.gov/pubmed/37886604
http://dx.doi.org/10.1016/j.omtm.2023.101128

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