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

Abstract:

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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Publication status:
Published

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Institution:
University of Oxford
Division:
MSD
Department:
Physiology Anatomy & Genetics
Role:
Author
Journal:
Molecular therapy : the journal of the American Society of Gene Therapy More from this journal
Volume:
7
Issue:
5 Pt 1
Pages:
604-612
Publication date:
2003-05-01
DOI:
EISSN:
1525-0024
ISSN:
1525-0016

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