Genetic Disorders: The Pros And Cons Of Gene Therapy

Gene therapy is a technique that is still in the experimental stages. It uses genes to treat or prevent disease. They are hoping that in the future gene therapy will help doctors treat a disorder by inserting a gene into a patient’s cells rather than using drugs or surgery. Researchers are trying many different approaches to gene therapy. Some include, Replacing a mutated gene that causes disease with a healthy copy of the gene, Inactivating, or knocking out a mutated gene that is functioning improperly, introducing a new gene into the body to help fight a disease. Gene therapy is a very risky treatment. The technique is still under a study to make sure that gene therapy is safe and highly effective. Although gene therapy is a promising treatment

Such as, Gene delivery and activation, Immune response, disrupting important genes in target cells, and Commercial viability. Gene delivery and activation is when you can deliver a normal gene to a large number of cells in a tissue. Once the gene reaches the correct destination, it must be activated. To make the protein that it is encoded with. It must also stay on, cells can shut down genes that are showing unusual behaviors. But, if you introduce changes to the wrong cells you could cause health problems for the patient. Immune response, immune systems are normally very good at fighting off bacteria and viruses. When delivering the Gene’s they must be able to avoid the body’s immune response they may be able to do this by giving patients drugs to temporarily suppress the immune system during treatment. If it triggers an immune response it could cause serious illness or even death. Disrupting important genes in target cells, the cells “stich” itself into the cell’s DNA. But, if it stiches itself into the wrong DNA it could cause deadly consequences. There was a study done of this where researchers tested a gene therapy treatment that would restore the function of a crucial gene, gamma c, in cells of the immune system. The treatment appeared very successful, restoring immune function to the children who received it. Five years later they found that the gamma c gene had stitched

Ribozymes are RNA molecules that act as enzymes. They act as molecular scissors that cut RNA. In ribozyme gene therapy, ribozymes are designed to find and destroy mRNA encoded by the mutated gene so that no protein can be made from it.

Genetically modifying immune cells to target specific molecules, this is when the immune system makes large numbers of white blood cells. Researchers have learned how to confine an individual’s immune cells. They can also make improvements on the cells so that it will release a “disease-fighting product”.

Several inherited immune deficiencies have been treated successfully with gene therapy. Most commonly, blood stem cells are removed from patients, and retroviruses (a group of RNA viruses that insert a DNA copy of their genome into the host cell in order to replicate) are used to deliver working copies of the defective genes. After the genes have been delivered, the stem cells are returned to the patient. Because the cells are cared for outside the patient's body, the virus will infect and transfer the gene to only the desired target cells. Severe Combined Immune Deficiency (SCID) was one of the first genetic disorders to be treated successfully with gene therapy, this proved that the approach could in fact work. However, the first clinical trials ended when the viral vector triggered leukemia in some patients. Since then, researchers safer viral vectors that are much less