Molecular Therapies for Inherited Retinal Diseases 

<br/><br/><br/>English[eng] 
<br/><br/><label>Subjects--Topical Terms: </label><br/>induced pluripotent stem cell (iPSC)||clustered regularly interspaced short palindromic repeats (CRISPR)||homology-directed repair (HDR)||Enhanced S-Cone Syndrome (ESCS)||NR2E3||AAV||retina||gene therapy||dual AAV||gold nanoparticles||DNA-wrapped gold nanoparticles||ARPE-19 cells||retinal pigment epithelium||clathrin-coated vesicles||endosomal trafficking||retinitis pigmentosa||autosomal dominant||G56R||putative dominant negative effect||gapmer antisense oligonucleotides||allele-specific knockdown||Leber congenital amaurosis and allied retinal ciliopathies||CEP290||Flanders founder c.4723A &gt||T nonsense mutation||Cilia elongation||spontaneous nonsense correction||AON-mediated exon skipping||microRNA||photoreceptors||rods||cones||bipolar cells||Müller glia||retinal inherited disorders||retinal degeneration||antisense oligonucleotides||Stargardt disease||inherited retinal diseases||splicing modulation||RNA therapy||ABCA4||iPSC-derived photoreceptor precursor cells||cyclic GMP||apoptosis||necrosis||drug delivery systems||translational medicine||Usher syndrome||Leber congenital amaurosis||RPE65||nonprofit||patient registry||translational||protein trafficking||protein folding||protein degradation||chaperones||chaperonins||heat shock response||unfolded protein response||autophagy||therapy||IRD||DNA therapies||RNA therapies||compound therapies||clinical trials||Retinitis Pigmentosa GTPase Regulator||adeno-associated viral||Retinitis Pigmentosa (RP)||choroideremia||REP1||inherited retinal disease||treatment||apical polarity||crumbs complex||fetal retina||PAR complex||retinal organoids||retinogenesis||gene augmentation||adeno-associated virus (AAV)||n/a