Clinical Pharmacology and Therapeutics

96 CELL-BASED AND RECOMBINANT DNA THERAPIES
potential than adult stem cells for research and possible
therapy.
The Gene Therapy Advisory Committee (GTAC) is the
national research ethics committee for gene therapy clinical
research. GTAC’s definition of gene therapy is as follows: ‘The
deliberate introduction of genetic material into human
somatic cells for therapeutic, prophylactic or diagnostic purposes.’
This definition, and hence the remit of GTAC, encompasses
techniques for delivering synthetic or recombinant
nucleic acids into humans:
• genetically modified biological vectors, such as viruses or
plasmids;
• genetically modified stem cells;
• oncolytic viruses;
• nucleic acids associated with delivery vehicles;
• naked nucleic acids;
• antisense techniques (for example, gene silencing, gene
correction or gene modification);
• genetic vaccines;
• DNA or RNA technologies, such as RNA interference;
• xenotransplantation of animal cells, but not solid organs.
FURTHER READING
Anon. Understanding monoclonal antibodies. Drugs and Therapeutics
Bulletin 2007; 45.
Anson DS. The use of retroviral vectors for gene therapy – what are
the risks? A review of retroviral pathogenesis and its relevance to
retroviral vector-mediated gene delivery. Genetic Vaccines and
Therapy 2004; 2: 9.
Check E. A tragic setback. Nature 2002; 420: 116–18.
Guttmacher AE, Collins FS. Genomic medicine a primer. New England
Journal of Medicine 2002; 347: 1512–20.
Marshall E. Gene therapy death prompts review of adenovirus vector.
Science 1999; 286: 2244–5.
Nathwani AC, Davidoff AM, Linch DC. A review of gene therapy for
haematological disorders. British Journal of Haematology 2005; 128:
3–17.
Rang HP, Dale MM, Ritter JM, Flower RJ. Chapter 55
Biopharmaceuticals and gene therapy. In: Pharmacology, 6th edn.
Oxford: Elsevier, 2007.
Safer medicines. A report from the Academy. London: The Academy of
Medical Sciences, 2005.
Walsh G. Second-generation biopharmaceuticals. European Journal of
Pharmaceutics and Biopharmaceutics 2004; 58: 185–96.