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Lentiviral Vector Promoter is Decisive for Aberrant Transcript Formation

Authors :
Christof von Kalle
Daniela Cesana
Eugenio Montini
Cynthia C. Bartholomä
Manfred Schmidt
Raffaele Fronza
Irene Gil-Farina
Simone Scholz
Source :
Human gene therapy. 28(10)
Publication Year :
2017

Abstract

Lentiviral vectors hold great promise for the genetic correction of various inherited diseases. However, lentiviral vector biology is still not completely understood and warrants the precise decoding of molecular mechanisms underlying integration and post-translational modification. This study investigated a series of self-inactivating (SIN) and full long terminal repeat (LTR) lentiviral vectors that contained different types of promoters with or without a transgene to gain deeper insights in lentiviral target site selection and potential perturbation of cellular gene expression. Using an optimized nonrestrictive linear amplification-mediated polymerase chain reaction (nrLAM-PCR) protocol, vector structure-dependent integration site profiles were observed upon transduction of mouse lin

Details

ISSN :
15577422
Volume :
28
Issue :
10
Database :
OpenAIRE
Journal :
Human gene therapy
Accession number :
edsair.doi.dedup.....0b54c22d32cfd470b8f191a1ff307830