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The Goal of Gene Addition Therapy and How It Works

What is the goal of gene addition? 

By aiding the body’s ability to fight complex diseases, gene addition aims to improve outcomes for people with complex disorders and infectious diseases, either on its own or in combination with other non-genetic treatments. For example, gene therapy for cancer may be used in combination with chemotherapy.

As more research is done, progress using gene addition for specific diseases may lead to new treatments for other similar diseases.

Discover the science of gene addition

After scientists have created the gene to be added to the cell, a vector is used to carry the new gene to the cell nucleus where the cell’s DNA is stored. This new gene may stay as a separate piece of DNA, or it may become a part of the cell’s own DNA. Once this gene is added, the cell can make the protein that the missing or faulty gene has failed to make, helping the cell work properly. 

Gene addition therapies may work directly inside the body, or cells may be removed, treated, and returned to the body.