Infectious delivery of a 135-kb LDLR genomic locus leads to regulated complementation of low-density lipoprotein receptor deficiency in human cells.

Journal article

The ability to deliver efficiently a complete genomic DNA locus to human and rodent cells will likely find widespread application in functional genomic studies and novel gene therapy protocols. In contrast to a cDNA expression cassette, the use of a complete genomic DNA locus delivers a transgene intact with its native promoter, the exons, all the intervening introns, and the regulatory regions. The presence of flanking, noncoding genomic DNA sequences could prove critical for prolonged and a...

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Authors: Wade-Martins, R; Saeki, Y; Chiocca, E

• Publication status: Published
• Journal: Molecular therapy : the journal of the American Society of Gene Therapy
• Volume: 7
• Issue: 5 Pt 1
• Pages: 604-612
• Publication date: 2003-05-01
• DOI: 10.1016/s1525-0016(03)00060-1
• EISSN: 1525-0024
• ISSN: 1525-0016
• Language: English
• Keywords: Gene Transfer Techniques; Cricetinae; Genetic Complementation Test; Receptors, LDL; Luminescent Proteins; Green Fluorescent Proteins; Transgenes; Sterols; Epstein-Barr Virus Infections; Hyperlipoproteinemia Type II; Molecular Weight; Transformation, Genetic; Chromosomes, Artificial, Bacterial; Fibroblasts; Plasmids; Genetic Vectors; Animals; Genome; Herpesvirus 4, Human; Humans; Transfection; Genetic Therapy; CHO Cells; In Situ Hybridization, Fluorescence