C-terminal-truncated microdystrophin recruits dystrobrevin and syntrophin to the dystrophin-associated glycoprotein complex and reduces muscular dystrophy in symptomatic utrophin/dystrophin double-knockout mice
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Marginal level dystrophin expression improves clinical outcome in a strain of dystrophin/utrophin double knockout miceAdvances in gene therapy for muscular dystrophiesThe Dystrophin Complex: Structure, Function, and Implications for TherapyMicroutrophin delivery through rAAV6 increases lifespan and improves muscle function in dystrophic dystrophin/utrophin-deficient miceAn intronic LINE-1 element insertion in the dystrophin gene aborts dystrophin expression and results in Duchenne-like muscular dystrophy in the corgi breedFunctional deficits in nNOSmu-deficient skeletal muscle: myopathy in nNOS knockout miceBlastocyst injection of wild type embryonic stem cells induces global corrections in mdx mice.The polyproline site in hinge 2 influences the functional capacity of truncated dystrophins.Sarcolemmal nNOS anchoring reveals a qualitative difference between dystrophin and utrophin.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.Neuronal nitric oxide synthase-rescue of dystrophin/utrophin double knockout mice does not require nNOS localization to the cell membraneDystrobrevin increases dystrophin's binding to the dystrophin-glycoprotein complex and provides protection during cardiac stressRescue from respiratory dysfunction by transduction of full-length dystrophin to diaphragm via the peritoneal cavity in utrophin/dystrophin double knockout mice.SERCA2a gene transfer improves electrocardiographic performance in aged mdx miceAAV-microdystrophin therapy improves cardiac performance in aged female mdx mice.Duchenne muscular dystrophy gene therapy in the canine model.Humoral immunity to AAV-6, 8, and 9 in normal and dystrophic dogsAAV micro-dystrophin gene therapy alleviates stress-induced cardiac death but not myocardial fibrosis in >21-m-old mdx mice, an end-stage model of Duchenne muscular dystrophy cardiomyopathy.Membrane-stabilizing copolymers confer marked protection to dystrophic skeletal muscle in vivo.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 proteinChallenges and opportunities in dystrophin-deficient cardiomyopathy gene therapy.Microdystrophin ameliorates muscular dystrophy in the canine model of duchenne muscular dystrophyFrom the smallest virus to the biggest gene: marching towards gene therapy for duchenne muscular dystrophy.Efficient whole-body transduction with trans-splicing adeno-associated viral vectors100-fold but not 50-fold dystrophin overexpression aggravates electrocardiographic defects in the mdx model of Duchenne muscular dystrophy.Dystrophins carrying spectrin-like repeats 16 and 17 anchor nNOS to the sarcolemma and enhance exercise performance in a mouse model of muscular dystrophy.Dual AAV therapy ameliorates exercise-induced muscle injury and functional ischemia in murine models of Duchenne muscular dystrophySub-physiological sarcoglycan expression contributes to compensatory muscle protection in mdx miceCardiac expression of a mini-dystrophin that normalizes skeletal muscle force only partially restores heart function in aged Mdx mice.The function of dog models in developing gene therapy strategies for human health.Nanotherapy for Duchenne muscular dystrophy.Delivery of AAV2/9-microdystrophin genes incorporating helix 1 of the coiled-coil motif in the C-terminal domain of dystrophin improves muscle pathology and restores the level of α1-syntrophin and α-dystrobrevin in skeletal muscles of mdx mice.A Five-Repeat Micro-Dystrophin Gene Ameliorated Dystrophic Phenotype in the Severe DBA/2J-mdx Model of Duchenne Muscular Dystrophy.Dystrophin deficiency compromises force production of the extensor carpi ulnaris muscle in the canine model of Duchenne muscular dystrophy.Inhibition of the IKK/NF-κB pathway by AAV gene transfer improves muscle regeneration in older mdx mice.A canine minidystrophin is functional and therapeutic in mdx mice.Systemic AAV Micro-dystrophin Gene Therapy for Duchenne Muscular Dystrophy
P2860
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P2860
C-terminal-truncated microdystrophin recruits dystrobrevin and syntrophin to the dystrophin-associated glycoprotein complex and reduces muscular dystrophy in symptomatic utrophin/dystrophin double-knockout mice
description
2006 nî lūn-bûn
@nan
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
2006年论文
@zh
2006年论文
@zh-cn
name
C-terminal-truncated microdyst ...... ystrophin double-knockout mice
@en
type
label
C-terminal-truncated microdyst ...... ystrophin double-knockout mice
@en
prefLabel
C-terminal-truncated microdyst ...... ystrophin double-knockout mice
@en
P2860
P1433
P1476
C-terminal-truncated microdyst ...... ystrophin double-knockout mice
@en
P2093
Mingju Liu
Yongping Yue
P2860
P356
10.1016/J.YMTHE.2006.01.007
P577
2006-03-23T00:00:00Z