Gene editing refers to a technique that is designed to change genes to treat, prevent or potentially cure certain diseases. The focus of gene editing today is typically in diseases that are caused by mutations in a single gene. In these conditions, a person’s own gene has a mutation that affects the normal biological function of the protein made from this gene. Gene editing alters the mutated gene to allow it to function properly and can potentially override the effects of a disease-causing mutation in a person’s cells.
Gene editing can potentially work by using something called a vector (a kind of small, cellular package containing genetic information) to introduce, or transfer, a new gene into the body’s cells. The concept behind this is to either replace the malfunctioning gene or to add a new gene that can help improve the body. A gene therapy is inserted into the body typically via an I.V. infusion.
Refer a Friend
Do you know someone between the ages of 18-55 who has PKU? They may be eligible to participate in the pheEDIT study. Consider sharing information with them about how they can learn more.
