AAV	Adeno-associated virus
AEs	Adverse effects
ARL3	ADP-ribosylation factor-like GTPase 3
CME	Cystoid macular edema
dB	Decibels
GAP	GTPase-activating protein
GEF	Guanine nucleotide exchange factor
GRK1	G protein-coupled receptor kinase 1
IRBP	Interphotoreceptor retinoid-binding protein
iPSCs	Induced pluripotent stem cells
KO	Knockout
LCA	Leber congenital amaurosis
MAIA	Macular integrity assessment
nAbs	Neutralizing antibodies
ONL	Outer nuclear layer
PAM	Protospacer adjacent motif
PDE δ	Phosphodiesterase δ
RGCs	Retinal ganglion cells
rAAV	Recombinant AAV
ROS	Retinal organoids
RPE	Retinal pigment epithelium
RP	Retinitis pigmentosa
RPGR	Retinitis pigmentosa GTPase regulator
ROS	Reactive oxygen species
sgRNA	Single guide RNA
TEAEs	Treatment-emergent adverse events
TLRs	Toll-like receptors
UNC119	Uncoordinated 119 protein
VF	Visual field
VEGF	Vascular endothelial growth factor
XLRP	X-linked retinitis pigmentosa