gene ther·a·py
noun
the transplantation of normal genes into cells in place of missing or defective ones in order to correct genetic disorders.
Through the years, biology has made a multitude of significant breakthroughs that change the way we live today. One of the many incredible successes includes gene therapy. Commonly used for medical purposes, gene therapy is the process of gene insertion to an individual’s cells. Gene therapy is used for many purposes, especially with hereditary diseases when mutant alleles are replaced with properly functioning ones.
Although gene therapy is just in it’s early stages, this newly found treatment method has already proven to work for a number of inherited diseases and even some types of cancer. Gene therapy is working towards being a promising treatment option for muscular dystrophy and cystic fibrosis. As of today, gene therapy is only available in research settings. Scientists are evolving this new way of treatment that potentially can knock out all drugs and surgeries. Currently gene therapy is a very dangerous concept, and can put patients in risk of toxicity and inflammation. Since it is such a new idea to us, gene therapy is only being used to fight diseases that have no other found cures. more about gene therapy
If we tried inserting a gene directly into an individual’s cells, it typically would not work. Scientists have discovered a new way of delivering the genes, using a carrier called a vector. Quite often, they use viruses as vectors to infect the cell to allow a new gene. The vector can either be injected, or a sample of cells can be removed from a target area on one individual. The vector would be added to the cells in a laboratory setting, then inserted back into the individuals body. The cells should start producing regular proteins, to potentially stop the mutant. Not that long ago, scientists discovered that mutant genes had found a way to work around the gene therapy and still deliver the mutant genes to cells. Scientists are working on harnessing that problem by manipulating the mutant genome to deliver regular cells. vectors and more
Since gene therapy can cure some hereditary diseases, is it still possible to pass these diseases off to your kids? Current research has been made up of gene therapy where the target is either bone marrow or blood cells. In this case, the therapy would not be passed down through reproduction. In the future, scientists will aim to target the therapy into the eggs or sperm of an individual, which will hopefully allow the inserted gene to be passed down. This is obviously an important approach, (called germline therapy) because it provides the opportunity to completely eliminate certain diseases. There are also negative affects too, that can be very unpredictable such as affecting the development of a fetus. Because there are so many concerns, the US federal government refuses to fund germline therapy research. details
Germline gene therapy is looking at this treatment from one perspective, but lets take a look at somatic gene therapy. Somatic gene therapy is the opposite of germline, where the normal DNA is transferred to a part of the body that does not produce sperm or eggs. Any effects that may happen on the parent would not be passed down to the child. Scientists are currently working with somatic gene therapy, in attempt to make it safer and efficient. Since somatic therapy does not target any reproduction essentials, there are no concerns on affecting the fetus or new life. This makes it much easier to study, which is why scientists are focusing on somatic therapy, hoping to make germline work in the future.
My dad had genetic colon cancer 2 years ago. His grandfather had the same type of cancer far in the past. My dad managed to get rid of his cancer without chemo, and got his colon removed. I will have to go for regular tests starting in just a few years, to see if I have the same virus and if they can do anything to prevent it. I was wondering if gene therapy could do anything to stop this virus in it’s early stages, and for my genes to start producing regular proteins. Gene therapy has been tested successfully for certain types of cancer, but is not ready for testing on real human lives yet given it has only been executed in research lab settings. Possibly if I have to get treatment in the future, gene therapy will have advanced enough research to actually safely try.
Though all concepts of biology have been exceptional parts of our growing world, gene therapy is one of the most impactful and life-changing. We seised to imagine the possibilities that came along with one bright idea. Gene therapy has already proven to prevent hundreds of genetic viruses, and the research and tests have barely even started.
https://www.sciencedaily.com/terms/gene_therapy.htm