Is it going to be SIN? : A European Society of Gene Therapy commentary. Phasing‐out the clinical use of non self‐inactivating murine leukemia virus vectors: initiative on hold

Medientyp: E-Artikel
Titel: Is it going to be SIN? : A European Society of Gene Therapy commentary. Phasing‐out the clinical use of non self‐inactivating murine leukemia virus vectors: initiative on hold : A European Society of Gene Therapy commentary. Phasing‐out the clinical use of non self‐inactivating murine leukemia virus vectors: initiative on hold
Beteiligte: Buchholz, Christian J.; Cichutek, Klaus
Erschienen: Wiley, 2006
Erschienen in: The Journal of Gene Medicine, 8 (2006) 10, Seite 1274-1276
Sprache: Englisch
DOI: 10.1002/jgm.966
ISSN: 1099-498X; 1521-2254
Schlagwörter: Genetics (clinical) ; Drug Discovery ; Genetics ; Molecular Biology ; Molecular Medicine
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Beschreibung: <jats:title>Abstract</jats:title><jats:p>Insertional mutagenesis resulting in a leukaemia‐like lymphoproliferative disease, as observed in the X‐SCID (severe combined immunodeficiency) clinical trial using a γ‐retroviral vector that transferred a functional copy of the defective gene into hematopoietic precursor cells of affected children, sparked a debate about a ban on conventional γ‐retroviral vectors. This commentary summarizes the relevant data on this topic and concludes that there is no preclinical or clinical evidence as yet that SIN vectors, which self‐inactivate the retroviral long terminal repeats (LTRs), will indeed show an improved safety profile. Conventional murine leukaemia virus (MLV) vectors can thus be used further in clinical gene therapy trials but require a thorough case‐by‐case risk‐benefit analysis. Copyright © 2006 John Wiley & Sons, Ltd.</jats:p>

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