Title:Production of Lentivectors for Clinical Use
VOLUME: 13 ISSUE: 3
Author(s):David Escors, Therese Liechtenstein, Noemi Perez-Janices, Idoia Blanco-Luquin and David Guerrero-Setas
Affiliation:Immunomodulation group, Navarrabiomed-Fundacion Miguel Servet, C/ Irunlarrea N3, 31008 Pamplona, Navarra, Spain.
Keywords:Biosafety, clinical grade lentivector, gene therapy, genotoxicity, GMP guidelines, HIV, immunology, insertional
mutagenesis, mutagenesis assay, packaging cell lines, purification, replication competent lentivirus, replication competent retrovirus.
Abstract:Since the first human gene therapy clinical trials were proven successful in 2000, human gene therapy has witnessed
setbacks as well as encouraging progress. While these first clinical trials were performed with a γ-retrovirus vector
to correct X-linked severe combined immunodeficiency (SCID-X1) in children, others since then have applied similar or
alternative viral vector strategies. More recently, lentiviral vectors have been used for the correction of human β-
thalassaemia and adrenoleukodystrophy. They have also been successfully used in the treatment of leukaemia by modifying
cytotoxic T cells directed to cancer cells. Here we describe the production of clinical grade retro- and lentiviral vectors
for application in human therapy.
