Journal
STEM CELL REPORTS
Volume 12, Issue 5, Pages 906-919Publisher
CELL PRESS
DOI: 10.1016/j.stemcr.2019.03.002
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Funding
- Foundation Fighting Blindness USA [TA-GT-0313-0607-NIN, TA-GT-0715-0665-LUMC]
- Netherlands Organisation for Health Research and Development (ZonMw grant) [43200004]
- Curing Retinal Blindness Foundation (CRBF)
- Stichting Retina Fonds
- Landelijke Stichting voor Blinden en Slechtzienden (LSBS)
- Rotterdamse Stichting Blindenbelangen (RSB)
- Stichting Blindenhulp
- MaculaFonds
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Human retinal organoids from induced pluripotent stem cells (hiPSCs) can be used to confirm the localization of proteins in retinal cell types and to test transduction and expression patterns of gene therapy vectors. Here, we compared the onset of CRB protein expression in human fetal retina with human iPSC-derived retinal organoids. We show that CRB2 protein precedes the expression of CRB1 in the developing human retina. Our data suggest the presence of CRB1 and CRB2 in human photoreceptors and Muller glial cells. Thus the fetal CRB complex formation is replicated in hiPSC-derived retina. CRB1 patient iPSC retinal organoids showed disruptions at the outer limiting membrane as found in Crb1 mutant mice. Furthermore, AAV serotype 5 (AAV5) is potent in infecting human Muller glial cells and photoreceptors in hiPSC-derived retinas and retinal explants. Our data suggest that human photoreceptors can be efficiently transduced by AAVs in the presence of photoreceptor segments.
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