Molecular Therapies for Inherited Retinal Diseases

Following the implementation of next-generation sequencing technologies (e.g., exome and genome sequencing) in molecular diagnostics, the majority of genetic defects underlying inherited retinal disease (IRD) can readily be identified. In parallel, opportunities to counteract the molecular consequen...

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Drugi avtorji:	Collin, Rob W.J (Editor), Garanto, Alejandro (Editor)
Format:	Book Chapter
Izdano:	Basel, Switzerland MDPI - Multidisciplinary Digital Publishing Institute 2020
Teme:	Research & information: general Biology, life sciences 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
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Molecular Therapies for Inherited Retinal Diseases

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