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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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Guardat en:
Format: Online
Idioma:anglès
Publicat: MDPI - Multidisciplinary Digital Publishing Institute 2021
Matèries:
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
thema EDItEUR::G Reference, Information and Interdisciplinary subjects::GP Research and information: general
thema EDItEUR::P Mathematics and Science::PS Biology, life sciences
Accés en línia:ONIX_20210501_9783039431762_920
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