Essay on A Look at Gene Therapy

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.

First of all, it could be possible to change disease genes and change the risk of getting diseases for future generations (Doc. 3). It might also be possible “to install genes that offer lifelong protection against infection.” (Doc. 3). It is also voted most popular to genetically change humans for the cause of reducing the risk of serious diseases than to make the baby more intelligent (Doc. 4). Lastly, germline editing could also potentially decrease or eliminate many serious genetic diseases, which would reduce human suffering worldwide (OI).

DNA has about 12 million single nucleotide polymorphisms and thousands of copy number variants, most of which are not harmful. Sometimes these mutations in genes can cause genetic disorders that end up altering or inhibiting protein function. Gene Therapy focuses on correcting the mutated genes usually by inserting a normal gene into the genome. Researchers treat these diseases this way by repairing dysfunctional gene or by providing copies of the missing gene. They isolate the normal DNA and package it into a vector. Other ways include replacing the abnormal gene with a normal one, repair the mutated gene, or altering the regulation of a certain gene. As of right now, reparative results have been marginal, there is potential of gene therapy treating single-gene disorders.

I believe that there is nothing wrong with the therapeutic gene modification when it involves somatic cells. I think that it is our moral responsibility to do what we can to help save the lives of people who are suffering. The doctors who would be proscribing the treatment have all made a hippocratic oath so it would be their moral duty to follow through on using this particular treatment. Therapeutic gene manipulation with somatic cells would break any moral, ethical, or legal laws. But the other forms of genetic manipulation are a little tricky. Therapeutic gene manipulation of germ cells can be a useful treatment of disease but its effect plays a role in a larger scope then just the patient alone. I think that it poses a greater risk to the whole of humanity of we allow changes to germ cells and future generations. It would open the door to all other types of genetic manipulation. So rules have to be put in place so no abuse of medical treatment occurs. Finally there should not be any type of enhancement gene modification occurring. I think that it will overall produce more harm then good. It is not a medical procedure that is used to help people who are suffering it would simply be a mechanism that would only play a negative role in human development, simultaneously breaking many ethical rules along the way. Genetic

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

Humans have been able to use the principles of DNA replication, gene transfer and gene expression (as observed in nature) as tools to manipulate specific genes towards preferred outcomes. An advantage of being able to manipulate genetic therapy is the ability to eliminate/cure genetic disease/disorders with otherwise no other cure. Humans are able to remove the genes causing the genetic disorder from the gene pool of populations, allowing organisms to live longer and healthier lives. Two ways of manipulating genetic transfer which will be explored further in this report are selective breeding and gene therapy.

Fifty years after the idea of gene therapy was first proposed, gene therapy has become a possible treatment for a couple different diseases. Before this treatment was approved, some serious unfavorable effects were found in clinical trials. However, these effects fueled more basic research in order to improve, in efficiency and safety. Gene therapy has been used for patients with blindness, neuromuscular disease, hemophilia, immunodeficiencies, and cancer.

Whether gene therapy should be allowed to be used in practice is currently a very controversial topic. In my opinion, I do not think that it should be allowed. Gene therapy is a technique where genetic material is inserted into a patient to replace missing or defective genes. Gene therapy can be further broken down into two categories: somatic and germline. From there, somatic gene therapy can be approached in two ways: in-vivo and ex-vivo. For the process of gene therapy to begin, the proper vector needs to be acquired. Once a vector is chosen, the vector delivers the new gene into the target cell. Then, the target cell becomes infected with the vector. Next, the vector’s genetic material is inserted into the target cell. Then finally, functional

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).

There are three techniques that researchers are working on. The first and most common is ex vivo ( or "outside the living body") therapy. The defective cells are removed from the patient and replaced with the normal DNA before returning to the body. This therapy targets the blood cells because many genetic defects alter the functioning of one type of these cells or another. But since blood cells have limited life spans follow-up treatments are required. Future efforts will most likely target stem cells of the bone marrow. Stem cells are ideal for gene therapy because they appear to be immortal. Researchers have obtained stem cells from human bone marrow, but they are having difficulties getting genes into the cell as well as inducing the cells to produce many new blood cells (Anderson, 1995).

If a treatment is implicated before the child is born – germline gene therapy – he or she could lose all trace of that defective gene, and therefore wouldn’t pass the disease on to future generations. In somatic gene therapy, treatment is conducted when the patient is an

Gene Therapy is described as the “treatment of a genetic disorder by replacing, supplementing, or manipulating the expression of abnormal genes with normally functioning genes” by the Genetics Home Reference, a branch of the NIH. This is a part of a wave of modern medicine which is changing the way we look at diseases, the causes of diseases and new ways that we can treat disorders that previously had previously seemed to have no cure. Gene Therapy has been trialled in a variety of single gene mutations that cause known diseases, it is not yet a full time option for many diseases at this point in time but in the future may cure many life threatening diseases. Biology online defines selective breeding as “The intentional breeding of organisms with desirable trait in an attempt to produce offspring with similar desirable

This would mean that this testing would be more affordable for the commoner. Once testing has been completed and data interpreted, someone will know if they are a carrier for a genetic disorder. If it turns out that they are, instead of waiting for symptoms to arise, genetic treatment of the disease can be done to save the person’s life before it is even in jeopardy. This could prove to be very helpful. People spend thousands of dollars on treatments for a genetic disorder, and the treatments do not even fully rid the patient of the disease. The patient will have to take the medication for the rest of their life to calm the symptoms of that disease. With the advancement of gene therapy, a patient could receive a minimal number of treatments which will not just calm the symptoms but rid the patient of their genetic disorder completely. This, in the long run, could save the patient time, money, suffering, and ultimately it will save their life. This is only the beginning. As time passes, doctors will have the knowledge to save many more lives in the same manner.