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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...
| Autores principales: | , , , , , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
American Society of Gene & Cell Therapy
2023
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| Materias: | |
| 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 |