AAV-mediated gene therapy in the guanylate cyclase (RetGC1/RetGC2) double knockout mouse model of Leber congenital amaurosis.
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A comprehensive review of retinal gene therapyHomozygosity mapping and targeted sanger sequencing reveal genetic defects underlying inherited retinal disease in families from pakistan.Successful gene therapy in the RPGRIP1-deficient dog: a large model of cone-rod dystrophy.Gene therapy: charting a future course--summary of a National Institutes of Health Workshop, April 12, 2013.Vector platforms for gene therapy of inherited retinopathies.Capsid Mutated Adeno-Associated Virus Delivered to the Anterior Chamber Results in Efficient Transduction of Trabecular Meshwork in Mouse and RatOutcome measure for the treatment of cone photoreceptor diseases: orientation to a scene with cone-only contrastEffects of Subretinal Gene Transfer at Different Time Points in a Mouse Model of Retinal Degeneration.Gene Therapy Fully Restores Vision to the All-Cone Nrl(-/-) Gucy2e(-/-) Mouse Model of Leber Congenital Amaurosis-1.It's never too late to save a photoreceptor.Retinal Gene Therapy: Current Progress and Future Prospects.Let There Be Light: Gene and Cell Therapy for Blindness.RD3 gene delivery restores guanylate cyclase localization and rescues photoreceptors in the Rd3 mouse model of Leber congenital amaurosis 12.Advances in Gene Therapy for Diseases of the Eye.Functional study of two biochemically unusual mutations in GUCY2D Leber congenital amaurosis expressed via adenoassociated virus vector in mouse retinas.Dual adeno-associated virus vectors result in efficient in vitro and in vivo expression of an oversized gene, MYO7A.Stem cells for investigation and treatment of inherited retinal disease.Insights gained from gene therapy in animal models of retGC1 deficiency.Gene therapies for inherited retinal disorders.Leber congenital amaurosis caused by mutations in GUCY2D.Animal modelling for inherited central vision loss.AAV8(Y733F)-mediated gene therapy in a Spata7 knockout mouse model of Leber congenital amaurosis and retinitis pigmentosaGene and cell-based therapies for inherited retinal disorders: An update.Leber congenital amaurosis/early-onset severe retinal dystrophy: clinical features, molecular genetics and therapeutic interventions.A system to measure the pupil response to steady lights in freely behaving mice.Dysfunction of outer segment guanylate cyclase caused by retinal disease related mutations.Retinal gene therapy using adeno-associated viral vectors: multiple applications for a small virus.Synthetic adeno-associated viral vector efficiently targets mouse and non-human primate retina in vivo.Targeting iodothyronine deiodinases locally in the retina is a therapeutic strategy for retinal degeneration.GUCY2D Cone-Rod Dystrophy-6 is a 'Phototransduction Disease' Triggered by Abnormal Calcium Feedback on Retinal Membrane Guanylyl Cyclase 1.The R838S Mutation in Retinal Guanylyl Cyclase 1 (RetGC1) Alters Calcium Sensitivity of cGMP Synthesis in the Retina and Causes Blindness in Transgenic Mice.Photoreceptor Guanylate Cyclase () Mutations Cause Retinal Dystrophies by Severe Malfunction of Ca-Dependent Cyclic GMP Synthesis
P2860
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P2860
AAV-mediated gene therapy in the guanylate cyclase (RetGC1/RetGC2) double knockout mouse model of Leber congenital amaurosis.
description
2013 nî lūn-bûn
@nan
2013年の論文
@ja
2013年論文
@yue
2013年論文
@zh-hant
2013年論文
@zh-hk
2013年論文
@zh-mo
2013年論文
@zh-tw
2013年论文
@wuu
2013年论文
@zh
2013年论文
@zh-cn
name
AAV-mediated gene therapy in t ...... of Leber congenital amaurosis.
@ast
AAV-mediated gene therapy in t ...... of Leber congenital amaurosis.
@en
type
label
AAV-mediated gene therapy in t ...... of Leber congenital amaurosis.
@ast
AAV-mediated gene therapy in t ...... of Leber congenital amaurosis.
@en
prefLabel
AAV-mediated gene therapy in t ...... of Leber congenital amaurosis.
@ast
AAV-mediated gene therapy in t ...... of Leber congenital amaurosis.
@en
P2093
P2860
P921
P356
P1433
P1476
AAV-mediated gene therapy in t ...... of Leber congenital amaurosis
@en
P2093
Alexander Dizhoor
Christine N Kay
Frank M Dyka
Igor V Peshenko
Issam McDoom
Sanford L Boye
Seok Hong Min
Shannon E Boye
Sukanya Karan
Thomas C Foster
P2860
P304
P356
10.1089/HUM.2012.193
P577
2013-02-01T00:00:00Z