Efficient in vivo gene expression by trans-splicing adeno-associated viral vectors. - Wikidata - awgldk - TriplyDB

Efficient in vivo gene expression by trans-splicing adeno-associated viral vectors.

Efficient in vivo gene expression by trans-splicing adeno-associated viral vectors.

http://www.wikidata.org/entity/Q36968876

about

In Vivo Delivery Systems for Therapeutic Genome Editing Genome-editing Technologies for Gene and Cell Therapy Gene therapy for cardiovascular disease: advances in vector development, targeting, and delivery for clinical translation Gene therapy for human hearing loss: challenges and promises Gene therapy of inherited retinal degenerations: prospects and challenges Marginal level dystrophin expression improves clinical outcome in a strain of dystrophin/utrophin double knockout mice Advances in gene therapy for muscular dystrophies Animal models of Duchenne muscular dystrophy: from basic mechanisms to gene therapy An intronic LINE-1 element insertion in the dystrophin gene aborts dystrophin expression and results in Duchenne-like muscular dystrophy in the corgi breed Viral transduction of the neonatal brain delivers controllable genetic mosaicism for visualising and manipulating neuronal circuits in vivo.Designer gene delivery vectors: molecular engineering and evolution of adeno-associated viral vectors for enhanced gene transfer Systemic delivery of AAV8 in utero results in gene expression in diaphragm and limb muscle: treatment implications for muscle disorders.Systemic Trans-splicing adeno-associated viral delivery efficiently transduces the heart of adult mdx mouse, a model for duchenne muscular dystrophy Evidence for the failure of adeno-associated virus serotype 5 to package a viral genome > or = 8.2 kb.Full-length dystrophin reconstitution with adeno-associated viral vectors Sarcolemmal nNOS anchoring reveals a qualitative difference between dystrophin and utrophin.The potential of adeno-associated viral vectors for gene delivery to muscle tissue.Adeno-associated virus serotype-9 microdystrophin gene therapy ameliorates electrocardiographic abnormalities in mdx mice.iNOS ablation does not improve specific force of the extensor digitorum longus muscle in dystrophin-deficient mdx4cv mice.Safety of AAV factor IX peripheral transvenular gene delivery to muscle in hemophilia B dogs.Efficient production of dual recombinant adeno-associated viral vectors for factor VIII delivery Efficient transgene reconstitution with hybrid dual AAV vectors carrying the minimized bridging sequences Gene therapy progress and prospects: Duchenne muscular dystrophy.Cystic fibrosis transmembrane conductance regulator with a shortened R domain rescues the intestinal phenotype of CFTR-/- mice.High throughput screening in duchenne muscular dystrophy: from drug discovery to functional genomics Duchenne muscular dystrophy gene therapy: Lost in translation?Natural history of cone disease in the murine model of Leber congenital amaurosis due to CEP290 mutation: determining the timing and expectation of therapy.SERCA2a gene transfer improves electrocardiographic performance in aged mdx mice Novel mini-dystrophin gene dual adeno-associated virus vectors restore neuronal nitric oxide synthase expression at the sarcolemma Manipulation of mtDNA heteroplasmy in all striated muscles of newborn mice by AAV9-mediated delivery of a mitochondria-targeted restriction endonuclease.Hybrid adeno-associated virus bearing nonhomologous inverted terminal repeats enhances dual-vector reconstruction of minigenes in vivo.Gene therapy progress and prospects--vectorology: design and production of expression cassettes in AAV vectors.α2 and α3 helices of dystrophin R16 and R17 frame a microdomain in the α1 helix of dystrophin R17 for neuronal NOS binding.Preservation of muscle force in Mdx3cv mice correlates with low-level expression of a near full-length dystrophin protein Challenges and opportunities in dystrophin-deficient cardiomyopathy gene therapy.Expressing Transgenes That Exceed the Packaging Capacity of Adeno-Associated Virus Capsids.Proximity-dependent and proximity-independent trans-splicing in mammalian cells Synthetic intron improves transduction efficiency of trans-splicing adeno-associated viral vectors.Engineering Factor Viii for Hemophilia Gene Therapy.Progress in gene therapy of dystrophic heart disease.

