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Gene Addition Therapy


About gene addition

While gene replacement can work to treat monogenic diseases (caused by a single missing or malfunctioning gene), targeting a single gene may not work for complex disorders or infectious diseases. Complex disorders (such as cancer and heart disease) are caused by multiple genes as well as environmental factors and are common and can be debilitating. 

Gene addition supplies the body with a new, functional gene that can help the body address the defects causing the disease and fight one or more of the disease-causing agents. For example, a common factor in heart failure is a defect in the ability of the cardiac muscle cells to transport calcium. Adding a gene that helps the body produce more of a protein needed for transporting calcium may improve cardiac function.


Viral vectors may also be altered to only replicate in tumor cells. Using such vectors, gene addition therapy can deliver genes that could trigger the immune system to attack only unhealthy cancer cells. With infectious diseases, like HIV or influenza, adding a gene can instruct the body to produce antibodies to target the virus that causes the disease, which may allow the body to detect and clear the infection.

Next  Learn more about the goal of gene addition and how it works