Almost everybody knows someone with a genetic disease. An aunt, a daughter, a friend, a coworker, every body is susceptible to have a genetic based disease. There are many types of genetic diseases ranging from skin malformations to lethal heart problems that could cause death. Genetic diseases affect newborns “Of the 3 to 6 percent of newborns with a recognized birth defect, at least half involve a predominantly genetic contribution.” (Fridovich, Fridovich-Kelly &Robinson, 2013, p.1); though there are other diseases such as cancer or aids that are obtained through an individual’s life and a big percentage of people are affected. That is why many scientists have searched for a solution to these problems that have affected so many people. Somatic cell gene therapy is the best cure to treat genetic diseases.
Somatic cell gene therapy, or replacement cell gene therapy, is a treatment that consists in modifying or restoring the function of a gene that is not working correctly or that is not working at all. It is a way to correct mutations of genes as well as inserting or deleting a gene within the DNA. Somatic cell gene therapy is the best cure because it cures genetic based diseases. There are not many solutions genetic based problems. For example, let’s say that someone has Severe Combined Immune Deficiency (ADA-SCID) People with this disease lack of protection against bacteria, viruses and fungi and are “prone to repeated and persistent infections that can be very serious or life-threatening” (N.A., ADA deficiencies, 2013). ADA-SCID is a very rare disease affecting about 1 in 200,000 to 1,000,000 newborns worldwide and because of this there are not many methods of treatment. The only known therapies for this genetic disease, so fa...
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...e new gene and having a successful process.
Lately there have been a great number of clinical trials to research how many of the diseases are treatable through Somatic cell gene therapy and how many other therapies can be perfected. Somatic cell gene therapy is still on an early stage. The recent advances have been made by nucleases discovery and design. Also, understanding how the techniques work and what factors, as temperature or altitude, affect during the cutting of the DNA makes advances on this field of science. As well as researching what kinds of enzyme work better for each type of genetic manipulation. After these research scientist would have mastered the Gene therapy treating this patients with genetic diseases will not only be easier but also cheaper and more available making somatic cell gene therapy the best cure to treat genetic diseases.
Somatic cell gene therapy is where genes can be put into specific cells and areas of the body which are affected by the disease. All cellular DNA in our body is essentially the same because it was replicated from the same zygote (fertilized egg). Cel ls differentiate into their respective tissues depending upon which part of the total genome is used.
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.
It was in the 1980’s that scientist began looking at alternative ways of treatments, one is gene therapy. Scientist would insert human genes into a bacteria cell. Then the bacteria cell would transcribe and translate the information into a protein. Once that is done the scientist would then introduce the protein into human cells. Gene therapy can be simply viewed as inserting bits of foreign DNA into a patient’s tissue in hope...
Human gene therapy is a method used in the medical field that treats diseases at a molecular level, by solving the source of the problem; our genes. Today, diseases and disorders are commonly treated by solving the symptoms, the surface of the problem. Many disorders and diseases are caused by defective proteins and within those defective proteins are damaged and defective genes. These defective genes can be treated through gene therapy. Gene therapy is not new and has been developed and improved by researchers for the past couple years. Being an experimental technique, gene therapy also has its pros and cons, but so far is showing positive and rising success rates.
USA Today 127.2644 (1999): 28. " In Vivo Somatic Gene Therapy." Brown University. Web. 18 Nov. 2011.
Gene therapy gives people who suffer from genetic diseases a chance to lead a normal life. Dangerous diseases, such as AIDS, SCID, Thalassemia and ADA can be cured successfully. In September 5, 2006, two people with advanced melanoma received Gene therapy and they got recovery soon. This is a breakthrough in cancer gene therapy. Gene therapy uses patients own cells to cure diseases, and, therefore, no rejection to their bodies. Furthermore, patients could get permanent cure from gene therapy without recurrence.
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
The types of diseases that can be treated by gene therapy are those where replacement of a single gene is able to cure the disorder. Some of the main diseases currently being researched in gene therapy studies include cancer, hemophilia, sickle cell anemia, muscular dystro...
"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 the therapeutic delivery of nucleic acid into a patient’s cells as a drug to treat diseases such as Severe Combined Immunodeficiency (SCID) and Adenosine Deaminase Deficiency (ADA) and possibly cancer as well. It was first researched in 1985
Since its inception, gene therapy has captured the attention of the public and ethics disciplines as a therapeutic application of human genetic engineering. The latter, in particular, has lead to concerns about germline modification and questions about the distinction between therapy and enhancement. The development of the gene therapy field and its progress to the clinic has not been without controversy. Although initially considered as a promising approach for treating the genetic of disease, the field has attracted disappointment for failing to fulfil its potential. With the resolution of many of the barriers that restricted the progress of gene therapy and increasing reports of clinical success, it is now generally recognised that earlier expectations may have been premature.
Human genetic engineering can provide humanity with the capability to construct “designer babies” as well as cure multiple hereditary diseases. This can be accomplished by changing a human’s genotype to produce a desired phenotype. The outcome could cure both birth defects and hereditary diseases such as cancer and AIDS. Human genetic engineering can also allow mankind to permanently remove a mutated gene through embryo screening, as well as allow parents to choose the desired traits for their children. Negative outcomes of this technology may include the transmission of harmful diseases and the production of genetic mutations.
Gene therapy enables patients to survive incurable diseases. In the field of genetic diseases, ADA-SCID, CGD and hemophilia are three main ones. ADA-SCID is known as the bubble boy disease. CGD is related to immune system that would lead to fungal infections which are fatal. Patients with Hemophilia are not able to induce bold bleeding (Gene therapy for diseases, 2011). Gene therapy also has good effects on cancer treatment and neurodegenerative diseases, which include Parkinson’s disease and Huntington’s disease. Viral infections, including influenza, HIV and hepatitis can also be treats by it (Gene therapy for diseases, 2011). According to the Science Daily in 2011, gene therapy now can apply to heart failures and neurologic diseases as well.
Germline therapy entails altering the genes in egg or sperm cells before they are fertilized. It necessitates the use of in-vitro fertilization when dealing with embryos (“Human Gene Transfer Research,” 2011) and any genetic variations will be passed to future offspring (“Germ Line Therapy,” 2011). Currently, germline therapy is still in its infancy and has only been tested on animals (Darnovsky, 2013). Although this type of therapy decreases the risk of defective genes and thus, has the possibility of preventing congenital diseases, it has elicited substantial ethical controversies.
Position Paper: Gene Therapy in Humans. Advancements in science and medicine are usually accompanied by a myriad of ethical and moral implications. The fairly recent advancement in genetics, called gene therapy, is no exception to the baggage of polarizing views that come with new technology. Gene therapy is an extremely hot topic in both the scientific world and everyday life. New technology, discoveries, and breakthroughs are rapidly occurring in the field every day.