Body-wide gene therapy of Duchenne muscular dystrophy in the mdx mouse model. - Wikidata - awgldk - TriplyDB

Body-wide gene therapy of Duchenne muscular dystrophy in the mdx mouse model.

Body-wide gene therapy of Duchenne muscular dystrophy in the mdx mouse model.

http://www.wikidata.org/entity/Q34574529

about

miR-31 modulates dystrophin expression: new implications for Duchenne muscular dystrophy therapy A long noncoding RNA controls muscle differentiation by functioning as a competing endogenous RNA Inhibitory control over Ca(2+) sparks via mechanosensitive channels is disrupted in dystrophin deficient muscle but restored by mini-dystrophin expression Animal models of Duchenne muscular dystrophy: from basic mechanisms to gene therapy AAV-mediated intramuscular delivery of myotubularin corrects the myotubular myopathy phenotype in targeted murine muscle and suggests a function in plasma membrane homeostasis RNA and disease L-type Ca2+ channel function is linked to dystrophin expression in mammalian muscle Long-term improvement in mdx cardiomyopathy after therapy with peptide-conjugated morpholino oligomers.Exon exchange approach to repair Duchenne dystrophin transcripts Delivery is key: lessons learnt from developing splice-switching antisense therapies Cationic PMMA nanoparticles bind and deliver antisense oligoribonucleotides allowing restoration of dystrophin expression in the mdx mouse Gene and cell-mediated therapies for muscular dystrophy.Gene therapy progress and prospects: Duchenne muscular dystrophy.Therapeutic approaches to muscular dystrophy.Intratumoral decorin gene delivery by AAV vector inhibits brain glioblastomas and prolongs survival of animals by inducing cell differentiation.Relationships of thigh muscle contractile and non-contractile tissue with function, strength, and age in boys with Duchenne muscular dystrophy.Long-term systemic myostatin inhibition via liver-targeted gene transfer in golden retriever muscular dystrophy.Long-term restoration of cardiac dystrophin expression in golden retriever muscular dystrophy following rAAV6-mediated exon skipping Viral-mediated gene therapy for the muscular dystrophies: successes, limitations and recent advances Approaching a new age in Duchenne muscular dystrophy treatment.Human galectin 3 binding protein interacts with recombinant adeno-associated virus type 6 Molecular and cellular mechanisms of skeletal muscle atrophy: an update.Targeting RNA splicing for disease therapy.Efficient whole-body transduction with trans-splicing adeno-associated viral vectors Systemic microdystrophin gene delivery improves skeletal muscle structure and function in old dystrophic mdx mice.AAV vectors for RNA-based modulation of gene expression.Gene doping: the hype and the reality.A single intravenous injection of adeno-associated virus serotype-9 leads to whole body skeletal muscle transduction in dogs.Designing heart performance by gene transfer.Gene therapy for muscular dystrophy: current progress and future prospects.Progress in therapeutic antisense applications for neuromuscular disorders.AAV-directed muscular dystrophy gene therapy.Skeletal muscle fibre diversity and the underlying mechanisms.Physiological and tissue-specific vectors for treatment of inherited diseases.Opportunities and challenges for the development of antisense treatment in neuromuscular disorders.Viral Vector-Mediated Antisense Therapy for Genetic Diseases.Exon skipping and duchenne muscular dystrophy therapy: selection of the most active U1 snRNA antisense able to induce dystrophin exon 51 skipping.Functional and Morphological Improvement of Dystrophic Muscle by Interleukin 6 Receptor Blockade.Exon 45 skipping through U1-snRNA antisense molecules recovers the Dys-nNOS pathway and muscle differentiation in human DMD myoblasts.Double-target Antisense U1snRNAs Correct Mis-splicing Due to c.639+861C>T and c.639+919G>A GLA Deep Intronic Mutations.

