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

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 immunocompetent, adult mdx (dystrophin-deficient) mice has been challenging because of the size of the gene, immune responses aga...

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Vydáno v:Proc Natl Acad Sci U S A
Hlavní autoři: DelloRusso, Christiana, Scott, Jeannine M., Hartigan-O'Connor, Dennis, Salvatori, Giovanni, Barjot, Catherine, Robinson, Ann S., Crawford, Robert W., Brooks, Susan V., Chamberlain, Jeffrey S.
Médium: Artigo
Jazyk:Inglês
Vydáno: National Academy of Sciences 2002
Témata:
On-line přístup:https://ncbi.nlm.nih.govhttps://pmc.ncbi.nlm.nih.gov/articles/PMC130572/
https://ncbi.nlm.nih.govhttps://pubmed.ncbi.nlm.nih.gov/12271128/
https://ncbi.nlm.nih.govhttps://doi.org/10.1073/pnas.202300099
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