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Stable correction of a genetic deficiency in human cells by an episome carrying a 115 kb genomic transgene.

Abstract:

Persistent expression of a transgene at therapeutic levels is required for successful gene therapy, but many small vectors with heterologous promoters are prone to vector loss and transcriptional silencing. The delivery of genomic DNA would enable genes to be transferred as complete loci, including regulatory sequences, introns, and native promoter elements. These elements may be critical to ensure prolonged, regulated, and tissue-specific transgene expression. Many studies point to considera...

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Publication status:
Published

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Publisher copy:
10.1038/82444

Authors


More by this author
Institution:
University of Oxford
Division:
MSD
Department:
Physiology Anatomy & Genetics
Role:
Author
More by this author
Institution:
University of Oxford
Division:
MSD
Department:
Pathology Dunn School
Role:
Author
Journal:
Nature biotechnology
Volume:
18
Issue:
12
Pages:
1311-1314
Publication date:
2000-12-01
DOI:
EISSN:
1546-1696
ISSN:
1087-0156
Source identifiers:
15792

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