Question: Who Invented Gene Therapy?

Has gene therapy been used on humans?

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..

Why was gene therapy created?

The first gene therapy was approved in Europe nine years later. It was developed by UniQure, a Dutch company for treating lipoprotein lipase deficiency, a rare metabolic disease that causes acute and recurrent abdominal pain and inflammation of the pancreas.

Is gene therapy a permanent cure?

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.

How long will Gene therapy last?

Possible Effects of Gene Therapy Such trials usually last 2 to 4 years and go through several phases of research. In the United States, the U.S. Food and Drug Administration (FDA) must then approve the new therapy for the marketplace, which can take another 2 years.

What are the two types of gene therapy?

There are two types of gene therapy treatment: Somatic cell gene therapy and germline therapy. Somatic cell gene therapy involves obtaining blood cells from a person with a genetic disease and then introducing a normal gene into the defective cell (Coutts, 1998).

What countries use gene therapy?

Other countries where gene therapy trials have been performed are Italy (16 trials), China (12), Israel (7), Spain (6), Norway (4) and South Korea (13 trials), Finland and Sweden (both 3 trials), Austria, Singapore, New Zealand, and Denmark (2 trials each), and one trial each in Egypt, Mexico and Taiwan.

What was the first human genetic disease that was successfully treated with gene therapy?

Following 18 years of further research, the first gene therapy trial launched in 1990. A four-year-old girl named Ashanthi DeSilva underwent a 12-day treatment for a rare genetic disease known as severe combined immunodeficiency.

Where did the idea of gene therapy originated?

The first approved gene therapy clinical research in the US took place on 14 September 1990, at the National Institutes of Health (NIH), under the direction of William French Anderson. Four-year-old Ashanti DeSilva received treatment for a genetic defect that left her with ADA-SCID, a severe immune system deficiency.

When was the first gene therapy patient treated?

On September 14, 1990, W. French Anderson and his colleagues at the NIH performed the first approved gene therapy procedure on a four-year-old girl born with severe combined immunodeficiency (SCID) (Anderson, 1990).

How safe is gene therapy?

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.

What is the main goal of gene therapy?

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.

Who is the father of gene therapy?

W. French Anderson, M.D., a gene therapy pioneer, will be released from prison on May 17 after serving almost 12 of a 14-year sentence, having been convicted in 2006 of molesting the young daughter of one of his colleagues.