Linked Life Data

18386226

Source:http://linkedlifedata.com/resource/pubmed/id/18386226

Statements in which the resource exists as a subject.
PredicateObject
rdf:type
lifeskim:mentions
pubmed:issue
2
pubmed:dateCreated
2008-4-3
pubmed:abstractText
Antisense-mediated exon skipping is an approach that uses antisense oligonucleotides (AONs) to modulate splicing by hiding specific sites essential for exon inclusion from the splicing machinery. AONs that block aberrant splice sites can restore normal splicing, whereas AONs targeting alternative splice sites can switch splicing patterns from detrimental to beneficial isoforms or produce non-functional mRNAs that lead to gene knockdown. Furthermore, AONs have also been used to restore a disrupted reading frame, thereby generating semi-functional proteins instead of non-functional proteins. Proof-of-concept has been obtained for each of the above AON applications in vitro and for some applications in vivo. Antisense-mediated reading frame restoration is the most promising therapy for Duchenne muscular dystrophy. Data from a first clinical trial are encouraging and additional trials are ongoing or are expected to be initiated soon.
pubmed:language
eng
pubmed:journal
pubmed:citationSubset
IM
pubmed:chemical
pubmed:status
MEDLINE
pubmed:month
Apr
pubmed:issn
1464-8431
pubmed:author
pubmed:issnType
Print
pubmed:volume
10
pubmed:owner
NLM
pubmed:authorsComplete
Y
pubmed:pagination
140-9
pubmed:meshHeading
pubmed:year
2008
pubmed:articleTitle
The therapeutic potential of antisense-mediated exon skipping.
pubmed:affiliation
Leiden University Medical Center, Department of Human Genetics, Zone S4-P, PO Box 9600, 2300 RC Leiden, the Netherlands.
pubmed:publicationType
Journal Article, Review, Research Support, Non-U.S. Gov't