Gene therapy rescues photoreceptor blindness in dogs and paves the way for treating human X-linked retinitis pigmentosa. - Wikidata - awgldk - TriplyDB

Gene therapy rescues photoreceptor blindness in dogs and paves the way for treating human X-linked retinitis pigmentosa.

Gene therapy rescues photoreceptor blindness in dogs and paves the way for treating human X-linked retinitis pigmentosa.

http://www.wikidata.org/entity/Q35751018

about

The interplay between RPGR, PDEδ and Arl2/3 regulate the ciliary targeting of farnesylated cargo Lipid Nanoparticles for Ocular Gene Delivery Dawn of ocular gene therapy: implications for molecular diagnosis in retinal disease Immunology of AAV-Mediated Gene Transfer in the Eye Rapid cohort generation and analysis of disease spectrum of large animal model of cone dystrophy Canine CNGA3 Gene Mutations Provide Novel Insights into Human Achromatopsia-Associated Channelopathies and Treatment Canine retina has a primate fovea-like bouquet of cone photoreceptors which is affected by inherited macular degenerations Advancing therapeutic strategies for inherited retinal degeneration: recommendations from the Monaciano Symposium Restoration of vision in the pde6β-deficient dog, a large animal model of rod-cone dystrophy Ablation of the X-linked retinitis pigmentosa 2 (Rp2) gene in mice results in opsin mislocalization and photoreceptor degeneration Homozygosity mapping and targeted sanger sequencing reveal genetic defects underlying inherited retinal disease in families from pakistan.The human rhodopsin kinase promoter in an AAV5 vector confers rod- and cone-specific expression in the primate retina.Successful gene therapy in the RPGRIP1-deficient dog: a large model of cone-rod dystrophy.Simulation of thalamic prosthetic vision: reading accuracy, speed, and acuity in sighted humans Establishment and evolution of the Australian Inherited Retinal Disease Register and DNA Bank.Impact of Autoantibodies against Glycolytic Enzymes on Pathogenicity of Autoimmune Retinopathy and Other Autoimmune Disorders From caveman companion to medical innovator: genomic insights into the origin and evolution of domestic dogs.Quantitative Analysis of Retinal Structure Using Spectral-Domain Optical Coherence Tomography in RPGR-Associated Retinopathy A Coding Variant in the Gene Bardet-Biedl Syndrome 4 (BBS4) Is Associated with a Novel Form of Canine Progressive Retinal Atrophy.A novel form of progressive retinal atrophy in Swedish vallhund dogs Rd9 is a naturally occurring mouse model of a common form of retinitis pigmentosa caused by mutations in RPGR-ORF15 Successful arrest of photoreceptor and vision loss expands the therapeutic window of retinal gene therapy to later stages of disease.A digital atlas of the dog brain Vector platforms for gene therapy of inherited retinopathies.Evaluation of lateral spread of transgene expression following subretinal AAV-mediated gene delivery in dogs Evaluation of multimodal imaging in carriers of X-linked retinitis pigmentosa.Translational models of ocular disease.A large animal model for CNGB1 autosomal recessive retinitis pigmentosa.Recombinant AAV-mediated BEST1 transfer to the retinal pigment epithelium: analysis of serotype-dependent retinal effects.Gene augmentation for X-linked retinitis pigmentosa caused by mutations in RPGR Up-regulation of tumor necrosis factor superfamily genes in early phases of photoreceptor degeneration An Intronic SINE insertion in FAM161A that causes exon-skipping is associated with progressive retinal atrophy in Tibetan Spaniels and Tibetan Terriers A long-term efficacy study of gene replacement therapy for RPGR-associated retinal degeneration Differential targeting of feline photoreceptors by recombinant adeno-associated viral vectors: implications for preclinical gene therapy trials.RPGR: Its role in photoreceptor physiology, human disease, and future therapies.Stability and Safety of an AAV Vector for Treating RPGR-ORF15 X-Linked Retinitis Pigmentosa.Application of a high-throughput genotyping method for loci exclusion in non-consanguineous Australian pedigrees with autosomal recessive retinitis pigmentosa.What's in a name? RPGR mutations redefine the genetic and phenotypic landscape in retinal degenerative diseases.RPGR-associated retinal degeneration in human X-linked RP and a murine model.Inhibition of Matrix Metalloproteinase 9 Enhances Rod Survival in the S334ter-line3 Retinitis Pigmentosa Model.

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P2860

Gene therapy rescues photoreceptor blindness in dogs and paves the way for treating human X-linked retinitis pigmentosa.

http://www.wikidata.org/entity/Q35751018

description

2012 nî lūn-bûn

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2012年論文

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2012年論文

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2012年論文

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2012年論文

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2012年论文

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2012年论文

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2012年论文

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name

Gene therapy rescues photorece ...... X-linked retinitis pigmentosa.

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Gene therapy rescues photorece ...... X-linked retinitis pigmentosa.

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type

label

Gene therapy rescues photorece ...... X-linked retinitis pigmentosa.

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Gene therapy rescues photorece ...... X-linked retinitis pigmentosa.

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Gene therapy rescues photorece ...... X-linked retinitis pigmentosa.

@ast

Gene therapy rescues photorece ...... X-linked retinitis pigmentosa.

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PNAS.1118847109

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Proceedings of the National Academy of Sciences of the United States of America

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Gene therapy rescues photorece ...... X-linked retinitis pigmentosa.

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Alejandro J Román

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Alexander Sumaroka

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Artur V Cideciyan

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Diego S Fajardo

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Malgorzata Swider

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Sanford L Boye

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Sem Genini

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Simone Iwabe

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Tomas S Alemán

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Vince A Chiodo

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P2860

RPGR ORF15 isoform co-localizes with RPGRIP1 at centrioles and basal bodies and interacts with nucleophosmin

Positional cloning of the gene for X-linked retinitis pigmentosa 2

RP2 and RPGR mutations and clinical correlations in patients with X-linked retinitis pigmentosa

RPGR-ORF15, which is mutated in retinitis pigmentosa, associates with SMC1, SMC3, and microtubule transport proteins

Gene therapy rescues cone function in congenital achromatopsia.

A comprehensive mutation analysis of RP2 and RPGR in a North American cohort of families with X-linked retinitis pigmentosa

A retinitis pigmentosa GTPase regulator (RPGR)-deficient mouse model for X-linked retinitis pigmentosa (RP3)

Rod photoreceptor neurite sprouting in retinitis pigmentosa

X-linked retinitis pigmentosa: RPGR mutations in most families with definite X linkage and clustering of mutations in a short sequence stretch of exon ORF15

Retinal degeneration mutants in the mouse

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2132-2137

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10.1073/PNAS.1118847109

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6

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109

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Samuel G. Jacobson

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Gustavo D Aguirre

William W Hauswirth

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2012-01-23T00:00:00Z

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221808930

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22308428

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Photoreceptor protein

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3277562

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