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. 2025 Nov 19;15(11):1624. doi: 10.3390/biom15111624
adRP Autosomal Dominant Retinitis Pigmentosa.
arRP Autosomal Recessive Retinitis Pigmentosa.
ASOs Antisense Oligonucleotides.
AAV Adeno-Associated Virus.
ABCA4  ATP-binding cassette transporter A4.
ABE adenine base editor.
BPS Branch point sequence.
Brr2 Bad Response to Refrigeration 2 (also known as SNRNP200).
BBS2 Bardet–Biedl syndrome 2.
PSC Posterior Subcapsular Cataract.
CME Cystoid Macular Edema.
CNOT3 CCR4-NOT transcription complex subunit 3.
CCR4-NOT carbon catabolite repression 4-negative on TATA-less.
CRISPR/Cas9 Clustered Regularly Interspaced Short Palindromic Repeats/CRISPR-Associated Protein 9.
CWC22 complexed with CEF1 protein 22.
CWC27 complexed with CEF1 protein 27.
CLRN1 Clarin 1.
CEP290 centrosomal protein 290.
CBE cytosine base editor.
DExD/H-box aspartate-glutamate-X-aspartate/histidine box.
DHX38 DEAH-box helicase 38.
di-snRNP di-small nuclear ribonucleoprotein.
ESEs exonic splicing enhancers.
ESSs  exonic splicing silencers.
eQTL expression quantitative trait locus.
EFTUD2 elongation factor Tu GTP-binding domain containing 2.
eIF4A3 eukaryotic initiation factor 4A3.
EJC exon junction complex.
ER endoplasmic reticulum.
ERG  electroretinography.
eDCas9 enhanced dead Cas9.
eSpCas9 enhanced specificity Cas9.
FSCN2 fascin actin-bundling protein 2.
FDA  Food and Drug Administration.
GCL Ganglion cell layer.
GNAT1 guanine nucleotide-binding protein alpha transducing 1.
GNAT2 guanine nucleotide-binding protein alpha transducing 2.
GPKOW G-patch domain and KOW motifs.
GEF guanine nucleotide exchange factor.
hnRNPs heterogeneous nuclear ribonucleoprotein particles.
ROs  retinal organoids.
HSP70 heat shock protein 70.
hSnu114 human small nuclear U114 (alternative name for EFTUD2).
HBB hemoglobin subunit beta.
HDR homology-directed repair.
IPL Inner plexiform layer.
INL Inner nuclear layer.
ISSs  intronic splicing silencers.
IFT88  intraflagellar transport protein 88.
IFT122 intraflagellar transport protein 122.
iPSC induced pluripotent stem cell.
Jab1/MPN Jun activation domain-binding protein 1/Mpr1-Pad1 N-terminal.
LSm like-Sm proteins.
LCA Leber congenital amaurosis.
mRNA messenger RNA.
MSR1 macrophage scavenger receptor 1.
MOs morpholino oligonucleotides.
NHEJ non-homologous end joining.
OPL Outer plexiform layer.
ONL Outer nuclear layer.
OMCS Oliver–McFarlane Syndrome.
PHARC Polyneuropathy, Hearing loss, Ataxia, Retinitis pigmentosa, and Cataract syndrome.
PCARP Photoreceptor Ciliopathy And Retinal Pigmentation.
PPT Polypyrimidine tract.
pre-mRNA precursor messenger RNA.
PTBP1 polypyrimidine tract-binding protein 1.
PRPF3 pre-mRNA processing factor 3.
PRPF4 pre-mRNA processing factor 4.
PRPF6 pre-mRNA processing factor 6.
PRPF8 pre-mRNA processing factor 8.
PRPF31 pre-mRNA processing factor 31.
PAP-1 polyadenylation specificity factor 1.
PPIase peptidyl-prolyl cis-trans isomerase.
PDE6B Phosphodiesterase 6B.
PTMs pre-mRNA trans-splicing molecules.
RPE Retinal pigment epithelium.
RP Retinitis Pigmentosa.
RBM20 RNA-binding motif protein 20.
RPGR retinitis pigmentosa GTPase regulator.
RPCs retinal progenitor cells.
RHO rhodopsin.
RPGRIP1 retinitis pigmentosa GTPase regulator interacting protein 1.
RPE65 Retinal Pigment Epithelium 65.
rAAV recombinant adeno-associated virus.
RAB37 Ras-related protein 37.
REVeRT mRNA trans-splicing recombinant technology.
PPIH peptidyl prolyl isomerase H.
Sm Smith antigens.
snRNA small nuclear RNA.
snRNPs small nuclear ribonucleoprotein complexes.
SF1 Splicing Factor 1.
SR serine/arginine-rich.
SNRNP200 small nuclear ribonucleoprotein 200.
siRNA small interfering RNA.
SC35 serine/arginine-rich splicing factor 2.
SaCas9  Staphylococcus aureus Cas9.
sgRNA single guide RNA.
SMaRT spliceosome-mediated RNA trans-splicing.
tri-snRNP U4/U6.U5 tri-snRNP complex.
TPR tetratricopeptide repeat.
TTLL3 tubulin tyrosine ligase like 3.
U snRNAs uridine-rich small nuclear RNAs.
U2AF1 U2 Auxiliary Factor 35 kDa subunit.
U2AF2  U2 Auxiliary Factor 65 kDa subunit.
ULK1 unc-51 like autophagy activating kinase 1.
USH2A Usherin 2A.
U1_asRNA U1 antisense small nuclear RNA.
WD40  WD repeat domain 40.
XLRP X-Linked Retinitis Pigmentosa.
5′ ss 5′ splice site.
3′ ss 3′ splice site.