Characterization and AAV-mediated <i>CRB</i> gene augmentation in human-derived <i>CRB1</i>^KO and <i>CRB1</i>^KO<i>CRB2</i>^+/- retinal organoids.

"Declaration of interests The authors declare no competing interests. The LUMC is the holder of patent number PCT/NL2014/050549, which describes the potential clinical use of CRB2. J.W. is listed as co-inventor of this patent and is an employee of the LUMC. Horama (currently known as Coave Therapeutics) partially sponsored this study."

"The authors would like to thank Chelsey Linnenbank for technical assistance, the LUMC light and electron microscope facility for help during image acquisition, and all the previous and current members of the Wijnholds lab for advice on experiments and the manuscript. This work was funded by The Netherlands Organization for Health Research and Development (ZonMw grant 43200004, to J.W.), research grant MDBR-19-131-CRB1 from the University of Pennsylvania Orphan Disease Center in partnership with the 10.13039/100012711Curing Retinal Blindness Foundation (to J.W.), 10.13039/501100005040Bontius Stichting 32072-8231 (to J.W.), Leiden Regenerative Medicine Platform 2018-CRB1 (to J.W.), and HORAMA 41400-8231 (to J.W.). Author contributionsConceptualization, N.B. and J.W.; Formal analysis, N.B.; Investigation, N.B., X.L., C.A.A, M.O, and P.M.J.Q.; Data curation, N.B. and X.L.; Writing - original draft, N.B.; Writing - review & editing, all authors; Visualization, N.B.; Supervision, J.W.; Project administration, J.W.; Funding acquisition, J.W."