AAV |
Adeno-associated virus |
ADRP |
Autosomal dominant Retinitis Pigmentosa |
CEP290 |
Ciliopathy gene centrosomal protein 290 |
CHM |
Choroideremia |
CRISPR |
Clustered regularly interspaced short palindromic repeats |
DSB |
Double strand break |
GPCR |
G-protein coupled receptor |
gRNA |
Guide RNA |
HDR |
Homology-directed repair |
IRBP |
Interphotoreceptor retinoid-binding protein |
IRDs |
Inherited Retinal Dystrophies |
IVT |
Intravitreal injection |
LCA |
Leber’s Congenital Amaurosis |
LRAT |
Lecithin retinol acyltransferase |
MMEJ |
Microhomology-mediated end joining |
NHEJ |
Non-homologous end joining |
Nrl |
Neural retina-specific leucine zipper protein |
RHO |
Rhodopsin |
RP |
Retinitis Pigmentosa |
RPE |
Retinal pigment epithelium |
rAAV |
Recombinant Adeno-associated virus |
O.S. |
Outer segments |
PAM |
Protospacer adjacent motif |
XLRs |
X-linked juvenile retinoschisis |