Table 1.

Registered Clinical Trials of Gene Therapy for Retinal Diseases

Disease (Gene)	ClinicalTrials.gov Identifier	Vector Name	Locations	Key Publications
LCA2 (RPE65)	NCT00481546	rAAV2-CBSB-hRPE65	University of Pennsylvania University of Florida Health Shands Hospital	Bainbridge et al., 20086 Maguire et al., 20087 Cideciyan et al., 20088 Hauswirth et al., 20089 Simonelli et al., 201010 Cideciyan et al., 201311
	NCT00516477 NCT00999609 NCT01208389	AAV2-hRPE65v2 AAV2-hRPE65v2 AAV2-hRPE65v2	Children's Hospital of Philadelphia, University of Iowa
	NCT00643747	rAAV 2/2.hRPE65p.hRPE65	Moorfields Eye Hospital
	NCT00749957	rAAV2-CB-hRPE65	Oregon Health and Science University University of Massachusetts-Worcester
	NCT00821340	rAAV2-hRPE65	Haddasah Medical Organization
	NCT01496040	rAAV2/4.hRPE65	Nantes Hospital
RP38 (MERTK)	NCT01482195	rAAV2-VMD2-hMERTK	King Khaled Eye Specialist Hospital	Deng et al., 201212 Conlon et al., 201313
Choroideremia (CHM)	NCT01461213 NCT02077361	rAAV2.REP1	Moorfields Eye Hospital University of Alberta	Vasireddy et al., 201314 MacLaren et al., 201415
LHON (ND4)	NCT01267422	rAAV2-ND4	Tongji Hospital, Huazhong University of Science and Technology	Marella et al., 201016
				Yu et al., 201217
	NCT02064569	rAAV2/2-ND4 – GS010	Centre Hospitalier Nationale d'Ophthalmologie des Quinze-Vingts	Chadderton et al., 201318
	NCT02161380	scAAV2-P1ND4v2	Bascom-Palmer Eye Institute	Lam et al., 201419
Stargardt Disease (ABCA4)	NCT01367444 NCT01736592	StarGen (EIAV-ABCA4)	Oregon Health and Science University Centre Hospitalier Nationale d'Ophthalmologie des Quinze-Vingts	Kong et al., 200820 Binley et al., 201321
Usher1B (MYO7A)	NCT01505062 NCT02065011	UshStat (EIAV-MYO7A)	Oregon Health and Science University Centre Hospitalier Nationale d'Ophthalmologie des Quinze-Vingts	Hashimoto et al., 200722 Zallocchi et al., 201423