Improvement in survival and muscle function in an mdx/utrn−/− double mutant mouse using a human retinal dystrophin transgene

Gaedigk, Roger; Law, Douglas; Gustafson, Kathleen M.; McNulty, Steven G.; Nsumu, Ndona N.; Modrcin, Ann; Rinaldi, Robert; Pinson, David M.; Fowler, Stephen C.; Bilgen, Mehmet; Burns, Joanne; Hauschka, Stephen D.; White, Robert A.

doi:10.1016/j.nmd.2005.12.007

Cited by 12 publications

(10 citation statements)

References 53 publications

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“…In patients and animal models of MD, increased T 2 values have been shown to correlate with increased fat (31, 32) and damaged fibers (33), and to decrease following restoration of the missing dystrophin‐associated glycoprotein in an animal model for MD (33). MRI has also been used to monitor a decrease in kyphosis, or dorsal spinal curvature, following gene therapy for DMD (34) and skeletal muscle regeneration following crush injury (35). As a result, MRI may be useful for tracking locally delivered myogenic stem cells for the treatment of muscle disease and also for indirectly monitoring the progress of the stem cells by serial imaging following stem cell injection.…”

Section: Discussionmentioning

confidence: 99%

14.1 T whole body MRI for detection of mesoangioblast stem cells in a murine model of duchenne muscular dystrophy

Odintsov

Chun

Mulligan

et al. 2011

Magnetic Resonance in Med

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Noninvasive imaging procedures will be important for stem cell therapy for muscular dystrophy (MD). Mesoangioblasts regenerate muscle in animal models of muscular dystrophy. In this study, superparamagnetic iron oxide nanoparticles were used to visualize mesoangioblasts in vivo with MRI. Mesoangioblasts incorporated superparamagnetic iron oxide without transfection reagents, and cell differentiation was not negatively impacted. A custom-built radiofrequency coil with an adjustable field of view and 14.1 T magnet were used for whole-body MRI of mice. High-resolution images of mesoangioblasts in skeletal and cardiac muscle of Mdx mice were obtained following local delivery. Labeled cells were verified by Prussian blue staining and dystrophin expression, indicating that the wild-type mesoangioblasts survived and differentiated in muscle. Iron-labeled cells were detected with MRI in vivo 6 months following intracardiac injection but were determined to be activated macrophages. Iron-labeled cells were not detected by MRI following systemic delivery but were present in skeletal and cardiac muscle, visualized by Prussian blue staining. Systemically delivered mesoangioblasts were detected in lungs by Prussian blue staining and DiI but not by MRI in our study. MRI may be useful for short-term tracking of mesoangioblasts delivered locally but not for long-term monitoring or detection after systemic delivery. Magn Reson Med 66:1704-1714,

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Section: Discussionmentioning

confidence: 99%

14.1 T whole body MRI for detection of mesoangioblast stem cells in a murine model of duchenne muscular dystrophy

Odintsov

Chun

Mulligan

et al. 2011

Magnetic Resonance in Med

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“…Previous reports on mdx:utr Ϫ/Ϫ mouse life span is highly variable, ranging from as little as 4 wk to as many as 36 wk (5,8,10,13,15,17,24). This variability may reflect genetic drift within respective colonies and local environmental factors idiosyncratic to each housing facility (e.g., staff, cage sizes, husbandry, handling, food enrichment options, room noise, architecture, temperature, etc.).…”

Section: Discussionmentioning

confidence: 99%

TAT-μUtrophin mitigates the pathophysiology of dystrophin and utrophin double-knockout mice

Call

Ervasti

Lowe³

2011

Journal of Applied Physiology

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“…With the exception of Dp40 ( Fujimoto et al, 2014 ), they all contain the CT and CR domains but are missing the NT actin-binding domain. To determine whether these miniature isoforms are therapeutically relevant, the Chamberlain lab, as well as others, made transgenic mdx mice for Dp260, Dp116 and Dp71 (see supplementary material Table S1 for details) ( Cox et al, 1994 ; Gaedigk et al, 2006 ; Greenberg et al, 1994 ; Judge et al, 2011 ; Judge et al, 2006 ; Warner et al, 2002 ).…”

Section: Establishing the Foundations Of Gene Therapy: Transgenic mentioning

confidence: 99%

“…Two independent strains of Dp260 transgenic mice have been studied ( Gaedigk et al, 2006 ; Warner et al, 2002 ). Although Dp260 (also known as the retinal isoform of dystrophin) does not carry the NT domain, it contains the ABD2 domain ( Fig.…”

Section: Establishing the Foundations Of Gene Therapy: Transgenic mentioning

confidence: 99%

Animal models of Duchenne muscular dystrophy: from basic mechanisms to gene therapy

McGreevy

Hakim

McIntosh

et al. 2015

Disease Models &Amp; Mechanisms

403

422

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Duchenne muscular dystrophy (DMD) is a progressive muscle-wasting disorder. It is caused by loss-of-function mutations in the dystrophin gene. Currently, there is no cure. A highly promising therapeutic strategy is to replace or repair the defective dystrophin gene by gene therapy. Numerous animal models of DMD have been developed over the last 30 years, ranging from invertebrate to large mammalian models. mdx mice are the most commonly employed models in DMD research and have been used to lay the groundwork for DMD gene therapy. After ~30 years of development, the field has reached the stage at which the results in mdx mice can be validated and scaled-up in symptomatic large animals. The canine DMD (cDMD) model will be excellent for these studies. In this article, we review the animal models for DMD, the pros and cons of each model system, and the history and progress of preclinical DMD gene therapy research in the animal models. We also discuss the current and emerging challenges in this field and ways to address these challenges using animal models, in particular cDMD dogs.

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Improvement in survival and muscle function in an mdx/utrn−/− double mutant mouse using a human retinal dystrophin transgene

Cited by 12 publications

References 53 publications

14.1 T whole body MRI for detection of mesoangioblast stem cells in a murine model of duchenne muscular dystrophy

14.1 T whole body MRI for detection of mesoangioblast stem cells in a murine model of duchenne muscular dystrophy

TAT-μUtrophin mitigates the pathophysiology of dystrophin and utrophin double-knockout mice

Animal models of Duchenne muscular dystrophy: from basic mechanisms to gene therapy

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