Review Article

Progress in Gene Therapy to Prevent Retinal Ganglion Cell Loss in Glaucoma and Leber’s Hereditary Optic Neuropathy

Table 1

Promoters typically chosen for transduction of retinal ganglion cells (RGCs) within the eye.

PromoterSpecificity for RGCsStrength of expression in RGCsOff-target labellingSize (bp)
% of AAV cargo
References

CMV++++Muller glia
Amacrine cells
Bipolar cells
(508–800)
(10–17%)
[1014]

CAG+++++Muller glia
Amacrine cells
Bipolar cells
(584–1132)
(12–24%)
[1517]

SYN1+++++Amacrine cells(400–469)
(8–10%)
[20, 21]

Nefh+++++++(2251)
(48%)
[22]

Thy1++++++(6500)
(exceeds limit)
[23]

Mcp-1+++++injured cells560 (mouse only)
(12%)
[24]