P2860

Q24608004-119067BE-A113-4DE8-9F69-A8B4B391DF59 Q24630341-BA190F78-43B7-4A0C-9A52-3246730C43FE Q27438151-9B75EB79-7D7E-4629-B019-7C92F63E74D0 Q28084979-43869DE5-CC77-4201-BD57-5E3C2E49BC57 Q28508977-02C040E9-9954-459D-B15A-F2E117335B65 Q29615183-F4355620-A7DE-4E6B-A7A2-CF4762D8FB94 Q33339391-8BA38608-E43F-4126-9A14-0D63E196C772 Q33573648-40AA06A3-DBE3-402A-8976-8F70A34B3211 Q33598293-C79417B6-7319-49C1-90B6-883D7EDD4BE5 Q33625875-B18B9DEA-4930-46EF-B15D-317854431B0E Q33713471-966127CE-8CBE-4F1C-8C96-7CEFE1382FFE Q33831193-57D11AA0-CC7D-417A-8619-B783886C7E92 Q34576814-B3DAA970-15F1-4C0C-8064-F8D9A16ADFEC Q34982869-319A80FF-3242-4D3B-90CF-A8759CD328F4 Q35119851-A4491F86-FDA9-48FD-86E0-87D483D9B93C Q35549208-C2415042-5E02-4FF2-B53E-420D6837BFD0 Q35612576-7B41CB31-1780-4230-9F30-F90995166A8D Q35802092-632FC993-E476-478C-A428-D5BB9A7AEB41 Q35847178-D2D944F0-40E3-4786-990A-80750F592FCA Q35888398-513D2E2C-B9A2-4F19-BE5F-60C49D5E2924 Q36086772-8B182284-B548-4114-89C4-CCD735821193 Q36179105-869D275E-E94B-4C2F-AEB2-28FC7EE064D5 Q36780738-6515EC86-5D62-4583-BF63-01F7E3B8F323 Q36968872-849597EB-0C15-4773-A28A-9B7A98ED3643 Q37116446-623244BA-9687-45A5-A00A-540FF52E9AE4 Q37140645-06CDA1C6-986B-42EB-96A2-FC0317C9C16A Q37171838-8CD31D6A-8BBF-42A5-A24C-ED6DBC326C70 Q37245439-63538D61-80E0-465E-844E-6C894B38E96A Q37299072-5E682123-54FD-4071-8EEE-3F9547E3B982 Q37519650-CBFD958F-4D74-4335-A074-2433D4FB12C7 Q37609948-A6944F9C-95F0-43D1-BEFB-9D4238721328 Q37687423-8D95B513-7A80-4A6F-A30F-913160780B40 Q37719612-F022878F-26ED-4843-A18A-4C165CE47621 Q37801839-DCB2D2AE-464A-4EDB-9033-B6759A2887F3 Q37867701-5D29E6FF-CF21-47FF-97E8-2A99CB07BF74 Q38289261-807A6112-C2BB-40E1-AB91-F4E061FF310A Q39692533-DCC121DA-E5A0-4AB3-868E-E862C195B3ED Q40773636-4A3B4CBD-B547-487C-9398-38884F4EC04C Q41136576-D8FC3234-3342-441B-A3D1-DC2822E00A86 Q41671504-F48E8F71-988A-407B-93ED-5719D4120158

P2860

Body-wide gene therapy of Duchenne muscular dystrophy in the mdx mouse model.

http://www.wikidata.org/entity/Q34574529

description

2006 nî lūn-bûn

@nan

2006 թուականի Փետրուարին հրատարակուած գիտական յօդուած

@hyw

2006 թվականի փետրվարին հրատարակված գիտական հոդված

@hy

2006年の論文

@ja

2006年論文

@yue

2006年論文

@zh-hant

2006年論文

@zh-hk

2006年論文

@zh-mo

2006年論文

@zh-tw

2006年论文

@wuu

name

Body-wide gene therapy of Duchenne muscular dystrophy in the mdx mouse model.

@ast

Body-wide gene therapy of Duchenne muscular dystrophy in the mdx mouse model.

@en

Body-wide gene therapy of Duchenne muscular dystrophy in the mdx mouse model.

@nl

type

label

Body-wide gene therapy of Duchenne muscular dystrophy in the mdx mouse model.

@ast

Body-wide gene therapy of Duchenne muscular dystrophy in the mdx mouse model.

@en

Body-wide gene therapy of Duchenne muscular dystrophy in the mdx mouse model.

@nl

prefLabel

Body-wide gene therapy of Duchenne muscular dystrophy in the mdx mouse model.

@ast

Body-wide gene therapy of Duchenne muscular dystrophy in the mdx mouse model.

@en

Body-wide gene therapy of Duchenne muscular dystrophy in the mdx mouse model.

