Gene therapy is a still somewhat experimental treatment that involves introducing genetic material into a person’s cells to fight or prevent disease. Researchers are studying gene therapy for a number of diseases, such as severe combined immuno-deficiencies, haemophilia, Parkinson’s disease, cancer and HIV, via a number of different approaches.
A gene can be delivered to a cell using a carrier known as a “vector.” The most common types of vectors used in gene therapy are viruses. The viruses used in gene therapy are altered to make them safe, although some risks still exist with gene therapy. The technology is still in its infancy, but it has been used with some success.
In most gene therapy studies, a “normal” gene is inserted into the genome to replace an “abnormal,” disease-causing gene. In cancer, some cells become diseased because certain genes have been permanently turned off. Using gene therapy, mutated genes that cause disease could be turned off so that they no longer promote disease, or healthy genes that help prevent disease could be turned on so that they can inhibit the disease.
Types of Gene Therapy
Virtually all cells in the human body contain genes, making them potential targets for gene therapy. However, these cells can be divided into two major categories: somatic cells (most cells of the body) or cells of the germline (eggs or sperm). In theory it is possible to transform either somatic cells or germ cells.
Somatic Gene therapy falls into two main categories:
1. In Vivo: Genes are changed in cells still in the body. The gene is transferred to cells inside the patient’s body via direct injection of the gene therapy vector, carrying the desired gene, into the bloodstream or target organ.
2. Ex Vivo: A patient’s cells are removed, modified outside the body and then e-infusing them back into the patient again, as in haematopoietic stem cell transplant and CAR T-cell therapy.
Read more about Gene Therapy in Cancer Treatment on our static page, Gene Therapy