Table 1.
Current clinical trials in retinal gene therapy
| Disease | Encoded gene | Protein | Cell target | Vector | Clinical trial number |
|---|---|---|---|---|---|
| LCA2 | RPE65 | Retinal isomerase RPE65 | RPE | AAV | NCT00516477 |
| NCT00749957 | |||||
| NCT00821340 | |||||
| NCT01208389 | |||||
| NCT00999609 | |||||
| NCT00481546 | |||||
| NCT00643747 | |||||
| Choroideremia | CHM1 | REP1 | RPE + choroid | AAV | NCT02077361 |
| NCT01461213 | |||||
| NCT02341807 | |||||
| NCT02553135 | |||||
| NCT02407678 | |||||
| Usher syndrome | MYO7A | Myosin 7a | PR | Lentivirus | NCT01505062 |
| NCT02065011 | |||||
| Stargardt disease | ABCA4 | Photoreceptor-specific adenosine triphosphate-binding cassette transporter | PR | Lentivirus | NCT01367444 |
| NCT01736592 | |||||
| LHON | ND4 | NADH dehydrogenase | RGC | AAV | NCT01267422 |
| NCT02064569 | |||||
| NCT02161380 | |||||
| arRP | MERTK | MER tyrosine kinase receptor | RPE | AAV | NCT01482195 |
| Retinoschisis | RS1 | Retinoschisin | PR | AAV | NCT02317887 |
| NCT02416622 | |||||
| AMD | FLT1 | Soluble FMS-like tyrosine kinase receptor | Soluble | AAV |
NCT01494805 NCT01024998 |
| Endostatin and angiostatin | Lentivirus | NCT01301443 | |||
| PLG and COL18A1 | Angiostatin and endostatin | Soluble | Lentivirus |
NCT01301443 NCT01678872 |
AAV, adeno-associated virus; AMD, age-related macular degeneration; arRP, autosomal recessive retinitis pigmentosa; LCA2, Leber congenital amaurosis type 2; LHON, Leber hereditary optic neuropathy; PR, photoreceptor; REP1, Rab escort protein 1; RGC, retinal ganglion cell; RPE, retinal pigment epithelium.