Characterization and AAV-mediated CRB gene augmentation in human-derived CRB1KO and CRB1KOCRB2+/- retinal organoids.

Boon, Nanda; Lu, Xuefei; Andriessen, Charlotte A; Orlovà, Michaela; Quinn, Peter M J; Boon, Camiel J F; Wijnholds, Jan

Characterization and AAV-mediated CRB gene augmentation in human-derived CRB1^KO and CRB1^KOCRB2^+/- retinal organoids.

Boon, Nanda; Lu, Xuefei; Andriessen, Charlotte A; Orlovà, Michaela; Quinn, Peter M J; Boon, Camiel J F; Wijnholds, Jan.

Afiliação

Boon N; Department of Ophthalmology, Leiden University Medical Center (LUMC), Albinusdreef 2, 2333 ZA Leiden, the Netherlands.
Lu X; Department of Ophthalmology, Leiden University Medical Center (LUMC), Albinusdreef 2, 2333 ZA Leiden, the Netherlands.
Andriessen CA; Department of Ophthalmology, Leiden University Medical Center (LUMC), Albinusdreef 2, 2333 ZA Leiden, the Netherlands.
Orlovà M; Department of Ophthalmology, Leiden University Medical Center (LUMC), Albinusdreef 2, 2333 ZA Leiden, the Netherlands.
Quinn PMJ; Department of Ophthalmology, Leiden University Medical Center (LUMC), Albinusdreef 2, 2333 ZA Leiden, the Netherlands.
Boon CJF; Department of Ophthalmology, Leiden University Medical Center (LUMC), Albinusdreef 2, 2333 ZA Leiden, the Netherlands.
Wijnholds J; Department of Ophthalmology, Amsterdam University Medical Centers, 1000 AE Amsterdam, the Netherlands.

Mol Ther Methods Clin Dev ; 31: 101128, 2023 Dec 14.

Article em En | MEDLINE | ID: mdl-37886604

Palavras-chave

AAV; CRB1; CRB2; CRISPR-Cas9; Müller glial cells; gene therapy; hiPSC; photoreceptor; retinal degeneration; retinal organoids

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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Idioma: En Revista: Mol Ther Methods Clin Dev Ano de publicação: 2023 Tipo de documento: Article País de afiliação: Holanda País de publicação: Estados Unidos

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