학술논문
Differential expression and secretion of alpha 1 anti-trypsin between direct DNA injection and implantation of transfected myoblast.
Document Type
Article
Author
Source
Subject
*GENE therapy
*PLASMIDS
*MYOBLASTS
*ALPHA 1-antitrypsin
*DNA
*
*
*
*
Language
ISSN
0969-7128
Abstract
Muscle can be used for systemic delivery of non-muscle proteins. In order to investigate the relative effectiveness of direct DNA plasmid injection versus implantation of genetically modified myogenic cell lines, we have used the human alpha 1 anti-trypsin (α1AT) cDNA driven by either cytomegalovirus (CMV) or the muscle creatine kinase 3.3 kb (MCK) promoter in immunodeficient mice. We dem- onstrate that the implantation of transfected myoblasts stably expressing the human α1AT cDNA generates a more persistent production of α1AT than does direct intramuscular injection of the same construct as plasmid DNA. Moreover, immunohistological labelling of muscle sections implanted with myoblasts show that the newly formed muscle fibres are those containing the human protein. [ABSTRACT FROM AUTHOR]