Prevention of the dystrophic phenotype in dystrophin/utrophin-deficient muscle following adenovirus-mediated transfer of a utrophin minigene

PM Wakefield, JM Tinsley, MJA Wood, R Gilbert… - Gene therapy, 2000 - nature.com
Duchenne muscular dystrophy (DMD) is a progressive muscle wasting disorder caused by
the lack of a subsarcolemmal protein, dystrophin. We have previously shown that the …

Immune Response to Adenovirus-Delivered Antigens Upregulates Utrophin and Results in Mitigation of Muscle Pathology in mdx Mice

K Yamamoto, K Yuasa, Y Miyagoe, Y Hosaka… - Human gene …, 2000 - liebertpub.com
The upregulation of endogenous utrophin in skeletal muscle may lead to a new approach to
the treatment of Duchenne muscular dystrophy (DMD). We found that injection of an E1, E3 …

[HTML][HTML] Successful compensation for dystrophin deficiency by a helper-dependent adenovirus expressing full-length utrophin

JR Deol, G Danialou, N Larochelle, M Bourget… - Molecular Therapy, 2007 - cell.com
Helper-dependent adenovirus vector (AdV)–mediated full-length dystrophin expression
leads to significant mitigation of the dystrophic phenotype of the mdx mouse. However …

Adenovirus-mediated utrophin gene transfer mitigates the dystrophic phenotype of mdx mouse muscles

R Gilbert, J Nalbantoglu, BJ Petrof, S Ebihara… - Human gene …, 1999 - liebertpub.com
Utrophin is a close homolog of dystrophin, the protein whose mutations cause Duchenne
muscular dystrophy (DMD). Utrophin is present at low levels in normal and dystrophic …

Restoration of all dystrophin protein interactions by functional domains in trans does not rescue dystrophy

KL Gardner, JA Kearney, JD Edwards… - Gene therapy, 2006 - nature.com
Rescue of dystrophic skeletal muscle in mdx and utrophin/dystrophin-deficient (dko) mouse
models by reintroduction of dystrophin has validated gene therapy as a potential therapeutic …

Differential effects of dystrophin and utrophin gene transfer in immunocompetent muscular dystrophy (mdx) mice

S Ebihara, GH GUIBINGA, R Gilbert… - Physiological …, 2000 - journals.physiology.org
Ebihara, Satoru, Ghiabe-Henri Guibinga, Renald Gilbert, Josephine Nalbantoglu, Bernard
Massie, George Karpati, and Basil J. Petrof. Differential effects of dystrophin and utrophin …

Systemic human minidystrophin gene transfer improves functions and life span of dystrophin and dystrophin/utrophin‐deficient mice

B Wang, J Li, FH Fu, X Xiao - Journal of Orthopaedic Research, 2009 - Wiley Online Library
Duchenne muscular dystrophy (DMD) is the most common and lethal genetic muscle
disease, caused by mutations in the dystrophin gene. No efficacious treatment is currently …

Gene therapy for muscular dystrophies: current status and future prospects

S Takeda, Y Miyagoe-Suzuki - BioDrugs, 2001 - Springer
Since the identification in 1987 of the gene for Duchenne muscular dystrophy (DMD),
research on the molecular pathogenesis of muscular dystrophy has progressed extensively …

Functional correction of adult mdx mouse muscle using gutted adenoviral vectors expressing full-length dystrophin

C Dellorusso, JM Scott… - Proceedings of the …, 2002 - National Acad Sciences
Duchenne muscular dystrophy is a lethal X-linked recessive disorder caused by mutations in
the dystrophin gene. Delivery of functionally effective levels of dystrophin to …

[HTML][HTML] Transduction of full-length dystrophin to multiple skeletal muscles improves motor performance and life span in utrophin/dystrophin double knockout mice

R Kawano, M Ishizaki, Y Maeda, Y Uchida, E Kimura… - Molecular Therapy, 2008 - cell.com
Duchenne muscular dystrophy (DMD) is a fatal, progressive, muscle-wasting disease
caused by defects in the dystrophin. No viral vector except the helper-dependent adenovirus …