To see if gene therapy is safe and accurate, we need to understand the process that goes on.
In most gene therapy cases, a normal gene is inserted to the genome to replace the disease-causing gene. A vector serves as a carrier molecule and is used to deliver the therapeutic gene to the patient's targeted cells. The most common vector is a virus that has been genetically altered to carry normal human DNA(3). Viruses deliver their genes in pathogenic matter. Scientists manipulate the virus genome to remove disease-causing agents and insert therapeutic ones(10). Finally, target cells are infected with the vector.
There are also options where non viral techniques are used:
1. Direct introduction of therapeutic DNA into target cells. This technique can only be used with certain tissues and requires large amounts of DNA(12).
2. An artificial lipid sphere with an aqueous core, called a liposome, which carries the therapeutic DNA, is capable of passing the DNA through the target cell's membrane.
These are different processes of performing gene therapy.