Linked Life Data

18500943

Source:http://linkedlifedata.com/resource/pubmed/id/18500943

Statements in which the resource exists as a subject.
PredicateObject
rdf:type
lifeskim:mentions
pubmed:issue
6
pubmed:dateCreated
2008-6-26
pubmed:abstractText
Many mutations and deletions in the dystrophin gene, responsible for Duchenne muscular dystrophy (DMD), can be corrected at the posttranscriptional level by skipping specific exons. Here we show that long-term benefit can be obtained in the dystrophic mouse model through the use of adeno-associated viral vectors expressing antisense sequences: persistent exon skipping, dystrophin rescue, and functional benefit were observed 74 weeks after a single systemic administration. The therapeutic benefit was sufficient to preserve the muscle integrity of mice up to old age. These results indicate a possible long-term gene therapy treatment of the DMD pathology.
pubmed:language
eng
pubmed:journal
pubmed:citationSubset
IM
pubmed:chemical
pubmed:status
MEDLINE
pubmed:month
Jun
pubmed:issn
1557-7422
pubmed:author
pubmed:issnType
Electronic
pubmed:volume
19
pubmed:owner
NLM
pubmed:authorsComplete
Y
pubmed:pagination
601-8
pubmed:meshHeading
pubmed:year
2008
pubmed:articleTitle
Long-term benefit of adeno-associated virus/antisense-mediated exon skipping in dystrophic mice.
pubmed:affiliation
Department of Genetics and Molecular Biology, Institute Pasteur Cenci-Bolognetti, University of Rome La Sapienza, 00185 Rome, Italy.
pubmed:publicationType
Journal Article, Research Support, Non-U.S. Gov't