Gene Therapy to Treat Cystic Fibrosis

*please note that this is dramatization of gene therapy not how it actually occurs*
image from avensonline.org

Cell Therapy is one of the main ways to treat Cystic Fibrosis. Gene therapy is the insertion of stem cells to target bad cells and strength to weak cells. There are many forms of gene therapy. Germ line gene therapy is a very common form of gene therapy. Cystic Fibrosis is an excellent candidate for this treatment due to the fact that the disease occurs from a single gene defect, and the main organ affected is the lung which is easily accessible. To treat cystic fibrosis with gene therapy, the patient inhales medicine containing cells that hold the corrected CTFR protein. When the medicine makes its way into the lungs it is absorbed by the cells, and the corrected protein replaces the damaged one. This continues process allows the patient to breath normally, The most common stem cells used to repair the damage done by the bad CFTR protein is Embryonic Stem Cells (ESCs). There are many benefits to using ESCs due to the fact that they are still developing. ESCs can grow into almost whatever the patient needs. There is much debate about whether or not it is ethical to use ESCs because they are harvested from human embryos. One solution to this is the use of induced pluripotent stem cells (iPSCs). iPSCs are adult stem cells that have been genetically modified to be in an ESCs like state. This approach is seen as a more ethical way to treat the disease, but this method is relatively new (first seen in 2007)  and there are still many unknowns. Gene therapy is not a new thing, but there is still debate about the ethics of the treatment. Many communities, especially religious, find that it is morally wrong to attempt to play God and alter gene makeups. Also, there is speculation that altering genes will cause permanent damage to the gene pool of humans and cause modified "super" humans. Germ line gene therapy not only changes the patient, bit it also changes his or hers offspring. This has unknown effects on the human gene pool, and scientists have no way of knowing how long the effects of germ-line therapy will last for the patient.