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Mol Ther Methods Clin Dev. 2017 Jan 11;4:102-114. doi: 10.1016/j.omtm.2017.01.002. eCollection 2017 Mar 17.

Codon Optimization Leads to Functional Impairment of RD114-TR Envelope Glycoprotein.

Molecular therapy. Methods & clinical development

Eleonora Zucchelli, Monika Pema, Anna Stornaiuolo, Claudia Piovan, Cinzia Scavullo, Erica Giuliani, Sergio Bossi, Stefano Corna, Claudia Asperti, Claudio Bordignon, Gian-Paolo Rizzardi, Chiara Bovolenta

Affiliations

  1. MolMed S.p.A., 20132 Milano, Italy; Great Ormond Street Institute of Child Health, University College London, London WC1N 1EH, UK.
  2. MolMed S.p.A., 20132 Milano, Italy.

PMID: 28344996 PMCID: PMC5363313 DOI: 10.1016/j.omtm.2017.01.002

Abstract

Lentiviral vectors (LVs) are a highly valuable tool for gene transfer currently exploited in basic, applied, and clinical studies. Their optimization is therefore very important for the field of vectorology and gene therapy. A key molecule for LV function is the envelope because it guides cell entry. The most commonly used in transiently produced LVs is the vesicular stomatitis virus glycoprotein (VSV-G) envelope, whose continuous expression is, however, toxic for stable LV producer cells. In contrast, the feline endogenous retroviral RD114-TR envelope is suitable for stable LV manufacturing, being well tolerated by producer cells under constitutive expression. We have previously reported successful, transient and stable production of LVs pseudotyped with RD114-TR for good transduction of T lymphocytes and CD34

Keywords: RD114-TR; RNA structure; codon optimization; envelope glycoprotein; gene therapy; gene vector delivery; gene vector engineering; lentiviral vector; packaging cell line

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