scholarly journals Human Involucrin Promoter Mediates Repression-Resistant and Compartment-Specific LEKTI Expression

2012 ◽  
Vol 23 (1) ◽  
pp. 83-90 ◽  
Author(s):  
Wei-Li Di ◽  
Ekaterina Semenova ◽  
Fernando Larcher ◽  
Marcela Del Rio ◽  
John I. Harper ◽  
...  
Keyword(s):  
Blood ◽  
2000 ◽  
Vol 95 (9) ◽  
pp. 2799-2805 ◽  
Author(s):  
Steven S. Fakharzadeh ◽  
Yue Zhang ◽  
Rita Sarkar ◽  
Haig H. Kazazian

To test the hypothesis that factor VIII expressed in the epidermis can correct hemophilia A, we generated transgenic mice in a factor VIII–deficient background that express human factor VIII under control of the involucrin promoter. Mice from 5 transgenic lines had both phenotypic correction and plasma factor VIII activity. In addition to the skin, however, some factor VIII expression was detected in other tissues that have stratified squamous epithelia. To determine whether an exclusively cutaneous source of factor VIII could correct factor VIII deficiency, we grafted skin explants from transgenic mice onto mice that are double knockouts for the factor VIII and RAG-1 genes. Two graft recipients had plasma factor VIII activity of 4% to 20% of normal and improved whole blood clotting compared with factor VIII–deficient mice. Thus, expression of factor VIII from the epidermis can correct hemophilia A mice, thereby supporting the feasibility of cutaneous gene therapy for systemic disease.


1998 ◽  
Vol 16 ◽  
pp. S131
Author(s):  
Jean F. Welter ◽  
Chapla Agarwal ◽  
Tatiana Efimova ◽  
Richard L. Eckert

2011 ◽  
Vol 21 (3) ◽  
pp. 683-689 ◽  
Author(s):  
Petr Kasparek ◽  
Pavel Krenek ◽  
Halka Buryova ◽  
Sarka Suchanova ◽  
Inken Maria Beck ◽  
...  

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