Gene Transfer

To understand how gene therapy works, one needs to have a basic understanding of the human body and how a human cell functions. In this section, we give a very brief introduction to human cellular biology, providing enough background so that the layman can understand how gene therapy works. The aim of the section is to attempt to dispel any possible misconceptions the public has about gene therapy, and to introduce the topic to those interested in pursuing further education in this area.

Gene Therapy Approaches

Effective transfer of genetic material into human cells is perhaps the biggest challenge in Gene Therapy. A gene transfer agent has to be safe, introduce its DNA cargo into a sufficiently large population of cells to produce a biological effect and mediate expression of the desired gene for a sustained period of time. Identifying a gene transfer tool that meets all of these criteria has proven to be a difficult task.

Gene Therapy is normally used in diseases where the cells of a particular organ or system cannot function correctly due to the fact that they do not have an essential protein that is required to perform a specific task. In order to replace the malfunctioning protein, a gene transfer vector (such as a disabled adenovirus in the diagram above) is modified so that it contains the gene that encodes for this protein. The modified vector is then given to the patient where it enters the affected cells. The gene tranfer vector then transfers the therapeutic gene to the cell and the cell's own machinery turns the therapeutic gene into correct version of the protein, which in essence fixes the malfunctioning cell.

In our section on gene transfer vectors we provide more information on the most widely used gene transfer systems studied to date. The information provided in those pages is targeted to non-experts in the field, with some knowledge in biology, and aims to provide a further educational tool for those wishing to learn about gene therapy.