Article

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

2021-03-02

Abstract excerpt

<h4>Summary</h4> Gene therapies using adeno-associated viruses (AAVs) are amongst 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 (iP...

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Literature Corpus work: b9f12b70-d17d-5a2e-83b8-fe4a561eb40d
DOI: 10.1101/2021.03.02.433550

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