Gene Therapy Essay

Gene therapy focuses on the replacement of defective genes with modified functioning genes. Many diseases are caused by a defective gene meaning the body is incapable of producing essential proteins or enzymes. In its simplest form, gene therapy aims to identify the defective gene and fix this gene with the replacement of a normal gene (Senn).

2.2 Types of Gene Therapy

There are two types of gene therapy, somatic gene therapy and germ line gene therapy. Somatic gene therapy involves fixing the defective gene. With this form of therapy, only somatic cells are targeted and not the germ line cells, otherwise known as the gametes or sex cells. If the modification of DNA is confined to body cells only, then the altered gene only affects the patient treated and not any offspring by that patient. Germ line gene therapy changes the genetic pool of the person treated and involves incorporation of an engineered gene into the gametes permanently altering the genes of the patient and these genes would be inherited by future generations. This type of therapy is banned in many countries such as Australia, Canada and Germany due to ethical and technical concerns, including insufficient knowledge about possible risks to future generations (Strachnan). The USA has no federal legislation specific to germ line or somatic gene therapy other than the general US Food and Drug Administration (FDA) testing regulations that apply to all therapies (Is Gene Therapy Safe?).

The types of diseases that can be treated by gene therapy are those where replacement of a single gene is able to cure the disorder. Some of the main diseases currently being researched in gene therapy studies include cancer, hemophilia, sickle cell anemia, muscular dystro...

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...he removal of millions of white blood cells called T cells, which are then genetically modified and restructured to target the cancer cells. These cells are then returned to the patient’s body where they multiply to fight off the cancer (Marchione). This and other recent clinical successes are most encouraging and have led to a renewal of interest in gene therapy with scientists, doctors, commentators all calling for continued investment in the area.

What is more, we are now seeing gene therapy treatment getting closer to commercialization. Approval of the first gene therapy treatment in either the US or Europe occurred in November 2012 when the European Commission endorsed the recommendation of the European Medicines Agency to approve Glybera, a gene therapy, which address lipoprotein lipase deficiency which is the cause of severe pancreatitis (Richards).