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

JW McGreevy, CH Hakim… - Disease models & …, 2015 - journals.biologists.com
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 …

Function and genetics of dystrophin and dystrophin-related proteins in muscle

DJ Blake, A Weir, SE Newey… - Physiological …, 2002 - journals.physiology.org
The X-linked muscle-wasting disease Duchenne muscular dystrophy is caused by mutations
in the gene encoding dystrophin. There is currently no effective treatment for the disease; …

[图书][B] Biomedical engineering handbook 2

JD Bronzino - 2000 - books.google.com
As we enter the new millennium, the prospects for the field of Biomedical Engineering are
bright. Individuals interested in pursuing careers in this field continue to increase and the …

Heterologous protection against influenza by injection of DNA encoding a viral protein

JB Ulmer, JJ Donnelly, SE Parker, GH Rhodes… - Science, 1993 - science.org
Cytotoxic T lymphocytes (CTLs) specific for conserved viral antigens can respond to different
strains of virus, in contrast to antibodies, which are generally strain-specific. The generation …

A role for the dystrophin-glycoprotein complex as a transmembrane linker between laminin and actin

JM Ervasti, KP Campbell - The Journal of cell biology, 1993 - rupress.org
The dystrophin-glycoprotein complex was tested for interaction with several components of
the extracellular matrix as well as actin. The 156-kD dystrophin-associated glycoprotein (156 …

[HTML][HTML] Dystrophin expression in the mdx mouse restored by stem cell transplantation

E Gussoni, Y Soneoka, CD Strickland, EA Buzney… - Nature, 1999 - nature.com
The development of cell or gene therapies for diseases involving cells that are widely
distributed throughout the body has been severely hampered by the inability to achieve the …

The structural and functional diversity of dystrophin

AH Ahn, LM Kunkel - Nature genetics, 1993 - nature.com
Duchenne and Becker muscular dystrophies are caused by defects of the dystrophin gene.
Expression of this large X-linked gene is under elaborate transcriptional and splicing control …

Adeno-associated virus vector carrying human minidystrophin genes effectively ameliorates muscular dystrophy in mdx mouse model

B Wang, J Li, X Xiao - … of the National Academy of Sciences, 2000 - National Acad Sciences
Duchenne muscular dystrophy (DMD) is the most common and lethal genetic muscle
disorder, caused by recessive mutations in the dystrophin gene. One of every 3,500 males …

A brief history of gene therapy

T Friedmann - Nature genetics, 1992 - nature.com
The concepts of gene therapy arose initially during the 1960s and early 1970s whilst the
development of genetically marked cells lines and the clarification of mechanisms of cell …

Long-term persistence of plasmid DNA and foreign gone expression in mouse muscle

JA Wolff, JJ Ludtke, G Acsadi… - Human molecular …, 1992 - academic.oup.com
Plasmid pRSVL persisted and expressed luciferase for at least 19 months in mouse skeletal
muscle after intramuscular Injection. Other injected plasmids also stably expressed long …