Gene therapy progress and prospects: Duchenne muscular dystrophy.
about
A highly stable and nonintegrated human artificial chromosome (HAC) containing the 2.4 Mb entire human dystrophin geneRescue of dystrophic skeletal muscle by PGC-1α involves a fast to slow fiber type shift in the mdx mouseNanopolymers improve delivery of exon skipping oligonucleotides and concomitant dystrophin expression in skeletal muscle of mdx mice.Femtosecond laser treatment enhances DNA transfection efficiency in vivoLong-term improvement in mdx cardiomyopathy after therapy with peptide-conjugated morpholino oligomers.Therapeutic effects of long-circulating miR-135a-containing cationic immunoliposomes against gallbladder carcinoma.One-year treatment of morpholino antisense oligomer improves skeletal and cardiac muscle functions in dystrophic mdx mice.Understanding hereditary diseases using the dog and human as companion model systems.A translational approach for limb vascular delivery of the micro-dystrophin gene without high volume or high pressure for treatment of Duchenne muscular dystrophyPolyethylenimine-modified pluronics (PCMs) improve morpholino oligomer delivery in cell culture and dystrophic mdx mice.Engraftment of ES-Derived Myogenic Progenitors in a Severe Mouse Model of Muscular Dystrophy.WL-276, an antagonist against Bcl-2 proteins, overcomes drug resistance and suppresses prostate tumor growth.Effective rescue of dystrophin improves cardiac function in dystrophin-deficient mice by a modified morpholino oligomer.The pharmacology of regenerative medicineSustained dystrophin expression induced by peptide-conjugated morpholino oligomers in the muscles of mdx mice.Poly(ester amine) Composed of Polyethylenimine and Pluronic Enhance Delivery of Antisense Oligonucleotides In Vitro and in Dystrophic mdx Mice.Cardiac expression of a mini-dystrophin that normalizes skeletal muscle force only partially restores heart function in aged Mdx mice.Gene therapy in large animal models of muscular dystrophy.AAV-directed muscular dystrophy gene therapy.Efficient expression of vascular endothelial growth factor using minicircle DNA for angiogenic gene therapy.Atelocollagen-mediated local and systemic applications of myostatin-targeting siRNA increase skeletal muscle mass.Thermodynamic stability, unfolding kinetics, and aggregation of the N-terminal actin-binding domains of utrophin and dystrophin.Ex vivo correction of selenoprotein N deficiency in rigid spine muscular dystrophy caused by a mutation in the selenocysteine codon.Capsid-modified adenoviral vectors for improved muscle-directed gene therapyDose-dependent restoration of dystrophin expression in cardiac muscle of dystrophic mice by systemically delivered morpholino.A non-viral vector for potential DMD gene therapy study by targeting a minidystrophin-GFP fusion gene into the hrDNA locus.Tween 85-Modified Low Molecular Weight PEI Enhances Exon-Skipping of Antisense Morpholino Oligomer In Vitro and in mdx Mice.Challenges of biomarkers in drug discovery and development.
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P2860
Gene therapy progress and prospects: Duchenne muscular dystrophy.
description
2006 nî lūn-bûn
@nan
2006 թուականի Հոկտեմբերին հրատարակուած գիտական յօդուած
@hyw
2006 թվականի հոտեմբերին 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
Gene therapy progress and prospects: Duchenne muscular dystrophy.
@ast
Gene therapy progress and prospects: Duchenne muscular dystrophy.
@en
Gene therapy progress and prospects: Duchenne muscular dystrophy.
@nl
type
label
Gene therapy progress and prospects: Duchenne muscular dystrophy.
@ast
Gene therapy progress and prospects: Duchenne muscular dystrophy.
@en
Gene therapy progress and prospects: Duchenne muscular dystrophy.
@nl
prefLabel
Gene therapy progress and prospects: Duchenne muscular dystrophy.
@ast
Gene therapy progress and prospects: Duchenne muscular dystrophy.
@en
Gene therapy progress and prospects: Duchenne muscular dystrophy.
@nl
P2093
P2860
P356
P1433
P1476
Gene therapy progress and prospects: Duchenne muscular dystrophy.
@en
P2093
P2860
P2888
P304
P356
10.1038/SJ.GT.3302877
P577
2006-10-26T00:00:00Z