Table 1. Currently active clinical trials for inherited retinal dystrophies and age-related macular degeneration.
| Active Clinical Trials | ||||||||
|---|---|---|---|---|---|---|---|---|
| Disease | Gene | Coded protein | Expression | Frequency | Vector platform | Delivery | Locations | ClinicalTrials.gov ID |
| LCA2 | RPE65 | RPE-specific 65kDa protein | RPE | 1:1,000,000 | AAV2/2 | SR | USA, UK, Israel | NCT00999609; NCT00749957; NCT01208389; NCT00516477; NCT00643747; NCT00481546; NCT00821340 |
| Choroideremia | CHM | Rab Escort Protein 1 (REP-1) | ubiquitous | 1:50,000 | AAV2/2 | SR | UK, Canada | NCT01461213; NCT02077361 |
| Wet AMD | FLT1 | FMS-like tyrosine kinase 1 | soluble | 1:2,000 | AAV2/2 | SR or IVT | Australia, USA | NCT01494805; NCT01024998 |
| PLG and COL18A1 | Angiostatin and endostatin | soluble | 1:2,000 | EAIV lentivirusr | SR | USA | NCT01301443;NCT01678872 | |
| USH1B | MYO7A | Myosin VIIa | PR | 1:60,000 | EAIV lentivirus | SR | USA, France | NCT01505062; NCT02065011 |
| Stargardt | ABCA4 | PR-specific adenosine triphosphate-binding cassette transporter | PR | 1:8-10,000 | EAIV lentivirus | SR | USA, France | NCT01367444; NCT01736592 |
| Leber hereditary optic neuropathy | ND4 | NADH dehydrogenase | RGC | 1:30-50,000 | AAV2/2 | SR | China, France | NCT01267422; NCT02064569 |
| ArRP | MERTK | Mer receptor tyrosine kinase | RPE | Very rare | AAV2/2 | SR | Saudi Arabia | NCT01482195 |
Abbreviations: LCA, Leber Congenital Amarousis; USH1B, Usher syndrome type 1b; ArRP, Autosomal recessive Retinitis Pigmentosa;; RPE, Retinal Pigment Epithelium, PR, Photoreceptors; RGC, Retinal Ganglion Cells; SR, Subretinal; IVT, Intravitreal