MoBio Gene Therapy Chapter 10

Gene therapy is a genetic engineering technique that uses DNA as an agent to treat genetic diseases. The most common form involves the introduction of a normal gene at a random location in the genome to compensate for the loss of function due to its mutation. The normal gene is usually packaged within a "vector", such as an adenovirus vector, to deliver the foreign DNA into the genome of the targeted cell. Once inside the cell, the normal gene can make functional protein to treat a disease (Figure 10-C-1).

In 2012, Glybera became the first gene therapy treatment to be approved for clinical use in Europe and the United States. Glybera compensates for lipoprotein lipase deficiency (LPLD), which can cause severe pancreatitis.

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Figure 10-C-1. Gene therapy using an adenovirus vector. [Credit: NIH]