Correction of the disease phenotype in the mouse model of Stargardt disease by lentiviral gene therapy - Test2 - elhamkhani - TriplyDB

Correction of the disease phenotype in the mouse model of Stargardt disease by lentiviral gene therapy

Correction of the disease phenotype in the mouse model of Stargardt disease by lentiviral gene therapy

http://www.wikidata.org/entity/Q24621941

about

Gene therapy of inherited retinal degenerations: prospects and challenges Immunology of AAV-Mediated Gene Transfer in the Eye A comprehensive review of retinal gene therapy Advancing therapeutic strategies for inherited retinal degeneration: recommendations from the Monaciano Symposium Development of gene and stem cell therapy for ocular neurodegeneration Biology and therapy of inherited retinal degenerative disease: insights from mouse models Retinal degeneration progression changes lentiviral vector cell targeting in the retina Quantitative Fundus Autofluorescence and Optical Coherence Tomography in PRPH2/RDS- and ABCA4-Associated Disease Exhibiting Phenotypic Overlap.Phospholipid meets all-trans-retinal: the making of RPE bisretinoids CLINICAL PROGRESS IN INHERITED RETINAL DEGENERATIONS: GENE THERAPY CLINICAL TRIALS AND ADVANCES IN GENETIC SEQUENCING.Effects of organic solvents on two retinal pigment epithelial lipofuscin fluorophores, A2E and all-trans-retinal dimer Enzymatic degradation of A2E, a retinal pigment epithelial lipofuscin bisretinoid.The retinal pigment epithelium in health and disease Photoreceptor cell degeneration in Abcr (-/-) mice Defective lipid transport and biosynthesis in recessive and dominant Stargardt macular degeneration Genetic basis of inherited macular dystrophies and implications for stem cell therapy.Successful arrest of photoreceptor and vision loss expands the therapeutic window of retinal gene therapy to later stages of disease.Vector platforms for gene therapy of inherited retinopathies.Analysis of the ABCA4 genomic locus in Stargardt disease.Deuterium enrichment of vitamin A at the C20 position slows the formation of detrimental vitamin A dimers in wild-type rodents Quantitative fundus autofluorescence in mice: correlation with HPLC quantitation of RPE lipofuscin and measurement of retina outer nuclear layer thickness Gene therapeutic approach using mutation-adapted U1 snRNA to correct a RPGR splice defect in patient-derived cells.Light filtering in a retinal pigment epithelial cell culture model.Clinical characteristics and current therapies for inherited retinal degenerations Natural history of cone disease in the murine model of Leber congenital amaurosis due to CEP290 mutation: determining the timing and expectation of therapy.EIAV-based retinal gene therapy in the shaker1 mouse model for usher syndrome type 1B: development of UshStat.Gene therapy for ocular diseases Republished review: Gene therapy for ocular diseases.Analysis of the ABCA4 gene by next-generation sequencing.Membrane receptors and transporters involved in the function and transport of vitamin A and its derivatives.C1q enhances cone photoreceptor survival in a mouse model of autosomal recessive retinitis pigmentosa.Functional and anatomic consequences of subretinal dosing in the cynomolgus macaque Gene Therapy of ABCA4-Associated Diseases.Gene Therapy for the Retinal Degeneration of Usher Syndrome Caused by Mutations in MYO7A The bisretinoids of retinal pigment epithelium X-linked juvenile retinoschisis: clinical diagnosis, genetic analysis, and molecular mechanisms.Predicting Progression of ABCA4-Associated Retinal Degenerations Based on Longitudinal Measurements of the Leading Disease Front.Prospectives for gene therapy of retinal degenerations Gene therapy of inherited retinopathies: a long and successful road from viral vectors to patients.DNA nanoparticle-mediated ABCA4 delivery rescues Stargardt dystrophy in mice.