P2860

Q26748886-E26569AB-20DD-4622-968D-C1155BDA0DDE Q26769662-4D09B9FA-EBE4-478F-A8EF-DD5AF68FA113 Q26799437-092EED89-5B8A-47FD-8574-08D7292670EB Q26996322-9911BFAF-4AA0-42EB-8383-952445E2D510 Q27009261-34077615-58EB-4F75-B90A-F668B4CA1676 Q27312780-33541C5C-C889-4AB7-86AF-271E8DF533AA Q28073994-2AE39C09-D487-4A5A-9B4D-3BF7FD8F72B8 Q28084979-E026DE60-7EFE-44F3-B9A3-A081D6DF64D8 Q30497616-746C946E-A873-448C-B7EA-8A45676573C5 Q30538923-8E0B755B-3963-4A66-A340-21C8157D7A18 Q33296224-13DDB5E1-DA7D-489E-83AE-99160BDB0EFE Q33617271-87EBE761-4D17-491C-B6A8-A4BBB2D0F272 Q33691883-D8E86CC7-C350-4E24-A8BE-37CE0BDE6690 Q33730469-237741E3-3879-4CBB-BF15-1484993A2592 Q33782799-A7962C10-7828-4204-A9C6-96D76E0FC3BB Q33891300-8A84E2FA-C921-433F-A825-635F213CF1AD Q33902499-8BE60414-718F-493D-B629-318603F7E979 Q33925312-2906BD90-55CC-48D6-BD60-57FE2FB7FF99 Q33955159-A622C5FA-707F-46B7-BDEE-E483D0B804A4 Q34025098-FAE56067-ED39-4F7D-BBD0-85266E96CA70 Q34082003-B29E804C-299D-4B7E-B700-2183CD20E4E4 Q34511733-1EF10CE2-64B0-4C28-BF15-902CE94A4A20 Q34576814-6607D372-E6E2-43B0-86B2-1C98940584CB Q34582621-2696FDF5-48D1-4B26-ADB1-94434750F2F5 Q34792390-677136B5-F352-4773-88CF-CDC45F44D3FB Q35052506-C72DBE8B-DE0B-40EA-82BA-A6DB19A0A9A0 Q35132187-59FE8EBC-CFBF-4A64-9263-30A01DCF53FB Q35183186-DB1DD075-4297-4CD6-998D-11B85366D790 Q35678425-A877A67F-3F80-4C8D-A1AD-41EF148D2A69 Q36137186-7E39D1CB-A404-4E7E-B0B2-8FC8ED145351 Q36238762-FD344C6B-5141-4172-B526-57230E7FDC43 Q36384464-18C93505-DC3C-440C-8E2E-EDAF3169D77D Q36535303-2E305D78-3BD8-473B-B3F8-F74B9ADD9FC8 Q36579847-C5402688-CDB8-4A38-B00E-0E7466336CA2 Q36599311-A70A9DA2-B64D-4F34-8BD3-B2FD6045F856 Q36601946-675E6EDD-84DF-4F9A-9DE7-1052332CE98A Q36666307-09819802-FC41-4F5F-B15C-1E3AA493329E Q36728109-82294EF5-2D2E-4F28-970D-7CB60E809B05 Q36737263-A872A0FC-CAD1-4945-996B-862242B8AB3C Q36773242-883FBFF5-BD76-4EB8-8F78-1E848C39DD77

P2860

Efficient in vivo gene expression by trans-splicing adeno-associated viral vectors.

http://www.wikidata.org/entity/Q36968876

description

2005 nî lūn-bûn

@nan

2005年の論文

@ja

2005年論文

@yue

2005年論文

@zh-hant

2005年論文

@zh-hk

2005年論文

@zh-mo

2005年論文

@zh-tw

2005年论文

@wuu

2005年论文

@zh

2005年论文

@zh-cn

name

Efficient in vivo gene expression by trans-splicing adeno-associated viral vectors.

@en

type

label

Efficient in vivo gene expression by trans-splicing adeno-associated viral vectors.

@en

prefLabel

Efficient in vivo gene expression by trans-splicing adeno-associated viral vectors.

