Gene Therapy What is it? If genes don’t produce the right protein or don’t produce it correctly a person can have a genetic disorder. Gene therapy is an experimental technique that involves the transfer of a working copy of a gene into a cell to repair or replace a faulty gene so it is able to produce functioning proteins again. There are several approaches. The most common one is to insert a normal gene to replace the non-functional gene. Another approach is to swap a non-functional gene with a normal gene or the faulty ene could be repaired through selective mutations.
Also genes can be switched on and off to a certain degree. How does gene therapy work? A carrier gene, called a Vector brings the normal gene into the target cell. Currently a virus is commonly used for that. The virus “infects” the target area with the healthy gene and so the cells can produce functioning proteins again. The genes can also be directly introduced to the target cells; this can only used in some tissues though. Also an artificial sphere, called
Liposome, can be used which carries the DNA and is capable of passing the DNA through the target cell’s membrane. Another way is to chemically link the DNA to a molecule that will bind to specific cell receptors. Then the DNA will be passed into the target cell. This though seems to be less effective than other options. Advantages: ??? has the potential to restore health to many who are suffering with the disease Disadvantages: controversy surrounding the medical treatment ??? relative newness of the technology