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rAAV6-microdystrophin preserves muscle function and extends lifespan in severely dystrophic mice

Article Abstract:

Mice carrying mutations in both the dystrophin and utrophin genes die prematurely due to severe muscular dystrophy. Investigations in this area have revealed that intravascular administration of re-combinant adeno-associated viral (rAAV) vectors carrying a microdystrophin gene restores expression of dystrophin in the respiratory, cardiac and limb musculature of these mice, reducing skeletal muscle pathology, and extending lifespan.

Author: Meuse, Leonard, Finn, Eric, Chamberlain, Jeffrey S., Gregorevic, Paul, Blankinship, Michael J., Allen, James M., Murry, Charles E., Minami, Elina, Haraguchi, Miki, Adams, Marvin E., Forehner, Stanley C.
Publisher: Nature America, Inc.
Publication Name: Nature Medicine
Subject: Health
ISSN: 1078-8956
Year: 2006
Health aspects, Muscular dystrophy, Dystrophin, Utrophin

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Systemic delivery of genes to striated muscle using adeno-associated viral vectors

Article Abstract:

A simple and highly efficient method to transfer genes systemically to the cardiac and skeletal muscles of adult mammals is presented. This approach is used to administer the recombiant adeno-associated virus pseudotype 6, which is extremely effective at transducing skeletal muscles after intramuscular injection.

Author: Meuse, Leonard, Miller, Daniel G., Chamberlain, Jeffrey S., Russell, David W., Gregorevic, Paul, Blankinship, Michael J., Allen, James M., Crawford, Robert W.
Publisher: Nature America, Inc.
Publication Name: Nature Medicine
Subject: Health
ISSN: 1078-8956
Year: 2004
Muscles, Mammals, Genetic research, Skeletal muscle

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Successful treatment of canine leukocyte adhesion deficiency by foamy virus vectors

Article Abstract:

The article discusses the use of foamy virus vectors in treating canine leukocyte adhesion deficiency (CLAD) in dogs. The results show that these foamy vectors could also be used for the treatment of human hematopoietic diseases.

Author: Russell, David W., Allen, James M., Bauer, Thomas R., Jr., Hai, Mehreen, Tuschong, Laura M., Khan, Iram F., Olson, Erik M., Adler, Rima L., Burkholder, Tanya H., Yu-Chen Gu, Hickstein, Dennis D.
Publisher: Nature America, Inc.
Publication Name: Nature Medicine
Subject: Health
ISSN: 1078-8956
Year: 2008
Care and treatment, Gene therapy, Hematopoietic stem cells, Cell adhesion, Immunodeficiency, Leukocytes, White blood cells

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Subjects list: Genetic aspects, Research
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