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Process of gene transfer
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Mechanism of Transfer in Gene Therapy
Abstract:
Gene therapy is the transfer of “normal” genes into the body to replace defective or undesired genes. The transfer may be in somatic or germline cells and may take place in vivo or in vitro. The DNA may be inserted in a retrovirus, adenovirus, adeno-associated virus, herpes simplex virus, or liposome, or it may be naked DNA. The vector travels to a target cell and inserts the gene, which goes to the host cell’s nucleus and may integrate into the genome. In cells with dominant negative genes, resulting protein must be prevented from translation or transcription using oligonucleotides or antisense technology. Though many obstacles delay the development of gene therapy, this new field will surely improve the future of medicine.
Mechanism of Transfer in Gene Therapy
Gene therapy is a method of replacing defective or undesired genes in the body with “normal” genes. A vector is re-engineered to deliver the gene to a target cell. Then the gene is transferred to the cell’s nucleus and must be activated in order to function. The gene must be integrated into the cell’s genome in order for it to continue to function and be replicated. Side effects are possible because the vector may be detected as a foreign substance by the body’s immune system. Gene therapy may take place either in vivo or in vitro, the transfer may be in either somatic or germline cells, and a variety of viral and non-viral vectors may be used.
In vivo gene therapy is done inside a living organism. This is accomplished by inhalation, oral administration, intramuscular injection, or intravenous administration (Brooks, 24). Currently, most in vivo experiments have taken place in animals other than humans. Most gen...
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...ocated, ensure that gene therapy will be a revolutionary aspect of medicine’s future.
Bibliography
Brooks, Gavin. Gene Therapy: The use of DNA as a drug. London: Pharmaceutical Press, 2002.
“Gene Therapy.” Human Genome Project Information. 18 November 2005. U.S. Department of Energy and the National Institutes of Health. 28 July 2007.
“Gene Therapy: Molecular Bandage?” Learn.Genetics. 2007. The University of Utah, Genetic Science Learning Center. 28 July 2007.
Januario E. Castro, Thomas J. Kipps. “Chapter 26. Principles of Gene Transfer for Therapy.” Williams Hematology, 7th Edition. United States of America: The McGraw-Hill Companies, Inc., 2006. 28 July, 2007.
Gene therapy is the application of the technique where the defect-causing "bad" genes are replaced by correct "good" genes. The idea of gene therapy is to treat the disease by correcting the "bad" DNA (Deoxyribonucleic acid) rather than the current me thod of providing drugs, or proteins not produced by the defective gene. Gene therapy addresses the problem first hand by directly working with the genetic information causing the disease. From the book Shaping Genes, Dr. Darryl Macer says "It is like f ixing a hole in the bucket, rather than trying to mop up the leaking water." There are two kinds of gene therapy, somatic cell gene therapy and germline gene therapy.
There have been four somewhat recent successful gene therapy treatments. The four deal with correcting hemophilia, bone marrow transplants, skin cancer, and vessel growth. In the success with the bone marrow transplants, French researchers collected bone marrow cells from patients, used gene therapy to correct the bone marrow, and then returned the bone marrow to the patient. This was 80% successful as reports 16 months after the transplants showed. Squamous cell carcinoma, skin cancer of the head and neck, was treated using gene therapy as well. The fourth trial was where DNA was used to carry a substance that stimulates blood vessel growth to damaged heart tissue and in this trial there was much success noted.
Gene therapy is an experimental technique that allows doctors to insert a gene into a patient’s cell rather than using drugs or surgery. Gene therapy is a process of which defective or undesired genes in the body with “normal” genes. A vector is re-engineered to deliver the gene to a target cell. Then the gene is transferred to the cell’s nucleus and must be activated in order to function. The main focus of gene therapy is to replace a lost or improper gene with a new functional copy into a vector that is inserted into the subject’s genome by way of penetrating its DNA. Gene therapy can be done outside of the body known as ex vivo by way of taking cells form patients bone marrow or blood and then growing them in a laboratory. Thus the corrected copy of the gene is inserted into the cells before being put back into the body. Gene therapy can also be done in vivo which can be done directly to the patient’s body. The word gene therapy really defines the management of genetic information that is encased in the cells, however, in most recent procedures the available technology is closely related in adding new genetic information, and many researchers favor the term gene transfer rather than gene therapy to mirror the reason that the purpose of gene work cannot always be therapeutic.
Gene therapy works by introducing new and functioning genetic material to damaged genes to help it function and to produce beneficial proteins. If a gene is inserted directly into a cell, it usually will not function. So to complete this task, a vector, a modified virus is used to carry and deliver the new gene. There are two different categories of vectors than can be utilized in this process; recomb...
