What is Gene Therapy and what is it used for?
Gene therapy (otherwise known as somatic gene therapy or therapeutic gene therapy) is a process that is used to correct defective genes responsible for development of various diseases. (www.accessexcellence.org/RC/AB/BA/Gene_Therapy_Overview.php) There are two main types of gene therapy; somatic (body) cell gene therapy and germ line gene therapy. As it suggests, somatic gene therapy is correcting the faulty genes of normal body cells. Germ line therapy is altering genes in gametes. Everyone on earth carries a few defective genes. Most of us are unaware of this fact however, unless we are afflicted with a genetic disease. Approximately one in ten people have, or will develop, a disease that is
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This is because viruses are able to deliver their genes to human cells (such as the influenza virus) which usually are pathogenic. Scientists have removed that original disease causing genes of the viruses and insert the needed correct genes for the human target cells. (www.ghr.nlm.nih.gov/handbook/therapy/genetherapy)
The target cells are then infected with the virus vector. The vector then unloads its genetic material (which contains the needed correct genes) to the target cell. The correct gene is then able to generate the correct functional protein and so restores the target cell to the normal state. (www.ghr.nlm.nih.gov/handbook/therapy/genetherapy)
The reason why carrier molecules are usually needed is because otherwise correct genes could only be inserted in certain tissues and would require a large amount of DNA. (www.ndsu.nodac.edu/instruct/mcclean/plsc431/students/bergeson.htm)
The second method is called ex vivo technique, and involves surgically removing cells from affected tissues, injecting the new DNA that will correct the disease into the cells and letting the cells divide by mitosis in an artificial medium. The new, corrected tissues are then placed back into the affected area of the patient. (Lyon, 1995) The reason why this method is not as often used as in vivo is due to the fact that the patient will then have to undergo two separate surgeries, one to get the tissue, and one to put the fixed tissue back.
There are two main groups of gene therapy and they are germ line gene therapy and somatic gene therapy (Baksh, 2007). Germ line therapy consists of germ cells being modified by the introduction of functional genes, which are ordinarily integrated into their genomes (Garbutt et al., 2011). DNA encodes the therapeutic gene and replaces the mutated gene so the new gene can treat the disease. Germ line
Human gene therapy is a procedure that is currently being used to help fight diseases with no known cures. Gene testing removes a gene that causes a disease and replaces it with a healthy copy of that gene. By placing a healthy copy of the gene inside the patient’s body, that gene can help fight any diseases, such as different forms of cancer, inherited disorders, immune system discords or viral infections (AIDS). Once inside the cell, the genes produce what the patient lacks, it kills the diseased cells and it activates cells in the immune system.
Gene therapy provides many benefits to the patients who undergo it. Biotechnologists believe that a genetic disease can be removed with this treatment. For example, on the U.S. National Library of Medicine website, it states, “Researchers are testing several approaches to gene therapy, including: Replacing a mutated gene that causes disease with a healthy copy of the gene. Inactivating, or ‘knocking out,’ a mutated gene that
The virus lays latent for a week until it causes cancer, and the host dies within a few days, as the virus continues to inject its DNA. Because the virus mutates to look like the host cells, the virus looks and behaves differently in each person. Everything that makes someone unique is determined by the 1% of their 20,000 genes, and the virus takes advantage of that difference, making personalized medicine the only cure. Being able to tailor make an antibiotic, and nanoparticles to deliver the antibiotic so it would target just the virus, would save millions of
Gene therapy is the insertion of normal or genetically altered genes into cells to replace defective genes. The gene is usually inserted into
Scientists have projected there to be many benefits to gene therapy in the near future. For starters, gene therapy could cure diseases like cancer, aids, as well as thousands of genetic disorders (Will). Sara Goering, a philosopher concerned with biomedical ethics, argues throughout “Gene Therapies and the Pursuit of a Better Human” that germline therapy could mean disorders would be eliminated for good, leading to the relief of pain for many. It poses the possibility to eliminate human suffering at the root, and in turn, better humanity. Throughout society, people already focus on ‘enhancing’ their kids through vaccines and vitamins. Kids are out through surgeries because they are believed to be what is best. Goering points out that because of people’s willingness for treatments like this, there really are not many moral concerns prevalent (Goering 403-40). For many diseases, treatments that people already feel comfortable with will not work as effectively (“List”). Currently, there are no real cures for genetic disorders besides replacing defective
Technology has advanced greatly in the last decade, everyday scientists are developing techniques to cure diseases. Millions of people die every year due to harmful diseases with no cure. The term virus holds a negative meaning to humans. However, scientists are now able to manipulate viruses in a beneficial way. The Talimogene Laherparepvec virus, which came from herpes, has been genetically modified in order to treat melanoma patients (Ledford, 2015).
