Affiliations

Scheie Eye Institute, Department of Ophthalmology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA Artur V. Cideciyan, Samuel G. Jacobson, Jason Charng, Alexandra V. Garafalo, Alejandro J. Roman & Alexander Sumaroka Department of Ophthalmology and Visual Sciences, Carver College of Medicine, University of Iowa, Iowa City, IA, USA Arlene V. Drack, Ian C. Han, Maria D. Hochstedler, Wanda L. Pfeifer, Elliott H. Sohn & Stephen R. Russell Wills Eye Hospital, Thomas Jefferson University, Philadelphia, PA, USA Allen C. Ho ProQR Therapeutics, Leiden, the Netherlands Magali Taiel, Michael R. Schwartz, Patricia Biasutto, Wilma de Wit, Peter Adamson, David M. Rodman, Gerard Platenburg & Maria D. Tome UCL Institute of Ophthalmology, London, UK Michael E. Cheetham & Peter Adamson Department of Ophthalmology, Ghent University and Ghent University Hospital, Ghent, Belgium Irina Balikova, Fanny Nerinckx, Julie De Zaeytijd, Caroline Van Cauwenbergh & Bart P. Leroy

Contributions

A.V.C. and S.G.J. contributed to the clinical study design and protocol development; performed clinical investigation of patients; reviewed, analyzed, and interpreted the data; and wrote the draft manuscript and its revisions. M.T., M.R.S., P.B., W.d.W., P.A., D.M.R., G.P., and M.D.T. developed the clinical study protocol, reviewed the data, and contributed to all drafts of the manuscript. A.V.D., B.P.L., and S.R.R. performed the clinical investigation of patients, contributed to the clinical study design and protocol development, and contributed to all drafts of the manuscript. A.C.H., F.N., and S.R.R. performed the injections. J.C., A.V.G., A.J.R., A.S., I.C.H., M.D.H., W.L.P., E.H.S., I.B., J.D.Z., and C.V.C. supported clinical investigation of the patients. A.J.R. performed the statistical analyses. P.B., P.A., and M.E.C. performed in vitro experiments determining clinical dosing strategy.

Corresponding authors

Correspondence to Artur V. Cideciyan or Samuel G. Jacobson.