@nl

P1433

Q34574529-5D494627-0C22-4C7C-B4C6-BAB68916A857

P1476

Q34574529-0F08AA15-3200-40E8-AC11-2D9A0632F292

P2093

Q34574529-2CD9FCA1-8613-4314-B076-5BBBFAA9BB16

Q34574529-3A2B9BD2-C46D-4390-8FB2-93A68D0D3784

Q34574529-4237DD9E-FC9B-480D-8E75-B4D0DC8116C7

Q34574529-4F60CA02-40D7-4400-8DBC-2C6338B3FCAC

Q34574529-7FE11EAF-A57F-4F27-B684-3D85D5A35790

Q34574529-ABDA4E78-BFAA-49C1-9F0E-9BB6394BBB22

P2860

Q34574529-10A99D7A-8653-466D-B834-96994F4B5520

Q34574529-12E6A914-2B40-46EB-A490-FD57559935A0

Q34574529-18AC4C2D-4D39-4061-9B19-550908C7D769

Q34574529-1C1CACA6-8F02-42D9-89B6-CB69F531AE66

Q34574529-3784C8B6-4650-440A-853D-7EBA26E5C25A

Q34574529-3CEB7486-0CCA-41D8-8769-C2FAC2B1B56E

Q34574529-40471A7E-2634-4296-8895-BE75D7303005

Q34574529-42A8D544-3759-496B-ACBC-9ADA9F391B2F

Q34574529-48C7B984-0B53-458B-99A0-8D1C9422BAF1

Q34574529-65520AA1-C37B-4255-AF1D-872683DC6612

P304

Q34574529-C985D815-DC46-4DD9-B0E2-ED35518D6056

P31

Q34574529-1EC3A4F9-E2D3-4C8A-9235-BC240FF71208

P356

Q34574529-6EA87568-E9D9-4CCC-BE5D-7ADC906455A1

P407

Q34574529-0D2A492A-EE65-4F8B-9ACA-8E42D022FEA1

P433

Q34574529-2EE21714-A6A2-4C78-A283-32DF271B319C

P478

Q34574529-F70C705C-C361-442F-B8E8-9457E7A76987

P50

Q34574529-3217FBE8-C2B3-4B09-9EBD-2DEA5647C028

Q34574529-39D7AEB7-998E-4354-8AD3-DEB175203579

Q34574529-5F640A7F-F90D-4E35-973D-0A87E2948F5D

Q34574529-683CDF81-06C6-4C40-A2CE-F70B485DC37C

Q34574529-82FF94E8-67F8-4939-ADD1-59F27E696049

Q34574529-A3F37A40-2646-4F65-B713-57D4443533AC

Q34574529-AC0CDCD3-2015-4213-9B07-18AC23717AED

P577

Q34574529-6FEC1AB6-9635-48C2-8CD3-AF4EA37DDBE7

P5875

Q34574529-BC1F194D-808E-4D8D-B7A4-AADA33FE7FEF

P698

Q34574529-5F21733B-C229-412F-86DD-F91F985865F9

P921

Q34574529-6966D3EE-C505-4E50-9952-D932A014C8ED

P932

Q34574529-ADC0F560-2076-4D45-9540-1AD312C3EE65

P356

PNAS.0508917103

P1433

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

P1476

Body-wide gene therapy of Duchenne muscular dystrophy in the mdx mouse model

@en

P2093

Fernanda Gabriella De Angelis

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

Irene Bozzoni

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

Mariacarmela Allocca

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

Olga Sthandier

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

Orietta Pansarasa

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

Roberto Bottinelli

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

P2860

U7 snRNAs induce correction of mutated dystrophin pre-mRNA by exon skipping

Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping

Cell Therapy of -Sarcoglycan Null Dystrophic Mice Through Intra-Arterial Delivery of Mesoangioblasts

Isolation of highly infectious and pure adeno-associated virus type 2 vectors with a single-step gravity-flow column.

Systemic delivery of antisense oligoribonucleotide restores dystrophin expression in body-wide skeletal muscles

Chimeric snRNA molecules carrying antisense sequences against the splice junctions of exon 51 of the dystrophin pre-mRNA induce exon skipping and restoration of a dystrophin synthesis in Delta 48-50 DMD cells

Functional correction of adult mdx mouse muscle using gutted adenoviral vectors expressing full-length dystrophin.

Advances in Duchenne muscular dystrophy gene therapy.

Systemic delivery of genes to striated muscles using adeno-associated viral vectors.

Modification of splicing in the dystrophin gene in cultured Mdx muscle cells by antisense oligoribonucleotides.

P304

3758-3763

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

P31

scholarly article

P356

10.1073/PNAS.0508917103

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

P407

P433

10

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

P478

103

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

P50

Michela A. Denti

Alessandro Rosa

Valeria Parente

Giuseppe D'Antona

Alberto Auricchio

Carmine Nicoletti

P577

2006-02-24T00:00:00Z

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

P5875

7276609

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

P698

16501048

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

P921

P932

1450150

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