How is it done? |
How Does Gene Therapy Work? |
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In order to travel properly, the new gene must be carried within a molecule called a vector. The most common vectors that are being tested are viruses. Naturally, viruses have a tendency to invade cells and inject their genetic material into the normal cell's genome. While being used as a vector, the virus' own genes are replaced with the therapeutic gene assigned to correct the damaged one. When the virus attacks the cell, it will implant the healthy genetic material it carries. Popular viruses that are used for this treatment include HIV and adenoviruses (ex. common cold.) The transfer will result in the cell now carrying the new gene that will correct the problem caused by the malfunctioning gene- if successful. Think of it like physiotherapy for genetics.
There are many non-viral vectors also being tested for gene therapy. Artificial lipid spheres called liposomes, are samples of DNA attached to a molecule that will bind to a receptor on the target cell with the faulty gene. The actual transfer of the new gene into the target cell can happen in two ways: ex vivo and in vivo. The ex vivo approach is when doctors extract cells from a patient’s body and healthy genes are transferred into the cells. After the cells with corrected genes have grown in a laboratory, they are returned to the patient. The in vivo approach involves the vector containing the therapeutic gene targeting cells directly in the patient’s body. It is currently being studied for the treatment of many diseases such as Huntington's disease, cancer and cystic fibrosis.
Click on the Video Below for a Demonstration
There are many non-viral vectors also being tested for gene therapy. Artificial lipid spheres called liposomes, are samples of DNA attached to a molecule that will bind to a receptor on the target cell with the faulty gene. The actual transfer of the new gene into the target cell can happen in two ways: ex vivo and in vivo. The ex vivo approach is when doctors extract cells from a patient’s body and healthy genes are transferred into the cells. After the cells with corrected genes have grown in a laboratory, they are returned to the patient. The in vivo approach involves the vector containing the therapeutic gene targeting cells directly in the patient’s body. It is currently being studied for the treatment of many diseases such as Huntington's disease, cancer and cystic fibrosis.
Click on the Video Below for a Demonstration
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