Systematic ranking of inborn errors of metabolism as targets for gene therapy

2010 ◽  
Vol 100 (3) ◽  
pp. 215-218 ◽  
2019 ◽  
Vol 30 (10) ◽  
pp. 1204-1210 ◽  
Author(s):  
Virginia Maria Ginocchio ◽  
Rita Ferla ◽  
Alberto Auricchio ◽  
Nicola Brunetti-Pierri

1995 ◽  
Vol 4 (3) ◽  
pp. 343-346 ◽  
Author(s):  
Norio Sakuragawa ◽  
Jun Tohyama ◽  
Hiroshi Yamamoto

Immunoreactivity of human cultured amniotic epithelial (AE) cells was investigated to evaluate the possible use of these cells as a transgene carrier in gene therapy for inborn errors of metabolism. AE cells were prepared and cultured by the methods described previously. Flow cytometry analysis revealed that these cells did not express any class II antigen at all on their surfaces. But the class I antigen was slightly expressed on their surfaces. Immunoperoxidase staining was slightly positive as to the class I antigen but not to the class II antigen at all. pSV-β-galactosidase was transfected into AE cells by means of electroporation, followed by staining of the cells with X-gal. Several cells in 60 mm dish expressed β-galactosidase activity. The possible gene transfer of β-galactosidase into cultured AE cells may suggest that these cells could be used as a transgene carrier in gene therapy for inborn errors of metabolism.


2016 ◽  
pp. 111-129
Author(s):  
Dolan Sondhi ◽  
Ronald G. Crystal ◽  
Stephen M. Kaminsky

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