Gene Therapy of Dominant CRX-Leber Congenital Amaurosis using Patient Stem Cell-Derived Retinal Organoids

Mutations in the photoreceptor transcription factor gene cone-rod homeobox (CRX) lead to distinct retinopathy phenotypes, including early-onset vision impairment in dominant Leber congenital amaurosis (LCA). Using induced pluripotent stem cells (iPSCs) from a patient with CRX-I138fs48 mutation, we e...

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Detalhes bibliográficos
Publicado no:Stem Cell Reports
Main Authors: Kruczek, Kamil, Qu, Zepeng, Gentry, James, Fadl, Benjamin R., Gieser, Linn, Hiriyanna, Suja, Batz, Zachary, Samant, Mugdha, Samanta, Ananya, Chu, Colin J., Campello, Laura, Brooks, Brian P., Wu, Zhijian, Swaroop, Anand
Formato: Artigo
Idioma:Inglês
Publicado em: Elsevier 2021
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7878833/
https://ncbi.nlm.nih.gov/pubmed/33513359
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.stemcr.2020.12.018
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