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Is it going to be SIN?
Author(s) -
Buchholz Christian J.,
Cichutek Klaus
Publication year - 2006
Publication title -
the journal of gene medicine
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.689
H-Index - 91
eISSN - 1521-2254
pISSN - 1099-498X
DOI - 10.1002/jgm.966
Subject(s) - insertional mutagenesis , genetic enhancement , long terminal repeat , vector (molecular biology) , clinical trial , viral vector , mutagenesis , lentivirus , virology , gene , severe combined immunodeficiency , biology , human immunodeficiency virus (hiv) , genetics , medicine , computational biology , mutation , bioinformatics , genome , viral disease , recombinant dna
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.