Widespread muscle expression of an AAV9 human mini-dystrophin vector after intravenous injection in neonatal dystrophin-deficient dogs.
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The emerging role of viral vectors as vehicles for DMD gene editingIntra-amniotic rAAV-mediated microdystrophin gene transfer improves canine X-linked muscular dystrophy and may induce immune toleranceCurrent understanding of molecular pathology and treatment of cardiomyopathy in duchenne muscular dystrophyAnimal models of Duchenne muscular dystrophy: from basic mechanisms to gene therapyEffects of systemic multiexon skipping with peptide-conjugated morpholinos in the heart of a dog model of Duchenne muscular dystrophy.Therapy of Genetic Disorders-Novel Therapies for Duchenne Muscular Dystrophy.The golden retriever model of Duchenne muscular dystrophy.Engineering liver-detargeted AAV9 vectors for cardiac and musculoskeletal gene transfer.The potential of adeno-associated viral vectors for gene delivery to muscle tissue.Systemic gene transfer reveals distinctive muscle transduction profile of tyrosine mutant AAV-1, -6, and -9 in neonatal dogs.Pharmacologic management of Duchenne muscular dystrophy: target identification and preclinical trialsSystemic gene delivery in large species for targeting spinal cord, brain, and peripheral tissues for pediatric disordersMammalian models of Duchenne Muscular Dystrophy: pathological characteristics and therapeutic applications.Progress and prospects of gene therapy clinical trials for the muscular dystrophies.Gene replacement therapies for duchenne muscular dystrophy using adeno-associated viral vectors.Phosphorylation within the cysteine-rich region of dystrophin enhances its association with β-dystroglycan and identifies a potential novel therapeutic target for skeletal muscle wasting.Terminal N-linked galactose is the primary receptor for adeno-associated virus 9.Changes in muscle T2 and tissue damage after downhill running in mdx mice.Duchenne muscular dystrophy gene therapy: Lost in translation?Restoring dystrophin expression in duchenne muscular dystrophy muscle progress in exon skipping and stop codon read through.Improving clinical efficacy of adeno associated vectors by rational capsid bioengineering.Long-term systemic myostatin inhibition via liver-targeted gene transfer in golden retriever muscular dystrophy.Duchenne muscular dystrophy gene therapy in the canine model.Signs of progress in gene therapy for muscular dystrophy also warrant cautionSafety and feasibility of high-pressure transvenous limb perfusion with 0.9% saline in human muscular dystrophy.Long-term restoration of cardiac dystrophin expression in golden retriever muscular dystrophy following rAAV6-mediated exon skippingAge-matched comparison reveals early electrocardiography and echocardiography changes in dystrophin-deficient dogs.The AAV vector toolkit: poised at the clinical crossroadsEffect of nuclear factor κB inhibition on serotype 9 adeno-associated viral (AAV9) minidystrophin gene transfer to the mdx mouse.High-Pressure Transvenous Perfusion of the Upper Extremity in Human Muscular Dystrophy: A Safety Study with 0.9% Saline.Human galectin 3 binding protein interacts with recombinant adeno-associated virus type 6Safe and bodywide muscle transduction in young adult Duchenne muscular dystrophy dogs with adeno-associated virus.Successful regional delivery and long-term expression of a dystrophin gene in canine muscular dystrophy: a preclinical model for human therapies.Glycan binding avidity determines the systemic fate of adeno-associated virus type 9.Gene Therapy for Duchenne muscular dystrophy.Microdystrophin ameliorates muscular dystrophy in the canine model of duchenne muscular dystrophyEnhancing the utility of adeno-associated virus gene transfer through inducible tissue-specific expression.Comparative Genomics of X-linked Muscular Dystrophies: The Golden Retriever ModelEngraftment of a galactose receptor footprint onto adeno-associated viral capsids improves transduction efficiency.Delivery of recombinant adeno-associated virus vectors to rat diaphragm muscle via direct intramuscular injection
P2860
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P2860
Widespread muscle expression of an AAV9 human mini-dystrophin vector after intravenous injection in neonatal dystrophin-deficient dogs.
description
2010 nî lūn-bûn
@nan
2010 թուականի Յունիսին հրատարակուած գիտական յօդուած
@hyw
2010 թվականի հունիսին հրատարակված գիտական հոդված
@hy
2010年の論文
@ja
2010年論文
@yue
2010年論文
@zh-hant
2010年論文
@zh-hk
2010年論文
@zh-mo
2010年論文
@zh-tw
2010年论文
@wuu
name
Widespread muscle expression o ...... tal dystrophin-deficient dogs.
@ast
Widespread muscle expression o ...... tal dystrophin-deficient dogs.
@en
Widespread muscle expression o ...... tal dystrophin-deficient dogs.
@nl
type
label
Widespread muscle expression o ...... tal dystrophin-deficient dogs.
@ast
Widespread muscle expression o ...... tal dystrophin-deficient dogs.
@en
Widespread muscle expression o ...... tal dystrophin-deficient dogs.
@nl
prefLabel
Widespread muscle expression o ...... tal dystrophin-deficient dogs.
@ast
Widespread muscle expression o ...... tal dystrophin-deficient dogs.
@en
Widespread muscle expression o ...... tal dystrophin-deficient dogs.
@nl
P2093
P2860
P356
P1433
P1476
Widespread muscle expression o ...... tal dystrophin-deficient dogs.
@en
P2093
Chunlian Chen
Chunping Qiao
Daniel J Bogan
James F Howard
Janet R Bogan
Joe N Kornegay
P2860
P304
P356
10.1038/MT.2010.94
P577
2010-06-01T00:00:00Z