Some gene therapy research indicates gene therapy may worsen symptoms or cause them to last longer. Additionally, complications of certain gene therapies may include cancer , toxicity and inflammation .
The ethical questions surrounding gene therapy include: How can “good” and “bad” uses of gene therapy be distinguished? Who decides which traits are normal and which constitute a disability or disorder? Will the high costs of gene therapy make it available only to the wealthy?
Although gene therapy is a promising treatment option for a number of diseases (including inherited disorders , some types of cancer, and certain viral infections), the technique remains risky and is still under study to make sure that it will be safe and effective.
Human gene therapy has been attempted on somatic (body) cells for diseases such as cystic fibrosis, adenosine deaminase deficiency, familial hypercholesterolemia, cancer, and severe combined immunodeficiency (SCID) syndrome.
Gene therapy offers the possibility of a permanent cure for any of the more than 10,000 human diseases caused by a defect in a single gene . Among these diseases, the hemophilias represent an ideal target, and studies in both animals and humans have provided evidence that a permanent cure for hemophilia is within reach.
Gene therapy is a potential approach to the treatment of genetic disorders in humans . This is a technique where the absent or faulty gene is replaced by a working gene , so the body can make the correct enzyme or protein and consequently eliminate the root cause of the disease (BIO, 1990).
Gene therapy does have risks and limitations. The viruses and other agents used to deliver the “good” genes can affect more than the cells for which they’re intended. If a gene is added to DNA, it could be put in the wrong place, which could potentially cause cancer or other damage.
The main reason gene therapy is so expensive , however, may be the paradigm used in the price-setting strategy. The cost of production is weighed against the value of a life saved or the improved quality of life over a specified timeframe.
In many countries there is a de facto moratorium on human germ line and embryo editing because such work is illegal. It is also completely unethical , not least of all because of lack of consent. The nontherapeutic use of gene editing on human embryos was and remains unethical and illegal on every level.
Many genetic disorders result from gene changes that are present in essentially every cell in the body. As a result, these disorders often affect many body systems, and most cannot be cured . However, approaches may be available to treat or manage some of the associated signs and symptoms.
Developing a gene therapy can cost an estimated $5 billion. This is more than five times the average cost of developing traditional drugs.
The U.S. Food and Drug Administration ( FDA ) has approved only a limited number of gene therapy products for sale in the United States. Hundreds of research studies (clinical trials) are under way to test gene therapy as a treatment for genetic conditions, cancer, and HIV/AIDS.
Gene therapy holds promise for treating a wide range of diseases, such as cancer , cystic fibrosis , heart disease , diabetes , hemophilia and AIDS . Researchers are still studying how and when to use gene therapy. Currently, in the United States, gene therapy is available only as part of a clinical trial.
Gene therapy is designed to introduce genetic material into cells to compensate for abnormal genes or to make a beneficial protein. If a mutated gene causes a necessary protein to be faulty or missing, gene therapy may be able to introduce a normal copy of the gene to restore the function of the protein.
Gene therapy aims to address specific mutations in an individual’s genetic instructions, allowing the body to produce the proteins it needs.