Human stem cell-based retina on chip as new translational model for validation of AAV retinal gene therapy vectors.

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 analy

SUBMITTER: Achberger K 

PROVIDER: S-EPMC8452599 | biostudies-literature | 2021 Sep

REPOSITORIES: biostudies-literature