P2860

Q27009261-52241235-45BD-4E3F-B035-FE089410AD3F Q27023474-20CEE20A-40D5-4CF4-BB31-37653D5FFED6 Q27023914-4D22581C-5432-468D-8F7B-58BD9687403A Q27694625-A30076E2-5B73-4D6D-BD2C-FC17D09300CE Q28081549-D7F55B94-7824-4586-813B-692370AA1FCC Q28084726-1AA77D88-08CA-4149-8081-6BF201378A17 Q28741007-E73F5A8A-C769-4462-B729-0D2E2C4E7953 Q30652635-64C1019D-EC92-4D3E-90A7-70276A15FA6E Q33577803-C95BEB9C-169E-4035-B675-1B416ED5CCA9 Q33782257-99CBB4BC-61F0-40C7-A66F-253EE965D876 Q33899116-2048E526-B4E0-4D8B-B7AF-B209A6FC3AD4 Q33956569-AA666D52-4474-4B94-BEC4-C946F8F698EC Q33998365-370CEDF9-E7A1-4192-9CB8-63342DE141F5 Q34037711-9BA0B18C-2367-4934-91EC-DF0A04A00D17 Q34157723-55422A7B-D2EC-4E28-A19B-D4805AF6CA2C Q34234455-4472A0E4-9DB4-4308-BA1F-74177946C201 Q34497784-59ADA7E2-5796-47FC-9639-F2A6E05F4559 Q34561531-AB076F97-7A75-44A2-AA60-67190E9C8044 Q34575705-4A81D7C7-FFAA-47D8-AE92-D3D4F42C85ED Q34624198-88D9D02E-998C-44CC-97DC-B8B555991AB6 Q34645637-324BD693-3DE2-4F3A-8D33-4D50ACA58D4A Q34994553-995D4172-79D7-4008-AEFA-1E387F5EF860 Q35000314-CCFCF662-39BA-42E0-A945-44FDBE6DD6CA Q35045067-5C3682FD-069F-4AAF-B3CF-5E8C6C4F3528 Q35132187-598A3183-3AA2-4C6E-AE83-859722C8C3DD Q35140281-13244E2A-6607-4C3A-93AE-EFA848E2C8D4 Q35160142-CD6D8487-06BE-436D-9A10-C89EC9C44B11 Q35210756-BF4E1077-F6EC-4C06-9A1F-F9D2176C535A Q35524609-FF405623-0453-46CE-B5DB-363D51A87A38 Q35567312-31837518-0BF8-428A-AB9F-D471912EE9F7 Q35603676-347C1260-C98D-46B2-80EA-A5BCD7821D78 Q35663467-A2DC0AC3-A5C6-4807-BA68-C5D21377F3B4 Q35663857-3D6545BC-2B45-48EA-9B36-712ACC1E4443 Q35664199-96C00978-41EE-4347-89CE-81EA7A84C879 Q35785390-08D8BA5A-B78E-4284-895A-F714E541A162 Q35907087-28F0F822-B584-4532-B331-62407032A855 Q36067981-DBF60C91-5195-49D1-BAC7-42DC520D3D12 Q36108106-D230D5A2-6983-41C6-A61D-79CFC5978756 Q36147481-D9E08251-5153-4B4D-AD0E-0CD297D5B6DF Q36190772-5B8071A2-070E-4736-8B5E-D3E33FA68144

P2860

Correction of the disease phenotype in the mouse model of Stargardt disease by lentiviral gene therapy

