From: Ocular gene transfer in the spotlight: implications of newspaper content for clinical communications
Diseaseb(number of articlesa) | Nature of retinopathy | Clinical trial number | Clinical trial phase | Gene targeted | Vector type | Number (n) and age of participants |
---|---|---|---|---|---|---|
Leber congential amaurosis (84) | Progressive retinopathy with severe visual problems beginning in infancy. | NCT00999609 | III | RPE65 | Adeno-associated viral vector; AAV2-hRPE65v2 | n = 24; 3 years and older |
NCT00749957 | I/II | RPE65 | Recombinant adeno-associated viral vector; rAAV2-CB-hRPE65 | n = 12; 6 years and older | ||
NCT01208389 | I/II | RPE65 | Adeno-associated viral vector; AAV2-hRPE65v2 | n = 12; 8 years and older | ||
NCT00643747 | I/II | RPE65 | Recombinant adeno-associated viral vector; rAAV 2/2.hRPE65p.hRPE65 | n = 12; 5-30 years old | ||
NCT01496040 | I/II | RPE65 | Recombinant adeno-associated viral vector; rAAV-2/4.hRPE65 | n = 9; 6-50 years old | ||
NCT00481546 | I | RPE65 | Recombinant adeno-associated viral vector; rAAV2-CBSB-hRPE65 | n = 15; 8 years and older | ||
NCT00821340 | I | RPE65 | Recombinant adeno-associated viral vector; rAAV2-hRPE65 | n = 10; 8 years and older | ||
NCT00516477 | I | RPE65 | Adeno-associated viral vector; AAV2-hRPE65v2 | n = 12; 8 years and older | ||
Retinitis pigmentosa (37) | Progressive retinopathy characterized by gradual peripheral vision loss | NCT01482195 | I | MERTK | Recombinant adeno-associated viral vector; rAAV2-VMD2-hMERTK | n = 6; 14-70 years old |
Age-related macular degeneration (24) | Progressive central vision loss usually occurring in older adults | NCT01301443 | I | Genes that encode endostatin and angiostatin | Lentiviral vector; RetinoStat | n = 18; 50 years and older |
NCT01678872 | I | Genes that encode endostatin and angiostatin | Lentiviral vector; RetinoStat | n = 18; 50 years and older | ||
Choroideremia (7) | Progressive retinopathy characterized by gradual peripheral vision loss | NCT01461213 | I/II | REP1 | Adeno-associated viral vector; AAV.REP1 | n = 12; 18 years and older |
Stargardt macular degeneration (7) | Progressive degeneration of the macula causing central vision loss beginning in childhood | NCT01367444 | I/IIa | ABCA4 | Lentiviral vector; StarGen | n = 28; 18 years and older |
Usher syndrome (7) | Progressive retinopathy (retinitis pigmentosa) combined with hearing loss | NCT01505062 | I/IIa | MYO7A | Lentiviral vector; UshStat | n = 18; 18 years and older |