Retina on a Chip
How combining stem cells with organ-on-chip technology presents a new translational model for gene therapy research
Biotechnology is ever evolving, with breakthroughs enabling researchers to more efficiently translate data produced in the lab to human need. The advent of the organ-on-chip (OOC) technology represents one such breakthrough – and now researchers at Eberhard Karls University Tübingen, Germany, have combined OOC and human induced pluripotent stem cells (iPSCs) to develop a retina-on-a-chip model to help determine the effectiveness of gene therapy vectors (1).
The group grows human iPSCs into 3D retinal organoids – containing all the cells of the retina in a mini-organ. These are contained within the chambers of a fluidic device (the chip), which allows imaging and feeding of the tissue. The researchers used the model to analyze the function and potency of adeno-associated virus (AAV) vectors – with correlating in vivo data, showing the translational effectiveness of their model. The team hope the retina-on-a-chip model will be used for high-throughput screening.
Human iPSCs can be generated from skin or hair cells, which are reprogrammed to a pluripotent state, enabling further differentiation into many different cell types. By using iPSCs, personalized OOC models are possible, which allows patient specific responses to gene therapies – or any therapeutic or potentially toxic agent – to be recorded.
- K Achberger et al., “Human stem cell-based retina on chip as new translational model for validation of AAV retinal gene therapy vectors,” Stem Cell Reports, 16, 2242 (2021). PMID: 34525384.
The lion’s share of my PhD was spent in the lab, and though I mostly enjoyed it (mostly), what I particularly liked was the opportunity to learn about the latest breakthroughs in research. Communicating science to a wider audience allows me to scratch that itch without working all week only to find my stem cell culture has given up the ghost on the Friday (I’m not bitter). Fortunately for me, it turns out writing is actually fun – so by working for Texere I get to do it every day, whilst still being an active member of the clinical and research community.