Why gene therapy

Cystic fibrosis is a relatively common genetic condition in which the lungs and digestive system become clogged with thick, sticky mucus. Some children with severe combined immunodeficiency SCID , a genetic disorder characterised by a reduced number of immune cells, have been treated using gene therapy.

Germline gene therapy targets the reproductive cells, meaning any changes made to the DNA will be passed on to the next generation. Consequently, the practice has dramatically divided opinion.

Genome editing is a way of making specific changes to the DNA of a cell or organism. If you have any other comments or suggestions, please let us know at comment yourgenome. Can you spare minutes to tell us what you think of this website? Open survey. In: Facts Targeting Disease. Gene therapy uses sections of DNA usually genes to treat or prevent disease.

The DNA is carefully selected to correct the effect of a mutated gene that is causing disease. The technique was first developed in but has, so far, had limited success in treating human diseases. Gene therapy may be a promising treatment option for some genetic diseases , including muscular dystrophy and cystic fibrosis.

Human gene therapy seeks to modify or manipulate the expression of a gene or to alter the biological properties of living cells for therapeutic use 1. Gene therapies can work by several mechanisms:. Gene therapy products are being studied to treat diseases including cancer, genetic diseases, and infectious diseases. Clinical studies in humans require the submission of an investigational new drug application IND prior to initiating clinical studies in the United States.

All viral vectors are tested many times for safety prior to being used. The vector can either be delivered outside the body ex-vivo treatment or the vectors can be injected into the body in-vivo treatment. Learn more about different gene therapy approaches, challenges, risks, and benefits. There are some FDA-approved gene therapies currently available to patients with diseases such as SMA , certain blood cancers , and an inherited eye disease.

However, most gene therapies are in clinical trials. Clinical trials are a required research process to understand how a treatment interacts with the body, and if it is safe and effective before it can be made available to the public. Gene therapy is aimed at diseases that are rare and often life threatening or debilitating.

As of now, there are around 7, rare diseases, affecting a total of approximately one in ten people. Many of these rare diseases are caused by a simple genetic mutation inherited from one or both parents. Of gene therapies up for FDA approval over the next five years, 45 percent are anticipated to focus on cancer treatments nearly half and 38 percent nearly 4 out of 10 are expected to treat rare inherited genetic disorders.

Some of these inherited diseases include but are not limited to :. Hematology blood disorders like sickle cell disease. Neurological disorders that affect the brain and spinal cord.