Is gene therapy helpful or harmful?

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.

Why is gene therapy potentially dangerous?

This technique presents the following risks: Unwanted immune system reaction. Your body’s immune system may see the newly introduced viruses as intruders and attack them. This may cause inflammation and, in severe cases, organ failure.

What kind of gene therapy is currently available?

Clinical trials are investigating gene therapy for the treatment of cancer, age-related macular degeneration and other eye diseases, certain genetic conditions and HIV/AIDS. Currently, one gene therapy medication, Luxturna®, has been approved by the U.S. Food and Drug Administration (FDA) for use in the United States.

Is gene therapy ethically acceptable?

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.

How is CRISPR used today?

Using the CRISPR system, researchers can precisely edit any target DNA locus – a feat that was not achievable using other gene editing tools. The possibility to edit a disease mutation to correct genetic errors creates opportunities for treating conditions that have long eluded the medical research community.

What are the safety issues with gene therapy?

Another concern associated with gene therapy is that it represents a form of eugenics, which aims to improve future generations through the selection of desired traits. While some have argued that gene therapy is eugenic, others claim that it is a treatment that can be adopted to avoid disability.

What diseases are treated by gene therapy?

Gene therapies for sickle cell disease, beta-thalessemia, and Fanconi anemia all show promise and are at various stages of clinical trials. Among the most developed gene therapies, Learn more about available treatments and others on the horizon for LCA, retinitis pegmentosa, choroideremia and more.

What are the negative effects of gene therapy?

There could be unwanted immune system reactions. The body’s immune system might see the various viruses that we use to replace unwanted genes as invaders that must be extinguished

Current gene therapy methods can sometimes target the wrong cells.

The delivery viruses might recover their ability to create disease.

What are the benefits and risks of gene therapy?

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.