Progress and problems with the use of viral vectors for gene therapy

By Clare E. Thomas, A. Ehrhardt, M. Kay. Nature reviews genetics, 2003

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Delivering CRISPR: a review of the challenges and approaches (2018, background): “In all cases of leukemia, the SCID X-1 correcting gene had inserted into the patient genome within or near tumor-promoting genes and caused transcriptional activation (Check, 2002; Kaiser, 2003; Thomas et al., 2003).”
State-of-the-art gene-based therapies: the road ahead (2011, background): “The various vectors' history, creation and use have been reviewe”
Clinical use of lentiviral vectors (2018, background): “U5 lopR R3UTPPvnegagPBSU5R pol cPPT cPPT tRNA tRNA cPPT cPPT cPPT cFLAP gag env PPT U3 RPBS pol gag env PPT U3 RPBS pol gag env PPT U3 RPBS pol gag env PPT U3 LTR RNA-DNA duplex U3 RPBSU5 U5 U5 U5 lopR RT RT LTR RT RT RT Fig.”
MicroRNA: Biogenesis, Function and Role in Cancer (2010, background): “While some vectors have been useful in animal studies they are not ideal for human because they can elicit an immune response that deflates their effectiveness [371-373].”
Towards safe, non-viral therapeutic gene expression in humans (2005, background): “, viral-based vectors urgently need to be reassessed with regard to their safety for human gene therap”
Virus entry: molecular mechanisms and biomedical applications (2004, background): “Rhabdoviridae ssRNA Enveloped Rabies virus Endocytosis, acetylcholine Rabies (11-15) (45-100) receptor, neural cell adhesion molecule”