Serotype-dependent packaging of large genes in adeno-associated viral vectors results in effective gene delivery in mice.
about
The adenovirus genome contributes to the structural stability of the virionPromising and delivering gene therapies for vision lossGene therapy of inherited retinal degenerations: prospects and challengesAAV-mediated gene therapy in mouse models of recessive retinal degenerationA comprehensive review of retinal gene therapyHomologous recombination mediates functional recovery of dysferlin deficiency following AAV5 gene transferComparative analysis of DNA nanoparticles and AAVs for ocular gene deliveryDisease rescue and increased lifespan in a model of cardiomyopathy and muscular dystrophy by combined AAV treatmentsStructural Insights into Adeno-Associated Virus Serotype 5Stargardt disease: clinical features, molecular genetics, animal models and therapeutic optionsGene therapy for hemophiliaRetinal pigment epithelium defects in humans and mice with mutations in MYO7A: imaging melanosome-specific autofluorescence.The N-terminal region of centrosomal protein 290 (CEP290) restores vision in a zebrafish model of human blindness.Myosin7a deficiency results in reduced retinal activity which is improved by gene therapy.Adenoviral-mediated gene transfer restores plasma ADAMTS13 antigen and activity in ADAMTS13 knockout mice.Characterization of genome integrity for oversized recombinant AAV vector.Inner limiting membrane barriers to AAV-mediated retinal transduction from the vitreous.Effect of genome size on AAV vector packaging.Evidence for the failure of adeno-associated virus serotype 5 to package a viral genome > or = 8.2 kb.Effects of organic solvents on two retinal pigment epithelial lipofuscin fluorophores, A2E and all-trans-retinal dimerThe potential of adeno-associated viral vectors for gene delivery to muscle tissue.Deletion of the B-B' and C-C' regions of inverted terminal repeats reduces rAAV productivity but increases transgene expressionEnzymatic degradation of A2E, a retinal pigment epithelial lipofuscin bisretinoid.The retinal pigment epithelium in health and diseaseSuppression and replacement gene therapy for autosomal dominant disease in a murine model of dominant retinitis pigmentosaModular dispensability of dysferlin C2 domains reveals rational design for mini-dysferlin molecules.Proteasome inhibitors enhance gene delivery by AAV virus vectors expressing large genomes in hemophilia mouse and dog models: a strategy for broad clinical application.Gene therapy of mdx mice with large truncated dystrophins generated by recombination using rAAV6.Vector platforms for gene therapy of inherited retinopathies.Cystic fibrosis transmembrane conductance regulator with a shortened R domain rescues the intestinal phenotype of CFTR-/- mice.Quantitative fundus autofluorescence in mice: correlation with HPLC quantitation of RPE lipofuscin and measurement of retina outer nuclear layer thicknessAssessment of tropism and effectiveness of new primate-derived hybrid recombinant AAV serotypes in the mouse and primate retinaLight filtering in a retinal pigment epithelial cell culture model.AAV and compacted DNA nanoparticles for the treatment of retinal disorders: challenges and future prospects.Subretinal gene delivery using helper-dependent adenoviral vectors.Recombinant AAV-directed gene therapy for type I glycogen storage diseases.Development of a liver-specific Tet-on inducible system for AAV vectors and its application in the treatment of liver cancerNatural history of cone disease in the murine model of Leber congenital amaurosis due to CEP290 mutation: determining the timing and expectation of therapy.Gene therapy for ocular diseasesAAV.Dysferlin Overlap Vectors Restore Function in Dysferlinopathy Animal Models
