Human stem cell-based retina on chip as new translational model for validation of AAV retinal gene therapy vectors.
Biomarkers
Cell Culture Techniques
Cell Culture Techniques, Three Dimensional
Cell Differentiation
Dependovirus
/ genetics
Fluorescent Antibody Technique
Gene Expression
Genes, Reporter
Genetic Therapy
Genetic Vectors
/ genetics
Humans
Induced Pluripotent Stem Cells
/ cytology
Lab-On-A-Chip Devices
Organoids
/ cytology
Retina
/ cytology
Transduction, Genetic
Transgenes
AAV vectors
gene therapy
human iPSC
organ-on-chip
retina on chip
retinal organoids
Journal
Stem cell reports
ISSN: 2213-6711
Titre abrégé: Stem Cell Reports
Pays: United States
ID NLM: 101611300
Informations de publication
Date de publication:
14 09 2021
14 09 2021
Historique:
received:
01
03
2021
revised:
16
08
2021
accepted:
16
08
2021
entrez:
15
9
2021
pubmed:
16
9
2021
medline:
11
3
2022
Statut:
ppublish
Résumé
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.
Identifiants
pubmed: 34525384
pii: S2213-6711(21)00426-4
doi: 10.1016/j.stemcr.2021.08.008
pmc: PMC8452599
pii:
doi:
Substances chimiques
Biomarkers
0
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Pagination
2242-2256Informations de copyright
Copyright © 2021 The Authors. Published by Elsevier Inc. All rights reserved.
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