Biomedical Engineering Reference
In-Depth Information
11
Delivery Systems
for Gene Transfer
Charlotte Lawson
1
and Louise Collins
2
1
Veterinary Basic Sciences, Royal Veterinary College, London, UK
2
Department of Clinical Sciences, Kings's College London School of Medicine,
James Black Centre, London, UK
11.1 Introduction
Insertion of genetic material into the cells of an individual to treat a disease or correct
a hereditary condition has evolved from the distant dream of molecular biologists to a
viable treatment option, with a number of successful clinical trials published to date. His-
torically, advancement within the gene therapy field has required development in other
areas of molecular biology. The therapeutic potential of being able to harness the power
of viruses that integrate their DNA into mammalian cells was recognized long before the
recombinant DNA technology was available to isolate and clone the genes to be delivered
[1]. Safety has always been a large consideration when designing 'viral vectors' for gene
therapy, from the point of view of toxicity of the introduced genetic material or its gene
products, the possibility of recombination events occurring that could lead to production
of mobilizable viruses, and insertional mutagenesis. In view of this there is also a large
literature on the development and use of 'non-viral' vectors for gene therapy applications.
Since before the first clinical trials were carried out to treat severe combined immunode-
ficiency (SCID) patients in the 1990s there has been significant debate about viral versus
non-viral gene transfer. Here, we will give a brief overview of some of the more commonly
used transfer strategies together with protocols for viral and non-viral gene transfer into
mammalian cells.
