how does gene therapy work?
Gene therapy is the introduction of a new gene to compensate for the abnormal gene. If a mutated gene causes a protein to be malfunctioning or missing, gene therapy may be a treatment that is able restore the function of the protein. [4]
If you insert a gene directly into a cell, it will not work. Instead, a carrier called a vector is genetically engineered to deliver the gene. Certain viruses are used as vectors because they can deliver the new gene by infecting the cell. The viruses are genetically modified so that a disease is not introduced when injected into humans. [4]
The vector can either be injected or given directly into a specific tissue in the human body, where it is taken up by individual cells. Alternately, a sample of the patient's cells can be removed and exposed to the vector. The cells containing the vector are then returned to the patient. If the treatment is successful, the new gene delivered by the vector will make a functioning protein. [4]
If you insert a gene directly into a cell, it will not work. Instead, a carrier called a vector is genetically engineered to deliver the gene. Certain viruses are used as vectors because they can deliver the new gene by infecting the cell. The viruses are genetically modified so that a disease is not introduced when injected into humans. [4]
The vector can either be injected or given directly into a specific tissue in the human body, where it is taken up by individual cells. Alternately, a sample of the patient's cells can be removed and exposed to the vector. The cells containing the vector are then returned to the patient. If the treatment is successful, the new gene delivered by the vector will make a functioning protein. [4]