@en

P1433

Q36968876-7BB3E2CC-FB96-4B9D-98A3-7A541A32A46D

P1476

Q36968876-F56D33AE-295B-4650-980B-95DEA97E691D

P2093

Q36968876-0F8BBF79-6838-4C07-B6D6-9D2AD6CDB03E

Q36968876-3FFC7E13-C7FA-49DC-872E-123207304494

Q36968876-78955DC2-FA9C-4D79-B3FD-7B2899A8AB02

Q36968876-A177990B-24A1-4F8A-8C37-576F7E2370F6

Q36968876-B0735013-7747-4384-A198-B54CB3F004D7

Q36968876-CE410860-837F-4AE9-8858-4D58B7D5693B

Q36968876-E388A4EC-8186-4A7A-A218-D2C15A692D3D

P2860

Q36968876-0230A1BC-DDE0-4976-9ED0-A063CF91202C

Q36968876-0A091E21-0D03-4F8E-8D36-2399B7CCC8E8

Q36968876-15F01BFA-30D0-4CB3-ADDD-025B0FE7563E

Q36968876-34DA52EF-5ED4-4083-97AF-561EF52B4A54

Q36968876-35492561-D069-4B30-A177-0C5EA3531C2D

Q36968876-37647515-D64A-4EEC-991D-2BFC9834F09F

Q36968876-3AE23820-7033-4BC4-81EE-9EF79F730E6D

Q36968876-41E11FF0-65EE-4379-AEC8-67CDCE7ED17D

Q36968876-43AE0301-F063-4618-89A7-E7F8BEA087E1

Q36968876-4F10488A-C993-4E35-858D-5E9759F04C28

P2888

Q36968876-079FF32C-5BAB-4844-839A-1ED137C36F1B

P304

Q36968876-474D6EC2-84A3-4558-B8BB-CE038A7F7640

P31

Q36968876-B3571662-1DD5-4DBF-A03D-105A475CE9C9

P356

Q36968876-35B5CE17-B808-42BF-BD1B-5552101E23CD

P433

Q36968876-C440D439-4DA5-4F62-8848-C94CADDCF47A

P478

Q36968876-BA741E90-ACBB-4CA6-A49C-4804389062F3

P577

Q36968876-626E0DAA-C656-4F69-B3E5-A29E050C9031

P5875

Q36968876-010D1DA4-3957-4574-B6F4-68BF16236F32

P698

Q36968876-1391B2A6-DDC0-440B-8E52-A92FD19C3741

P932

Q36968876-4AB95C0F-D7C1-405D-8C7B-1CF9BAE3DBFE

P356

P1433

Nature Biotechnology

P1476

Efficient in vivo gene expression by trans-splicing adeno-associated viral vectors.

@en

P2093

Arkasubhra Ghosh

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

Dongsheng Duan

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

Jeffrey S Chamberlain

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

John F Engelhardt

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

Mingju Liu

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

Yi Lai

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

Yongping Yue

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

P2860

Circular intermediates of recombinant adeno-associated virus have defined structural characteristics responsible for long-term episomal persistence in muscle tissue.

Adeno-associated virus type 6 (AAV6) vectors mediate efficient transduction of airway epithelial cells in mouse lungs compared to that of AAV2 vectors

Membrane organization of the dystrophin-glycoprotein complex

Listening to silence and understanding nonsense: exonic mutations that affect splicing

Serine/arginine-rich protein-dependent suppression of exon skipping by exonic splicing enhancers.

Very mild muscular dystrophy associated with the deletion of 46% of dystrophin.

Adeno-associated virus vectors in clinical trials.

Inactivation of the SR protein splicing factor ASF/SF2 results in genomic instability.

Trans-splicing vectors expand the utility of adeno-associated virus for gene therapy.

Selective loss of sarcolemmal nitric oxide synthase in Becker muscular dystrophy

P2888

P304

1435-1439

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

P31

scholarly article

P356

10.1038/NBT1153

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

P433

11

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

P478

23

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

P577

2005-10-09T00:00:00Z

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

P5875

7521313

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

P698

16244658

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

P932

2581721

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