Linked Life Data

19675594

Source:http://linkedlifedata.com/resource/pubmed/id/19675594

Statements in which the resource exists as a subject.
PredicateObject
rdf:type
lifeskim:mentions
pubmed:issue
11
pubmed:dateCreated
2009-11-12
pubmed:abstractText
ADAMTS13 is a plasma metalloprotease that regulates the size of the von Willebrand factor (VWF) multimers. Genetic or acquired deficiency of ADAMTS13 causes thrombotic thrombocytopenic purpura (TTP) in humans. Plasma infusion is the treatment of choice for patients with congenital ADAMTS13 deficiency. However, this practice exposes patients to the risk of infections, allergies and fluid volume overload. The search for alternative treatments is required. Here, we tested the ability of systemically administered adenovirus encoding human ADAMTS13 to restore the deficient protein in the circulation of Adamts13(-/-) mice. Injection of the adenovirus efficiently transduced the liver, kidney, lung, heart and spleen, resulting in the secretion of ADAMTS13 into plasma. A reduced area of thrombi was observed when blood from Ad-ADAMTS13-treated mice was perfused over a collagen-coated surface in a parallel plate flow chamber compared with blood of Ad-betaGal-treated controls. The secreted ADAMTS13 protein was functionally active even after 2 months from injection. The data provide the proof of principle for developing a novel therapy for the correction of ADAMTS13 deficiency in patients with hereditary TTP.
pubmed:language
eng
pubmed:journal
pubmed:citationSubset
IM
pubmed:chemical
pubmed:status
MEDLINE
pubmed:month
Nov
pubmed:issn
1476-5462
pubmed:author
pubmed:issnType
Electronic
pubmed:volume
16
pubmed:owner
NLM
pubmed:authorsComplete
Y
pubmed:pagination
1373-9
pubmed:meshHeading
pubmed:year
2009
pubmed:articleTitle
Adenoviral-mediated gene transfer restores plasma ADAMTS13 antigen and activity in ADAMTS13 knockout mice.
pubmed:affiliation
Department of Molecular Medicine, Mario Negri Institute for Pharmacological Research, Bergamo, Italy.
pubmed:publicationType
Journal Article, Research Support, Non-U.S. Gov't, Evaluation Studies