Long-term RNA interference gene therapy in a dominant retinitis pigmentosa mouse model

scientific article published on 31 October 2011

Long-term RNA interference gene therapy in a dominant retinitis pigmentosa mouse model is …
instance of (P31):
scholarly articleQ13442814

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P819ADS bibcode2011PNAS..10818476J
P356DOI10.1073/PNAS.1112758108
P932PMC publication ID3215008
P698PubMed publication ID22042849
P5875ResearchGate publication ID51760180

P50authorWilliam W HauswirthQ64478429
P2093author name stringLi Jiang
Jeanne M Frederick
Wolfgang Baehr
Houbin Zhang
Shannon E Boye
Alexander M Dizhoor
P2860cites workMolecular characterization of a third member of the guanylyl cyclase-activating protein subfamilyQ22008807
Identification and functional consequences of a new mutation (E155G) in the gene for GCAP1 that causes autosomal dominant cone dystrophy.Q24535806
A novel GCAP1 missense mutation (L151F) in a large family with autosomal dominant cone-rod dystrophy (adCORD)Q24539234
Guanylate cyclase-activating protein (GCAP) 1 rescues cone recovery kinetics in GCAP1/GCAP2 knockout miceQ24670608
A mutation in guanylate cyclase activator 1A (GUCA1A) in an autosomal dominant cone dystrophy pedigree mapping to a new locus on chromosome 6p21.1Q28118822
Autosomal dominant cone and cone-rod dystrophy with mutations in the guanylate cyclase activator 1A gene-encoding guanylate cyclase activating protein-1Q28142044
Current prospects for RNA interference-based therapiesQ28235733
Mutation in the gene GUCA1A, encoding guanylate cyclase-activating protein 1, causes cone, cone-rod, and macular dystrophyQ28256273
Constitutive activation of photoreceptor guanylate cyclase by Y99C mutant of GCAP-1. Possible role in causing human autosomal dominant cone degenerationQ28275397
GCAP1 (Y99C) mutant is constitutively active in autosomal dominant cone dystrophyQ28279451
Role of guanylate cyclase-activating proteins (GCAPs) in setting the flash sensitivity of rod photoreceptorsQ28593705
Ribozyme-mediated repair of defective mRNA by targeted, trans-splicingQ30465698
RNA interference by expression of short-interfering RNAs and hairpin RNAs in mammalian cellsQ30476196
The Y99C mutation in guanylyl cyclase-activating protein 1 increases intracellular Ca2+ and causes photoreceptor degeneration in transgenic mice.Q30764452
Small interfering RNA (siRNA) targeting VEGF effectively inhibits ocular neovascularization in a mouse model.Q30944070
Increased light exposure alleviates one form of photoreceptor degeneration marked by elevated calcium in the darkQ33521262
GCAP1 rescues rod photoreceptor response in GCAP1/GCAP2 knockout miceQ34078737
Suppression and replacement gene therapy for autosomal dominant disease in a murine model of dominant retinitis pigmentosaQ34158497
Role of guanylyl cyclase modulation in mouse cone phototransduction.Q34189068
Characterization of retinal guanylate cyclase-activating protein 3 (GCAP3) from zebrafish to man.Q34343088
Sequence- and target-independent angiogenesis suppression by siRNA via TLR3Q34764750
Self-complementary AAV vectors; advances and applicationsQ34804660
Long-term preservation of cone photoreceptors and restoration of cone function by gene therapy in the guanylate cyclase-1 knockout (GC1KO) mouse.Q35518971
Gene therapy progress and prospects: the eye.Q36534765
Applications of RNA interference in mammalian systems.Q36810329
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Hereditary retinal diseaseQ37137469
Constitutive excitation by Gly90Asp rhodopsin rescues rods from degeneration caused by elevated production of cGMP in the dark.Q37170561
Barriers for retinal gene therapy: separating fact from fictionQ37195446
AAV retinal transduction in a large animal model species: comparison of a self-complementary AAV2/5 with a single-stranded AAV2/5 vector.Q37346460
Recognizing and avoiding siRNA off-target effects for target identification and therapeutic applicationQ37665303
RNAi: a potential new class of therapeutic for human genetic diseaseQ37871417
Improved retinal function in a mouse model of dominant retinitis pigmentosa following AAV-delivered gene therapyQ39505058
AAV-mediated knockdown of peripherin-2 in vivo using miRNA-based hairpinsQ43221778
A role for GCAP2 in regulating the photoresponse. Guanylyl cyclase activation and rod electrophysiology in GUCA1B knock-out miceQ43252780
Adeno-associated virus-mediated rhodopsin replacement provides therapeutic benefit in mice with a targeted disruption of the rhodopsin gene.Q43262568
Simple RNA enzymes with new and highly specific endoribonuclease activitiesQ43501578
Mutations in the GUCA1A gene involved in hereditary cone dystrophies impair calcium-mediated regulation of guanylate cyclaseQ45103610
Assessment of ocular transduction using single-stranded and self-complementary recombinant adeno-associated virus serotype 2/8.Q45399272
Safety of recombinant adeno-associated virus type 2-RPE65 vector delivered by ocular subretinal injectionQ45417238
Recombinant adeno-associated virus purification using novel methods improves infectious titer and yieldQ45747699
Suppression of ocular neovascularization with siRNA targeting VEGF receptor 1.Q45887309
P433issue45
P407language of work or nameEnglishQ1860
P921main subjectRNA interferenceQ201993
gene therapyQ213901
retinitis pigmentosaQ847057
P304page(s)18476-18481
P577publication date2011-10-31
P1433published inProceedings of the National Academy of Sciences of the United States of AmericaQ1146531
P1476titleLong-term RNA interference gene therapy in a dominant retinitis pigmentosa mouse model
P478volume108

Reverse relations

cites work (P2860)
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