Table 1. Clinical trails: gene therapy.
| No. | Trial ID | Phase | conditons | Intervention | Gene types | Vector | PI |
| L1 | NCT00643747 | I/II-12 | LCA | rAAV 2/2.hRPE65p.hRPE65 | RPE65 | rAAV2 | Ali RR (University College, London) |
| L 2 | NCT00516477 | I-9 | LCA | AAV2-hRPE65v2-101 | RPE65 | rAAV2 | Maguire AM (Children's Hospital of Philadelphia) |
| L 3 | NCT00481546 | I-9 | LCA | rAAV2-CBSB-hRPE65 | RPE65 | rAAV2 | Jacobson SJ (University of Pennsylvania) |
| L 4 | NCT00749957 | I/II-12 | LCA | rAAV2-CB-hRPE65 | RPE65 | rAAV2 | Stout JT (Applied Genetic Technologies Corp) |
| L 5 | NCT00821340 | I-10 | LCA | rAAV2-hRPE65 | RPE65 | rAAV2 | Banin E (Hadassah Medical Organization) |
| L 6 | NCT00999609 | III | LCA | rAAV2-hRPE65 | RPE65 | rAAV2 | Maguire AM (Children's Hospital of Philadelphia) |
| L 7 | NCT01208389 | I/II | LCA | AAV2-hRPE65v2 | RPE65 | rAAV2 | Maguire AM (Children's Hospital of Philadelphia) |
| L 8 | NCT01496040 | I/II | LCA | rAAV2/4.hRPE65 | RPE65 | rAAV2 | Michel WEBER (Nantes University Hospital) |
| A1 | NCT01024998 | I | AMD | AAV2-sFLT01 | sFlt-1 | AAV2 | Genzyme, a Sanofi Company |
| A2 | NCT01494805 | I/II | AMD | rAAV.sFlt-1 | sFlt-1 | rAAV | Ian Constable, (Lions Eye Institute) |
| A3 | NCT01301443 | I | AMD | AAV2-sFLT01 | sFlt-1 | AAV2 | Peter Campochiaro, (Johns Hopkins University Hospital) |
| A4 | NCT01367444 | I/II | AMD/Stargardt | StarGen | ABCA4 | EIAV | Oxford BioMedica. Inc |
| A5 | NCT00109499 | I | AMD | AdGVPEDF.11D | PEDF | replication deficient adenovirus | GenVec. Inc |
| A6 | US-1061 | I | AMD | Endostatin Angiostatin | Endostatin Angiostatin | Lentivirus | Peter Campochiaro, (Johns Hopkins University Hospital) |
| A7-8 | US-X001/X002 | I | AMD | Cand5 (siRNA against VEGF) | siRNA | siRNA | Acuity PharmaceuticalsUSA |
| A9-10 | US-X007 | II | AMD | siRNA-027(siRNA against VEGF R1) | siRNA-027 | siRNA | Allergen PharmaceuticalsUSA |
| R1 | NCT01482195 | I | RP | rAAV2-VMD2-hMERTK | MERTK | rAAV | Fowzan S Alkuraya, (King Faisal Specialist Hospital) |
| R2 | NCT01505062 | I/II | RP/Usher | UshStat | MYO7A | EIAV | Oxford BioMedica. Inc |
| R3-5 | US-575/US-795/US-796 | II | RP | CNTF | Cytokine | Naked/Plasmid DNA | Sieving PA (National Institutes of Health USA) |
| 1 | US-539 | I/II | Corneal Scarring | dnG1 Cyclin | Cell cycle | Retrovirus | Song JC (Keck School of Medicine,University of Southern California,USA) |
| 2 | US-589 | I | Glaucoma | p21 WAF-1/Cip1 | Cell cycle | Adenovirus | Kaufman PL (University of Wisconsin-Madison Medical School Madison, USA) |
| 3 | NCT01461213 | I/II | Choroideremia | rAAV2.REP1 | REP1 | rAAV2 | Robert E MacLaren, (University of Oxford) |
| 4 | US-X003 | II | DME | Bevasiranib/Cand5 | siRNA | siRNA | Acuity Pharmac-euticals USA |
| 5 | CN-0025 | I/II | LHON | rAAV2-ND4 | ND4 | rAAV2 | Lin B, (Tongji Medical college) |
LCA: Leber congenital amaurosis; AMD: Age-related macular degeneration; RP: Retinitis pigmentosa; DME: Diabetic Macular Edema; LHON: Leber hereditary optic neuropathy; (r)AAV: (recombined) Adeno-associated virus vector; sFlt-1: Soluble vascular endothelial growth factor receptor 1; AdGVPEDF.11D: Replication deficient (E1, E3 and E4 deleted) adenovirus vector containing the gene for the pigment epithelium-derived factor (PEDF) protein; VEGF: Vascular endothelial growth factor; siRNA: short-interfering RNA; CNTF: Ciliary neurotrophic factor; dnG1: Matrix-targeted retroviral vector bearing a dominant negative cyclin G1 construct; REP1: Rab-escort protein 1; ND4: NADH dehydrogenase subunit 4; EIAV: Equine infectious anemia virus.