CRISPR-Cas9 correction of a nonsense mutation in <i>LCA5</i> rescues lebercilin expression and localization in human retinal organoids.

"Declaration of interests The authors declare that they have no conflict of interest related to the content of this manuscript."

"This work was supported by ZonMW 10.13039/100000205TOP grant (grant number 9121 6051) to R.R. and R.W.J.C.; the Foundation Fighting Blindness USA (grant numbers PPA-0517-0717-RAD to R.W.J.C. and A.G. and FFB-PPA-1719-RAD to R.R., M.E.C., and D.A.); Landelijke Stichting voor Blinden en Slechtzienden to R.R. and R.W.J.C.; Stichting Retina Nederland Fonds to R.R. and R.W.J.C.; Stichting Beheer Het Schild to R.R. and R.W.J.C.; Stichting Blinden-Penning to R.R. and R.W.J.C.; 10.13039/100016063Stichting Steunfonds Uitzicht (Uitzicht grant numbers 2016/2017-22, 2019-17) to R.R. and R.W.J.C.; Gelderse Blindenstichting to R.R. and R.W.J.C.; Rotterdamse Stichting Blindenbelangen to R.R. and R.W.J.C.; Stichting tot Verbetering van het Lot der Blinden to R.R. and R.W.J.C.; Stichting voor gehandicapte kinderen Dowilvo to R.R. and R.W.J.C.; and the Proefdiervrij project support to T.A.V.A. The Wellcome Trust (205041) and Fight for Sight to M.E.C. 10.13039/501100017645Moorfields Eye Charity and MRC to M.E.C. and J.v.d.S. The funding organizations had no role in the design or conduct of this review. They provided unrestricted grants. The unrelated control iPSC cell line was a kind gift from Dr. Brüstle."