P2860
Q26825101-D6885D11-3980-4434-9EB7-93EEE9C6A5DFQ27007079-AC579247-0F86-44A8-B97D-C0997830880EQ27009261-A725E910-79D0-4DE6-AFA1-9F23649824F7Q27009291-954FB51C-7ED5-45E9-AE6A-B93F18C7A69BQ27023914-1A0D3877-1BE7-41FA-8FEF-2894CA9EC92BQ27300915-B1C05226-6E77-489E-A4B9-CEED5F01AB1FQ27325842-896A69EF-2103-4F70-B13F-C5BC666B71FAQ27349992-E502A6FB-10EB-4688-AE40-88D0FD6D9D75Q27679454-2FD8C70B-3AF7-4386-A809-D78AB5A96584Q28075612-94BEC432-61F0-4053-A75B-FDF3C6CCA9F3Q28081911-030AE80C-9F69-42AA-9143-0AF92438C342Q30494855-208BE1C8-C9FA-447E-9C60-CC45C1590ABBQ30499095-C6F23F48-E499-4E5F-8DA1-96EF64DA38DBQ31132177-641BACAD-A13C-43BB-8625-044A5C9D9DEBQ33385791-039A4209-9B3E-4A5B-92D2-7095887FE339Q33576852-01759F81-5444-4297-8D52-5DBD5ED2041AQ33622746-0ACBE273-7475-4CCD-AD50-5C37A038E1CDQ33730405-24388AC8-C0F1-40F4-A2DC-D2092BEA4281Q33730469-2CC54C91-C869-405D-82DD-C1DB909FD814Q33899116-B3DF6DE2-6748-4CA8-A1F4-5AC6AC931495Q33902499-18F83B13-1E08-4250-B00E-B0BBF9C20C80Q33905659-41B56DF6-EEE4-4534-B54E-951F6690CFEAQ33956569-B03C4CF2-473E-4F4E-8ABE-5E987D6C2975Q33998365-90F97B63-54D0-478B-B4B2-DDC7D9C03996Q34158497-74335A60-7102-42CD-B8C1-684550A244FBQ34318021-8FD68405-8DA1-42EC-B919-EFE4ABD2FD80Q34343763-9D5EC983-5678-4900-8CD7-E745B2607C1EQ34473295-A8C52789-E2CB-4D35-B49D-35CF363572AEQ34561531-3608527F-D4E1-4AB9-BB1C-F5F531E317F7Q34582621-E45EC5D0-6545-489F-AFCD-7F33563D550EQ34645637-7F6CF3E3-9C77-4A22-99D0-3801EACC1CADQ34673011-26CB86BA-E515-4B9F-9EF7-56F92EBA196CQ35000314-223056B3-2EF3-466C-8675-4F0104CA1EF2Q35027496-822E5537-B7A0-4406-ABB9-09A526E47551Q35073956-3F2506DE-F5ED-44B7-AAAE-7AB20C8453ABQ35078590-84B3DDF0-8FD5-40E0-B7F9-EDCA6BD54E05Q35086111-F126E189-DD13-4F72-8203-584231456B57Q35132187-EFA3195E-780B-47FC-A409-E956D6C913C5Q35160142-DC818D9E-BE43-4E6B-9E3D-9B6F4517B6C9Q35204883-048A885F-4CB9-4AA9-A8E8-58CE46352FA8
P2860
Serotype-dependent packaging of large genes in adeno-associated viral vectors results in effective gene delivery in mice.
description
2008 nî lūn-bûn
@nan
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
2008年论文
@zh
2008年论文
@zh-cn
name
Serotype-dependent packaging o ...... fective gene delivery in mice.
@ast
Serotype-dependent packaging o ...... fective gene delivery in mice.
@en
type
label
Serotype-dependent packaging o ...... fective gene delivery in mice.
@ast
Serotype-dependent packaging o ...... fective gene delivery in mice.
@en
prefLabel
Serotype-dependent packaging o ...... fective gene delivery in mice.
@ast
Serotype-dependent packaging o ...... fective gene delivery in mice.
@en
P2093
P2860
P50
P356
P1476
Serotype-dependent packaging o ...... ffective gene delivery in mice
@en
P2093
Albert Maguire
Daniel Gibbs
David S Williams
Janet R Sparrow
Jean Bennett
Marco Petrillo
Maria Garcia-Hoyos
Mariacarmela Allocca
Monica Doria
P2860
P304
P356
10.1172/JCI34316
P407
P577
2008-05-01T00:00:00Z