TALEN-based Gene Correction for Epidermolysis Bullosa
TALEN-based Gene Correction for Epidermolysis Bullosa
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Author / Creator
Osborn, Mark J , Starker, Colby G , McElroy, Amber N , Webber, Beau R , Riddle, Megan J , Xia, Lily , DeFeo, Anthony P , Gabriel, Richard , Schmidt, Manfred , Von Kalle, Christof , Carlson, Daniel F , Maeder, Morgan L , Joung, J Keith , Wagner, John E , Voytas, Daniel F , Blazar, Bruce R and Tolar, Jakub
Publisher
United States: Elsevier Inc
Journal title
Language
English
Formats
Publication information
Publisher
United States: Elsevier Inc
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More information
Scope and Contents
Contents
Recessive dystrophic epidermolysis bullosa (RDEB) is characterized by a functional deficit of type VII collagen protein due to gene defects in the type VII collagen gene (COL7A1). Gene augmentation therapies are promising, but run the risk of insertional mutagenesis. To abrogate this risk, we explored the possibility of using engineered transcripti...
Alternative Titles
Full title
TALEN-based Gene Correction for Epidermolysis Bullosa
Authors, Artists and Contributors
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Primary Identifiers
Record Identifier
TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_3677309
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_3677309
Other Identifiers
ISSN
1525-0016
E-ISSN
1525-0024
DOI
10.1038/mt.2013.56