Gene therapy replaces a faulty gene or adds a new gene in an attempt to cure disease or improve your body’s ability to fight disease. Gene therapy holds promise for treating a wide range of diseases, such as cancer , cystic fibrosis, heart disease , diabetes , hemophilia and AIDS.
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 .
After initially receiving a type of gene therapy, the patient’s immune system may react to the foreign vector. Symptoms of a reaction may include fever, severe chills (called rigors), drop in blood pressure, nausea , vomiting, and headache .
Gene therapy is the introduction of genes into existing cells to prevent or cure a wide range of diseases. For example , suppose a brain tumor is forming by rapidly dividing cancer cells. The reason this tumor is forming is due to some defective or mutated gene .
But as the base of gene therapy broadened, other human disorders with genetic alternations have also been tried for treatment like rheumatoid arthritis, various types of cancer, cardiovascular (CV) diseases, renal disorders, hepatic disorders, acquired immunodeficiency syndrome (AIDS), severe combined immunodeficiency
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).
Current research is evaluating the safety of gene therapy ; future studies will test whether it is an effective treatment option. Several studies have already shown that this approach can have very serious health risks, such as toxicity, inflammation, and cancer.
The idea of germline gene therapy is controversial . While it could spare future generations in a family from having a particular genetic disorder, it might affect the development of a fetus in unexpected ways or have long-term side effects that are not yet known.
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 animal studies, gene transfer techniques achieved positive results in treating prostate, lung, and pancreatic tumors . Various approaches to gene transfer have been tested in clinical trials.
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.
Developing a gene therapy can cost an estimated $5 billion. This is more than five times the average cost of developing traditional drugs.
To date, only 1 gene therapy has been approved in the United States—Luxturna, a treatment for inherited retinal disease that carries a list price of $850,000 —but according to EvaluatePharma, the US healthcare system could see an influx of such therapies in the coming years, with combined sales forecasts of $16 billion