Document Detail

Ex vivo gene therapy cures a blistering skin disease.
MedLine Citation:
PMID:  17416553     Owner:  NLM     Status:  MEDLINE    
A recent publication that describes gene therapy treatment of a patient with an inherited blistering skin disease, epidermolysis bullosa, demonstrates for the first time that gene therapy can cure a disease of solid tissue. The treatment relies on ex vivo transduction of autologous epidermal stem cells with a normal copy of the defective gene, followed by reconstitution of the patient's skin with epithelial sheets that are grown from these genetically corrected cells. This approach holds promise for treatment not only of inherited disorders of the skin but also of other solid tissues that are becoming amenable to tissue engineering.
Carol Featherstone; Jouni Uitto
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Publication Detail:
Type:  Journal Article     Date:  2007-04-09
Journal Detail:
Title:  Trends in molecular medicine     Volume:  13     ISSN:  1471-4914     ISO Abbreviation:  Trends Mol Med     Publication Date:  2007 Jun 
Date Detail:
Created Date:  2007-06-06     Completed Date:  2008-04-04     Revised Date:  -    
Medline Journal Info:
Nlm Unique ID:  100966035     Medline TA:  Trends Mol Med     Country:  England    
Other Details:
Languages:  eng     Pagination:  219-22     Citation Subset:  IM    
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MeSH Terms
Clinical Trials as Topic / adverse effects
Epidermolysis Bullosa / genetics,  pathology,  therapy*
Gene Therapy* / adverse effects
Orphan Drug Production
Stem Cell Transplantation
Stem Cells / metabolism

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