AAV genome loss from dystrophic mouse muscles during AAV-U7 snRNA-mediated exon-skipping therapy.
about
Delivery is key: lessons learnt from developing splice-switching antisense therapiesTherapy of Genetic Disorders-Novel Therapies for Duchenne Muscular Dystrophy.Gene therapy prolongs survival and restores function in murine and canine models of myotubular myopathy.Forelimb treatment in a large cohort of dystrophic dogs supports delivery of a recombinant AAV for exon skipping in Duchenne patients.Effects of recombinant adeno-associated virus-mediated CD151 gene transfer on the expression of rat vascular endothelial growth factor in ischemic myocardium.Improvement of SMN2 pre-mRNA processing mediated by exon-specific U1 small nuclear RNA.Could exon skipping help dystrophic boys to run, hop, and jump?Short-lived recombinant adeno-associated virus transgene expression in dystrophic muscle is associated with oxidative damage to transgene mRNA.Perspective on Adeno-Associated Virus Capsid Modification for Duchenne Muscular Dystrophy Gene TherapyIn vivo gene editing in dystrophic mouse muscle and muscle stem cells.Reprogramming the Dynamin 2 mRNA by Spliceosome-mediated RNA Trans-splicing.The Dynamics of Compound, Transcript, and Protein Effects After Treatment With 2OMePS Antisense Oligonucleotides in mdx Mice.Viral Vector-Mediated Antisense Therapy for Genetic Diseases.Advances in genetic therapeutic strategies for Duchenne muscular dystrophy.Targeting Splicing in the Treatment of Human DiseaseSpecific targeting of TGF-β family ligands demonstrates distinct roles in the regulation of muscle mass in health and disease.Antisense pre-treatment increases gene therapy efficacy in dystrophic muscles.Simple downstream process based on detergent treatment improves yield and in vivo transduction efficacy of adeno-associated virus vectors.Human α7 Integrin Gene (ITGA7) Delivered by Adeno-Associated Virus Extends Survival of Severely Affected Dystrophin/Utrophin-Deficient MiceCorrection of the Exon 2 Duplication in DMD Myoblasts by a Single CRISPR/Cas9 System.Glycosaminoglycan modifications in Duchenne muscular dystrophy: specific remodeling of chondroitin sulfate/dermatan sulfate.Single-cut genome editing restores dystrophin expression in a new mouse model of muscular dystrophy.Allele-specific silencing therapy for Dynamin 2-related dominant centronuclear myopathy.Combined Therapies for Duchenne Muscular Dystrophy to Optimize Treatment Efficacy.Systemic AAV Micro-dystrophin Gene Therapy for Duchenne Muscular Dystrophy
P2860
Q33625875-F65F9ED6-26D2-4C4B-80C3-2558BE49680DQ33678893-7C9E8BD3-27B1-4D86-95C6-EA4B47C8CDDDQ33926944-9F6419F5-D9F8-413F-BBF0-1D1F5FD831F2Q34041966-7F12E084-C6CA-422A-BDF2-E28C8983384AQ34585403-7B366602-3E4B-4738-B361-D198C3ADFEB0Q34902920-B2EE6532-B322-465F-816A-C81F41E74BFDQ35600455-55D5031C-F2EE-4CBD-A788-8CF660369ACAQ35651108-D55CC205-FAF3-4D66-BFFB-1C8DAC489FF8Q36406869-6B1B7FD2-07B3-42B9-8C57-80DE04B29254Q37045850-FFAFBF3E-5863-4FC4-A160-78C81B1B8DECQ37325074-9B43B197-BD23-4D8F-BC3B-6505943734B7Q38257312-A2BB7241-9FB3-46EA-8DEA-34A09CCF7485Q38289261-CB5DE92C-437E-43B9-BE15-B5EB656FBF8EQ38542416-D4F1543C-AD85-4D19-AEC8-E249481389F5Q39154733-18F743F6-A562-4F3F-93C6-C33D45933F28Q40162611-71B06A68-018E-4383-B586-48EA6FC6F816Q40667121-BA6D6767-8D4B-4157-8D9B-D0857648062FQ41551945-C3E2336A-0A8D-40EE-AF22-DA329638ED5BQ41668084-8D161C05-8558-41E2-8A2D-4500919B472BQ42094546-626B6A88-0F4A-4D20-B5E2-A24150DD0464Q42670912-A8B47816-0AD2-4D0E-BDC3-0499834B0B01Q47153293-99828506-CB7D-4943-A29E-F65AE61716DAQ47778748-37FAFD8E-4435-4206-8536-DDF209B04262Q52564618-F03C30EA-5CD5-499D-9D55-011B71DF9C8CQ57039821-CE4AD1D2-6955-4551-8B59-4831F1C24062
P2860
AAV genome loss from dystrophic mouse muscles during AAV-U7 snRNA-mediated exon-skipping therapy.
description
2013 nî lūn-bûn
@nan
2013年の論文
@ja
2013年論文
@yue
2013年論文
@zh-hant
2013年論文
@zh-hk
2013年論文
@zh-mo
2013年論文
@zh-tw
2013年论文
@wuu
2013年论文
@zh
2013年论文
@zh-cn
name
AAV genome loss from dystrophi ...... ediated exon-skipping therapy.
@en
AAV genome loss from dystrophi ...... ediated exon-skipping therapy.
@nl
type
label
AAV genome loss from dystrophi ...... ediated exon-skipping therapy.
@en
AAV genome loss from dystrophi ...... ediated exon-skipping therapy.
@nl
prefLabel
AAV genome loss from dystrophi ...... ediated exon-skipping therapy.
@en
AAV genome loss from dystrophi ...... ediated exon-skipping therapy.
@nl
P2093
P2860
P50
P356
P1433
P1476
AAV genome loss from dystrophi ...... mediated exon-skipping therapy
@en
P2093
Guillaume Précigout
Kay E Davies
Luis Garcia
Maëva Le Hir
Thomas Voit
P2860
P304
P356
10.1038/MT.2013.121
P577
2013-06-11T00:00:00Z