期刊
CELLULAR AND MOLECULAR LIFE SCIENCES
卷 78, 期 19-20, 页码 6505-6532出版社
SPRINGER BASEL AG
DOI: 10.1007/s00018-021-03917-4
关键词
Retina; Organoid; Inherited; Omics; Degeneration; Therapy
资金
- ZonMW [9121 6051]
- Foundation Fighting Blindness USA [FFB-PPA-0717RAD, FFB-PPA-1719-RAD]
- Proefdiervrij project
- Stichting Steunfonds Uitzicht
- Gelderse Blindenstichting
- Stichting Oogfonds
- Landelijke Stichting voor Blinden en Slechtzienden
- Stichting Retina Nederland Fonds
- Stichting Beheer Het Schild
- Stichting Blinden-Penning
- Stichting tot Verbetering van het Lot der Blinden 'Het Lot'
- Stichting voor gehandicapte kinderen Dowilvo
- Stichting Blindenhulp
- Dowilwo
- Fight for Sight UK
- Wellcome Trust
- Rotterdamse Stichting Blindenbelangen
In the era of personalized medicine, induced pluripotent stem cells (iPSCs) have emerged as a valuable system for cell replacement and modeling of inherited retinal diseases (IRDs). Three-dimensional iPSCs-derived retinal organoids containing various retinal cell types show promise for advancing research and treatments for IRDs.
Inherited retinal diseases (IRDs) cause progressive loss of light-sensitive photoreceptors in the eye and can lead to blindness. Gene-based therapies for IRDs have shown remarkable progress in the past decade, but the vast majority of forms remain untreatable. In the era of personalised medicine, induced pluripotent stem cells (iPSCs) emerge as a valuable system for cell replacement and to model IRD because they retain the specific patient genome and can differentiate into any adult cell type. Three-dimensional (3D) iPSCs-derived retina-like tissue called retinal organoid contains all major retina-specific cell types: amacrine, bipolar, horizontal, retinal ganglion cells, Muller glia, as well as rod and cone photoreceptors. Here, we describe the main applications of retinal organoids and provide a comprehensive overview of the state-of-art analysis methods that apply to this model system. Finally, we will discuss the outlook for improvements that would bring the cellular model a step closer to become an established system in research and treatment development of IRDs.
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