To create the best version of virotherapy, scientists are discovering two ways to be confident the viruses hit target cells with no other harm to other cells. One strategy virotherapy researchers are exploring is called transductional targeting. With this form of targeting cancer cells, researchers are modifying the viruses used so they are only attracted to only cancerous cells to contaminate them. To prevent the viruses from entering normal cells, transductional targeting works by binding “adapter” molecules onto the antibodies, proteins on the outside of virus, or tweaking the antibodies so that they will not allow viruses to enter normal cells and rather let them gain up on tumor cells. The second strategy discussed in the article is named transcriptional targeting. The goal of this form of targeting is to modify the viruses
There are two types of gene therapy; germline gene and somatic gene therapy. Germline gene therapy is considered to be a safer option for humans. This method makes changes to the gamete cells that are used in the reproductive process thus, the functional genes need to be inserted into the chromosomes. Somatic gene therapy is when genes are transferred to body cells by inserting vectors into a person’s body. There are three things that gene therapy can do; replace a mutated gene that causes a disease with a healthy gene, deactivate a mutated gene that is not able to function properly, or introduce a new gene into the body to prevent diseases. There has been a clinical trial in the past where over 3000 people were treated with gene therapy and the results were good however, this treatment method has serious health
In 1990 the first gene therapy procedure gained approval and a four-year-old girl with SCIDs disease was finally able to fight off a simple cold. She is now able to live a normal life with the help of continued treatment of gene therapy. Although gene therapy is an innovative and ideally favorable procedure when it comes to treating diseases, cancer, or inherited disorders, it is still a delicate procedure and is continuously studied to insure it is not only effective but also and most importantly safe. In order to insure the safety of patients a gene therapy drug must get the approval of the FDA before becoming available, as every drug must do. Since gene therapy is still very experimental; there are only a few gene therapy drugs that have been approved by the FDA to treat patients. However, on August 30th, 2017 history was made when the FDA approved Kymriah, the first CAR-T cell therapy drug to be available in the United States. Kymriah is a one-time treatment for patients with B-cell acute lymphoblastic leukemia, it was developed through the research collaboration of Novartis and the University of Pennsylvania, and it is manufactured for each individual patient. Kymriah’s only drawback would be the price tag of $475,000 for a course of treatment, however Novartis plans to wave the fee for patients where Kymriah is not successful.
Gene therapy is an experimental form of treatment, where normal genes are placed into missing or defective ones in order to correct or alter genetic disorders. In other terms, it is the therapeutic delivery of nucleic acid polymers into a patient 's cells as a drug to treat disease (Gennady, 2015). After many attempts at modifying human DNA by nuclear gene transfer as well as insertion of human DNA in the nuclear genome, the first successful gene therapy was in 1990 where it was used to cure four-year-old Ashanti DaSilva who had Severe Combined Immune Deficiency (IDF 2013 National Conference). Since then gene therapy has been used to cure and treat serious diseases such as Chronic Granulomatus Disorder, Hemophilia, and a wide variety of Cancers, and other genetic and acquired diseases (American Society of GENE & CELL THERAPHY, online).
With our current society rapidly advancing in both the technological and medicinal world, many new treatments have been born that can be used to treat genetic conditions that regular antibiotics and surgeries simply have no positive or progressive effect. Gene therapy is when a newly developed gene is transferred, or introduced, to an already existing gene, it is the attempt to treat one’s genetic disorder at the molecular level and could significantly improve the human population and provides hope to people with disabilities. Gene therapy goes straight to the source and alleviates symptoms of the disorder. The gene receiving this new development is in some shape or form, mutated, or defective. If the gene therapy is a
Scientists and doctors alike have long been looking for cures to diseases like cancer. For years now, there have been countless fundraisers to cure this and cure that. And yet, it seems as if there's never any progress. There may be a brief headline in the news about a new discovery, but they always seem to never go anywhere. Maybe the problem is that they've been looking in the wrong places. Maybe the cure doesn't lie on a medicinal level, but instead the genetic level, in genetic engineering and gene therapy. Direct manipulation of an organism's genome can be used in gene therapy as a means to alter genes, causing both positive and negatives effects as well as discussions about the ethical boundaries of modern
Gene Therapy is an experimental form of treatment that aims to get rid of genetic conditions at their source. It also may be used to replace a faulty gene with a working version or introduce a new gene to cure a condition or modify its effects. ( Victoria State Government, 2011) It is designed to introduce genetic material into cells to compensate for abnormal genes or to create a beneficial protein within the gene. If a mutated or incomplete gene creates a protein needed that is faulty or not present, gene therapy has the ability to introduce a copy of that specific gene to restore the function of the protein within that gene.
The third technique is in vivo (or "in the living body"). This therapy does not exist yet but is the therapy of the future.