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.

What are the two main types of gene therapy?

There are two types of gene therapy treatment: Somatic cell gene therapy and germline therapy.

Who was the first gene therapy patient?

In 1990, 4-year-old Ashanthi de Silva became the first gene therapy success story. She was born with a severe combined immunodeficiency (SCID) due to lack of the enzyme adenosine deaminase (ADA). Without ADA, her T cells died off, leaving her unable to fight infections.

What was the first gene therapy?

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).

Has gene therapy cured anyone?

Gene-fixing treatments have now cured a number of patients with cancer and rare diseases. It was a notable year for gene therapy. The first such treatments in the U.S. came to market this year after winning approval from the Food and Drug Administration.

What are the disadvantages of gene therapy?

Cons

• Expensive. Gene therapy can be extremely pricey, making it inaccessible for some people.
• Experimental. Gene therapy is relatively new and there’s still a lot about it that we don’t know.
• Potentially dangerous.
• Ethical issues.
• May cause infection.

What does gene therapy cost?

A one-time treatment of the life-saving drug for a young child costs US$2.1 million. While Zolgensma’s exorbitant price is an outlier today, by the end of the decade there’ll be dozens of cell and gene therapies, costing hundreds of thousands to millions of dollars for a single dose.

Who is a good candidate for gene therapy?

Gene therapy is particularly attractive for diseases that currently do not have satisfactory treatment options and probably easier for monogenic disorders than for complex diseases. Cystic fibrosis (CF) fulfills these criteria and is therefore a good candidate for gene therapy-based treatment.