Disease Targets

Gene therapy can also be used to treat life-threatening infections where no other treatment is available. In this case, genetic information is specifically introduced into infected cells with a view to prevent the effective replication of the target pathogen. In this section we describe some of the approaches that have been adopted in the treatment of the most prevalent infectious diseases.

In general, genetic disorders are inherited diseases that arise when someone has two dysfunctional copies of a single gene on both of their chromosomes. The result of this is often devastating, resulting in abnormal functioning of specific cells and the development of an often life-threatening condition. In these cases gene therapy is employed to replace the dysfunctional gene with a correct copy and restore the normal functioning of affected cells. In this page we describe some of the most well-known genetic disorders that are being targeted by the gene therapy community.

Cancer can be described as a disease where cellular communication has broken down, allowing transformed cells to escape tight regulatory signals and to replicate autonomously and continously, ultimately invading and interfering with the functions of normal tissues. Under physiological conditions cells communicate with one another by activating receptors at the cell surface, which convey the signal through pathways of proteins located in the cytoplasm and subsequently through networks of transcription factors in the nucleus with control the expression of genes that mediate the cell's response. In this article we briefly describe the gene therapy approaches that have been adopted in an effort to treat cancer.

Typically, cancer develops as a result of aberrant growth factor signalling, where a pathway that instructs a cell to grow and divide becomes constitutively active. This arises through mutations in a growth factor receptor, or through mutations in the components of cell signalling pathways. (Collectively, genes that encode for mutated cellular proteins involved in promoting cell growth are termed oncogenes). Threrefore, in order to effectively treat a cancer, it is essential that all cells that carry a mutated oncogene, are destroyed, otherwise the cancer will continue to grow and spread.