Direct transfer of human dystrophin genes into striated muscles of mdx mice


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ABSTRACT Plasmid DNA constructs containing either a human full length dystrophin cDNA (G. Dickson, London, UK) or a deleted, Becker type, dystrophin cDNA (K.E.Davies, DR. Love, Oxford, UK.)


were injected into hearts and quadriceps of dystrophin deficient MDX mice. Seven days later, analysis of immunohistochemical staining for dystrophin showed expression of dystrophin proteins


in ∼1 % of quadriceps muscle cells localized mostly in the sarcolemma. 10-15 dystrophin positive cardiocyte were also seen in sections of injected hearts. Western-blot of quadriceps muscles


has also identified the dystrophin expressions. Peripherally localized nuclei of muscle cells were shown in about 50 percent of dystrophin positive myofibers compared with 20 percent of


dystrophin negativ myofibers suggesting the the functional effect of expressed human dystrophin protein. This direct gene transfer method has an alternative application for genetherapy of


inherited myopathies but the efficiency must be increased. ARTICLE PDF AUTHOR INFORMATION AUTHORS AND AFFILIATIONS * Department of Pediatrics, Univ. Medical School of Pécs, Hungary, Waisman


Center, University of Wisconsin, Madison, USA Gyula Acsádi, Ágnes Jáni & Jon A Wolff Authors * Gyula Acsádi View author publications You can also search for this author inPubMed Google


Scholar * Ágnes Jáni View author publications You can also search for this author inPubMed Google Scholar * Jon A Wolff View author publications You can also search for this author inPubMed 


Google Scholar RIGHTS AND PERMISSIONS Reprints and permissions ABOUT THIS ARTICLE CITE THIS ARTICLE Acsádi, G., Jáni, Á. & Wolff, J. Direct Transfer of Human Dystrophin Genes into


Striated Muscles of MDX Mice. _Pediatr Res_ 32, 621 (1992). https://doi.org/10.1203/00006450-199211000-00099 Download citation * Issue Date: November 1992 * DOI:


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