The Process of Human 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

Just in case you are a little shaky on the topic of gene therapy I will briefly explain. Gene therapy is a type of treatment used

Gene therapy is an experimental treatment, which is used to replace mutated, improperly functioning genes with regular DNA. There are several gene therapy methods, most of which involve using a vector to transport the corrected gene into targeted cells. Retro viruses, adeno viruses, adeno ascosciated viruses and liposomes are commonly used as vectors. Naked DNA (DNA without

Gene therapy is an experimental medical procedure that uses the genes in our bodies to either treat or prevent disease by introducing new genetic material into human cells (ie. cancer cells) to create the necessary proteins that allows the cell become healthy (SOURCE). Gene therapy can also be used as an alternative to other medical disorders rather than having a surgical procedure done or using drugs such as chemotherapy. For example, Joseph Panno, research

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

As years go by and technology advances, many medical techniques that failed in the past are starting to seem more possible. Researchers are discovering that a technique called gene therapy is able to help prevent and reverse the effect of many conditions. Originally in the early 1900's, gene therapy failed in

Gene therapy is an experimental technique that uses genes to treat or prevent disease. Currently, there are a lot of studies and experiments that allow doctors to treat a disorder by inserting a gene into a patient’s cells instead of using drugs or direct surgery. Researchers are testing several approaches to gene therapy, such as 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, and helping to regenerate precious cells. Although gene therapy has a very active and promising approach for a number of disease, there are still several risks and side effects. Hence, the therapy

There are two types of gene therapy: somatic gene therapy (SGT) and germline gene therapy (GGT). SGT involves treating a single individual by transferring therapeutic genes into somatic cells, most commonly bone marrow. GGT inserts genes into reproductive cells or embryos to fix genetic defects in the germ line. In other words, the genetic makeup of future generations is changed to prevent the recurrence of certain genetic disorders. Although gene therapy as a whole is controversial, germline gene therapy especially raises ethical concerns because it is capable of manipulating the human genome of a germ line. First, the risk is without the consent of future generations, and second, it may dangerously lead to genetic enhancement. Genetic enhancement has its own set of ethical issues, ranging from eugenics and social discrimination to messing with nature. Therefore, the potential consequences of gene therapy have raised a hotly contested bioethical question of whether gene therapy should be allowed in the future.

Since 1990, gene therapy had been tested in human clinical trials for treating such diseases as severe combined immunodeficiency disease (SCID), cystic fibrosis, Canavan's disease, and Gaucher's disease. ("Gene Therapy," n.d.). In the United States, gene therapy is only available in a research setting.

The process of gene therapy is the introduction of a “good,” or normal, gene into a person who is affected by a “bad,” or mutated, gene. The doctor is manipulating the genes in the child to try and treat whatever disease the individual has. There are 2 types of gene therapy, somatic and germline, and in both ways there is no way to get away from the disease. In somatic gene therapy, the doctor injects the normal gene into the patient’s targeted cells to try and treat them, but the new gene does not get passed on to the offspring of the patient. In germline gene

This technology can target specific diseases on genes such as Alzheimer’s, Parkinson’s, polio, cystic fibrosis and the list can go on forever (Hackett, 2013). A popular method used to implement new genes is the adenovirus. A gene cannot simply be integrated into cells, it must have a vector, or a carrier which in this case is the adenovirus which helps integrate the gene into the DNA of the targeted cells (NLM, 2015). Another option is available where the subject’s cells are removed and injected with the gene and reinserted into the body (NLM, 2015). Both methods have shown success. Recently, in Oxford retinal disease has been overcome by using gene therapy (McClements, 2013). Several cases in this study allowed men and women to overcome a range of retinal diseases and avoid blindness and this is all due to the amazing effects of gene therapy (McClements, 2013). They used the adenovirus as a vector to deliver the new genes into the retinal cells in order to make the change in the eye. It was a hard fought success. There are several cases just like this study that have allowed people to renew their lives through human gene

Genetic information is claimed to be sui generis. Within those double helix strands of deoxyribonucleic acid, DNA, are mutations, some which affect the development of the organism. Recent research have led scientists to develop a new type of treatment for mutations. Gene therapy is the name applied to the treatment of inherited diseases by corrective genetic engineering of the dysfunctional gene. [1] This revolutionary advancement in medical technology, however, has provoked a portion of society into ruling this treatment as inhuman and unethical. [2] Despite the large criticism, this procedure alone is dangerous to attempt since the human immune system could reject the new DNA, which causes the patient to fall ill. [3] Gene therapy, a twenty-six

Gene therapy pro-actively transfers new genes into a patient therefore treating or preventing the disease. Gene therapy protocols follow two paths. Somatic gene therapy is a process in which hybrid retro vector is inserted into the cell. The vector binds to the cell membrane, packages itself in the vesicle which breaks down releasing it into the nucleus. Retroviruses are vectors harnessed for this task because their RNA genetic information can easily be transcribed into DNA. Furthermore, retroviruses introduce enzymes like integrase into the cell. Integrase incorporates new genes into the genome of a host. However, integrase can insert the modified genetic material in any arbitrary position in the genome of the host leading to insertional mutagenesis, and possibly cancer (Gene Therapy Retrovirus). Alternatively, cloned DNA solution is physically injected into a solution of zygote pronuclei during germline gene therapy In this way, the zygote produced has modified DNA which is passed to future generations. Nevertheless, germline therapy has a low rate of transgene integration in which modified genes do not get incorporated into the genome of the zygotes (Smith Kevin

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).

The word science comes from the Latin word "to know" (Science Made Simple, 2014). In