http://www.wikidata.org/entity/Q24621941

description

2008 nî lūn-bûn

@nan

2008 թուականի Հոկտեմբերին հրատարակուած գիտական յօդուած

@hyw

2008 թվականի հոտեմբերին հրատարակված գիտական հոդված

@hy

2008年の論文

@ja

2008年論文

@yue

2008年論文

@zh-hant

2008年論文

@zh-hk

2008年論文

@zh-mo

2008年論文

@zh-tw

2008年论文

@wuu

name

Correction of the disease phen ...... ase by lentiviral gene therapy

@ast

Correction of the disease phen ...... ase by lentiviral gene therapy

@en

Correction of the disease phen ...... ase by lentiviral gene therapy

@nl

type

label

Correction of the disease phen ...... ase by lentiviral gene therapy

@ast

Correction of the disease phen ...... ase by lentiviral gene therapy

@en

Correction of the disease phen ...... ase by lentiviral gene therapy

@nl

prefLabel

Correction of the disease phen ...... ase by lentiviral gene therapy

@ast

Correction of the disease phen ...... ase by lentiviral gene therapy

@en

Correction of the disease phen ...... ase by lentiviral gene therapy

@nl

P1433

Q24621941-B3C72B81-D51F-4D1C-8161-46B9AE36BF96

P1476

Q24621941-4A7A3055-C994-46B6-A8B3-7CC6D15BD817

P2093

Q24621941-0B8E59EB-AA14-4241-BF10-236871DE5A1F

Q24621941-1A0250A9-B522-45AD-BFB2-CA61C33FE4CC

Q24621941-26CFDD44-9BC6-4148-9A6A-62925CCCCAF9

Q24621941-2A848E2C-7D2B-4EC7-8071-9B87D0D54EEB

Q24621941-5BDEF536-CEE8-4F9A-910C-1F35EF54C0F1

Q24621941-6E3A08A1-881D-477F-84D7-326DBD8D06CB

Q24621941-97C8FC4F-36F4-4D8F-884E-9EFEB06C2788

Q24621941-A8BF7E03-F98B-42B5-A5B5-45E00DE6A1B2

Q24621941-B98A0639-502E-4510-87B2-D4E842718760

Q24621941-BFF31D93-B66A-47A6-B6B8-6F372D52A855

P2860

Q24621941-04CA18E7-37E9-4659-AC02-7F0AE399C429

Q24621941-07660BD1-7F90-45AE-AECC-1D944100308A

Q24621941-09A726F6-7D83-4E71-AFAA-B9E597739730

Q24621941-0B60E86D-585E-40E4-AED7-272D64745EB7

Q24621941-0C0B167D-C3A8-4E1A-978E-0D9A2EC498C3

Q24621941-0E0B411E-83DD-4635-960F-7E8C8EB5A79F

Q24621941-1344768E-BF35-4CE4-B45E-2A07AF88A97F

Q24621941-195417D8-E646-4E42-AB82-768DA0B422C6

Q24621941-1D6AA49F-FAFE-42F1-8797-9C00CFB9D196

Q24621941-1ED24AFA-A5C7-4075-9263-729CA0E9A348

P2888

Q24621941-F5D97B42-E6FD-479E-AF54-F21206536934

P304

Q24621941-70CBBB27-5D8D-449C-B26B-112D64072D0E

P31

Q24621941-2E976B8C-226D-4EA6-963C-6B11D1F8F584

P3181

Q24621941-63CB1750-3C31-47DA-B86E-3A892EEF7334

P356

Q24621941-A3F6134F-6E7D-43E2-8527-C9173F4E4F0B

P407

Q24621941-C058DAD8-16F1-41DF-A109-60AF59686B03

P433

Q24621941-90786A3D-F87C-48ED-8DE6-12A6C2BC0D23

P478

Q24621941-0838A56A-7D18-483C-8929-A2442C5E2E6F

P577

Q24621941-F2C73C34-6710-4CF7-9050-2AE41E351833

P5875

Q24621941-2EBC4355-43A1-4091-A3AD-CD213B5B8DCC

P6179

Q24621941-69E55520-AB83-4532-925B-9061A75CED63

P698

Q24621941-99C52154-83A4-4099-83C2-703179742653

P921

Q24621941-0D4E835D-8744-4CC5-872A-1DC248156089

P932

Q24621941-E0CF4FD7-DD6B-436A-ABEB-DEADC844B065

P3181

P356

P1433

P1476

Correction of the disease phen ...... ase by lentiviral gene therapy

@en

P2093

I Pata

http://www.w3.org/2001/XMLSchema#string

J Kong

http://www.w3.org/2001/XMLSchema#string

J Mannik

http://www.w3.org/2001/XMLSchema#string

J R Sparrow

http://www.w3.org/2001/XMLSchema#string

J Zernant-Rajang

http://www.w3.org/2001/XMLSchema#string

K Binley

http://www.w3.org/2001/XMLSchema#string

K Doi

http://www.w3.org/2001/XMLSchema#string

O Kan

http://www.w3.org/2001/XMLSchema#string

P Gouras

http://www.w3.org/2001/XMLSchema#string

R Allikmets

http://www.w3.org/2001/XMLSchema#string

P2860

Lentiviral gene transfer of RPE65 rescues survival and function of cones in a mouse model of Leber congenital amaurosis

A photoreceptor cell-specific ATP-binding transporter gene (ABCR) is mutated in recessive Stargardt macular dystrophy

Genotype/Phenotype analysis of a photoreceptor-specific ATP-binding cassette transporter gene, ABCR, in Stargardt disease

The 2588G-->C mutation in the ABCR gene is a mild frequent founder mutation in the Western European population and allows the classification of ABCR mutations in patients with Stargardt disease

Pharmacological and rAAV gene therapy rescue of visual functions in a blind mouse model of Leber congenital amaurosis.

The photoreceptor rim protein is an ABC transporter encoded by the gene for recessive Stargardt's disease (ABCR)

Retinitis pigmentosa caused by a homozygous mutation in the Stargardt disease gene ABCR

Retinal stimulates ATP hydrolysis by purified and reconstituted ABCR, the photoreceptor-specific ATP-binding cassette transporter responsible for Stargardt disease

Insights into the function of Rim protein in photoreceptors and etiology of Stargardt's disease from the phenotype in abcr knockout mice

Variation of clinical expression in patients with Stargardt dystrophy and sequence variations in the ABCR gene

P2888

P304

1311-20

http://www.w3.org/2001/XMLSchema#string

P31

scholarly article

P3181

1651525

http://www.w3.org/2001/XMLSchema#string

P356

10.1038/GT.2008.78

http://www.w3.org/2001/XMLSchema#string

P407

P433

19

http://www.w3.org/2001/XMLSchema#string

P478

15

http://www.w3.org/2001/XMLSchema#string

P577

2008-10-01T00:00:00Z

http://www.w3.org/2001/XMLSchema#dateTime

P5875

5387318

http://www.w3.org/2001/XMLSchema#string

P6179

1006240059

http://www.w3.org/2001/XMLSchema#string

P698

18463687

http://www.w3.org/2001/XMLSchema#string

P921

P932

3110063

http://www.w3.org/2001/XMLSchema#string