Document Detail


Gene therapy in haemophilia--going for cure?
MedLine Citation:
PMID:  20586798     Owner:  NLM     Status:  MEDLINE    
Abstract/OtherAbstract:
Traditional treatment for haemophilia consists of bolus infusion of the missing coagulation factor, either prophylactically or on demand, but is complicated by the development of inhibitory antibodies to the infused factor. In those cases, as well as in patients with platelet defects or factor VII (FVII) deficiency, recombinant human activated FVII has been successfully used, but carries the disadvantage of a short plasma half-life. As an alternative, emerging methodology based on gene transfer may be utilized to provide effective haemostasis in patients with coagulation defects. The goal of this article is to introduce the novel concept of continuous expression of activated FVII from a donated gene for the treatment of haemophilia, and to review the safety and efficacy data that have been produced so far by this approach in small and large animal models.
Authors:
P Margaritis; K A High
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Publication Detail:
Type:  Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't; Review    
Journal Detail:
Title:  Haemophilia : the official journal of the World Federation of Hemophilia     Volume:  16 Suppl 3     ISSN:  1365-2516     ISO Abbreviation:  Haemophilia     Publication Date:  2010 May 
Date Detail:
Created Date:  2010-06-30     Completed Date:  2011-01-05     Revised Date:  -    
Medline Journal Info:
Nlm Unique ID:  9442916     Medline TA:  Haemophilia     Country:  England    
Other Details:
Languages:  eng     Pagination:  24-8     Citation Subset:  IM    
Affiliation:
Division of Hematology, The Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA.
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MeSH Terms
Descriptor/Qualifier:
Animals
Disease Models, Animal
Dogs
Factor VII / genetics*,  metabolism,  therapeutic use*
Gene Expression Regulation
Gene Therapy / methods*
Hemophilia A / therapy*
Hemophilia B / therapy*
Hemostasis
Mice
Grant Support
ID/Acronym/Agency:
P01 HL64190/HL/NHLBI NIH HHS; //Howard Hughes Medical Institute
Chemical
Reg. No./Substance:
9001-25-6/Factor VII

From MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine


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