Linked Life Data

15294178

Source:http://linkedlifedata.com/resource/pubmed/id/15294178

Statements in which the resource exists as a subject.
PredicateObject
rdf:type
lifeskim:mentions
pubmed:issue
2
pubmed:dateCreated
2004-8-5
pubmed:abstractText
Dystrophic epidermolysis bullosa (DEB) is a family of inherited mechanobullous disorders caused by mutations in the gene, COL7A1, that codes for type VII, (anchoring fibril), collagen, which is critical for epidermal-dermal adherence. Most gene therapy approaches have been ex vivo, involving cell culture and culture graft transplantation, which is logistically difficult. To develop a more simplified approach, we engineered a self-inactivating lentiviral vector expressing human type VII collagen and injected this vector intradermally into hairless, immunodeficient mice and into a human DEB composite skin equivalent grafted onto immunodeficient mice. In both situations, the vector transduced dermal cells, which in turn synthesized and exported type VII collagen into the extracellular space. Remarkably, the type VII collagen selectively adhered to and incorporated into the basement membrane zone (BMZ) between the dermis and the epidermis, where it formed anchoring fibril structures. In the case of the DEB skin equivalent, the newly expressed type VII collagen reversed the DEB phenotype characterized by poor epidermal-dermal adherence and anchoring fibril defects. A single lentiviral vector injection provided stable type VII collagen at the BMZ for at least 3 months. These data demonstrate efficient and long-term type VII collagen gene transfer in vivo using direct intradermal injection of an engineered lentiviral vector.
pubmed:grant
pubmed:language
eng
pubmed:journal
pubmed:citationSubset
IM
pubmed:chemical
pubmed:status
MEDLINE
pubmed:month
Aug
pubmed:issn
1525-0016
pubmed:author
pubmed:copyrightInfo
Copyright The American Society of Gene Therapy
pubmed:issnType
Print
pubmed:volume
10
pubmed:owner
NLM
pubmed:authorsComplete
Y
pubmed:pagination
318-26
pubmed:dateRevised
2008-11-21
pubmed:meshHeading
pubmed:year
2004
pubmed:articleTitle
Intradermal injection of lentiviral vectors corrects regenerated human dystrophic epidermolysis bullosa skin tissue in vivo.
pubmed:affiliation
Division of Dermatology, Department of Medicine, University of Southern California, Los Angeles, CA, 90033, USA.
pubmed:publicationType
Journal Article, Research Support, U.S. Gov't, P.H.S.