Human stem cell-based retina on chip as new translational model for validation of AAV retinal gene therapy vectors.
Stem Cell Reports
; 16(9): 2242-2256, 2021 09 14.
Article
em En
| MEDLINE
| ID: mdl-34525384
ABSTRACT
Gene therapies using adeno-associated viruses (AAVs) are among the most promising strategies to treat or even cure hereditary and acquired retinal diseases. However, the development of new efficient AAV vectors is slow and costly, largely because of the lack of suitable non-clinical models. By faithfully recreating structure and function of human tissues, human induced pluripotent stem cell (iPSC)-derived retinal organoids could become an essential part of the test cascade addressing translational aspects. Organ-on-chip (OoC) technology further provides the capability to recapitulate microphysiological tissue environments as well as a precise control over structural and temporal parameters. By employing our recently developed retina on chip that merges organoid and OoC technology, we analyzed the efficacy, kinetics, and cell tropism of seven first- and second-generation AAV vectors. The presented data demonstrate the potential of iPSC-based OoC models as the next generation of screening platforms for future gene therapeutic studies.
Palavras-chave
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Retina
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Transdução Genética
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Organoides
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Dependovirus
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Células-Tronco Pluripotentes Induzidas
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Dispositivos Lab-On-A-Chip
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Vetores Genéticos
Limite:
Humans
Idioma:
En
Revista:
Stem Cell Reports
Ano de publicação:
2021
Tipo de documento:
Article
País de afiliação:
Alemanha