A molecular biologist by the name of James Watson once said, “we used to think that the fate was in our stars, but now we know that, in large measure, our fate is in our genes.” The Oxford Dictionary defines gene therapy as the transplantation of normal genes into cells in place of missing or defective ones in order to correct genetic disorders. Gene therapy has the ability to prevent, treat, and even cure diseases by replacing a faulty gene with a stable, healthy one (American Medical Association). Aldous Huxley’s, Brave New World, relates to gene therapy because they program each embryo with how they should live. This essay will first talk about why gene therapy is done and how it works.
Over 40 years ago, two men by the names of James Watson and Francis Crick discovered deoxyribonucleic acid, or DNA. DNA is hereditary material in humans and almost all other organisms (What is DNA?). From this finding, gene therapy evolved. Today, researchers are able to isolate certain specific genes, repair them, and use them to help cure diseases such as cystic fibrosis and hemophilia. However, as great as this sounds, there are numerous ethical and scientific issues that will arise because of religion and safety.
Gene therapy is a technique which has developed in the wake of recombinant DNA (rDNA) technology. It is a process which results in the correction of a genetic disorder by the addition of a piece or fragment of DNA into the genetic material of a living, functioning cell. A mere thirty years ago this concept belonged to the realm of the human imagination made manifest in the works of science fiction. Today it belongs to the realm of the human imagination made manifest in the works of science, period. It is mind boggling to try to comprehend the far reaching effects of gene therapy. How is it affecting society? Who will benefit from its use? Should it be used at all? Should research continue? How do we answer all of these questions? The answers are not readily available, nor are they black and white, but an attempt at finding some solutions must be made. Before exploring this line of thought further, a basic understanding of the technical aspects of gene therapy is essential.
Despite being magical of gene therapy, it is high-risk. Few people got benefits from it, and it has a low rate of success. Prior to the human trial, Batshaw and Wilson had done experiment on animals to ensure the safety. Over 20 experiments have been done on mice but only 12 of them survived at last (Sophia, M. and Kolehmainen, J.D., 2000). More seriously, complicating diseases, which can be more dangerous than genetic diseases, might set in during the treatment period. In December 200...
Genetic engineering is now being used to create new medicines and therapies for many disorders and diseases, and also to improve agricultural plants and animals to produce bigger yields or enhanced nutrient composition and food quality. In Gene therapy, copies of healthy human genes produced in bacteria can be inserted into human cells with defective or missing genes, to fix the problem. Gene therapy is promising because it can use to treat genetic
Lopez, Gerald Gabriel. "Gene Therapy: the Scientific vs. the Societal" The Resource. Jan. 1998. 10 Apr. 2001. .
"The aim is to decrease the fear of a brave new world and to encourage people to be more proactive about their health. It [Gene therapy] will help humans become better physically and even mentally and extend human life. It is the future” (Hulbert). Dr. Hulbert, a genetic engineer, couldn’t be anymore right; more time, money, and research needs to be put into gene therapy and genetic engineering, since it can cure certain illness and diseases that are incurable with modern medicine, has fewer side-effects than conventional drugs or surgery, and allows humans to be stronger physically and mentally at birth. Gene therapy or genetic engineering is the development and application of scientific methods, procedures, and technologies that permit direct manipulation of genetic material in order to alter the hereditary traits of a cell, organism, or population (NIH). It essentially means that we can change DNA to make an organism better. Genetic engineering is used with animals and plants every day; for example with genetic...
Gene therapy is a provisional technique that is the insertion of normal genes into the cells where there is a missing or miscoded gene to fix a genetic disorder. In the 1960s and early 1970s,
...matopoietic compartment using integrating vectors particularly need to understand genotoxicity risks in relation to the risks of conventional bone marrow transplantation. A QPL could direct them to ask questions about risk, benefits and survival rates following transplantation at local centres; the prognosis of patients in the different haematopoietic gene therapy trials; the number and status of patients that developed leukaemia in the SCID-X1 gene therapy trials; and whether there are any differences between the proposed vector and the vector used in the SCID-X1 trial and any possible safety developments. This kind of guidance may help patients understand both what is known and unknown about specific applications of gene therapy.
Safdar, M. (2010) Gene Therapy: Advantages and Disadvantages [Online] Available at: http://www.biotecharticles.com/Genetics-Article/Gene-Therapy-Advantages-and-Disadvantages-271.html [Accessed July 17 2011]
In the end, gene therapy in humans needs to come a long way before it will be widely accepted, but there is great potential in the technology and it needs to be pursued. Bibliography Anderson, W. F. (1992). The Species of the World. Human Gene Therapy -. Science, 256 (5058), 808-813.