Introduction The usage of genes as medicine give rise to the concept of 'Gene Therapy'. In the process of Gene therapy, a defective gene copy is repaired by transmitting therapeutic gene copy into those cells of an individual. So thereby, a faulty gene could be replaced or a medical condition may be corrected by introducing a new gene. Gene therapy has a promising future as it is powerful enough to treat many genetic conditions and also other deathly disease like AIDS, cancer etc. Though the potential of "gene therapy" is realized and recognized, it remains still on an experimental level and needs to worked on before accepting as the best solution. Gene therapy was put into practical use for treating immune deficiency disease called "adenosine deaminase" ADA deficiency in 1990s. This is when the gene transmittal of "corrective genes" into stem cells happened for the first time in practice. Method The task of making gene therapy to work yielding positive results is substantial. In order to put it to practical use, firstly we must understand well the defect in question and locate the faulty genes in the system. In addition to that, the identified defectives genes should be accessible, the appropriate healthy gene set or curative gene set should be made available and lastly a way to transmit this gene to that defective area must be clearly constructed. The final part is the most challenging procedure in the process of gene therapy. "Gene Delivery" denotes how to make the curative gene reach the defective area. Few of these vectors would assist us in the problem of 'gene delivery'. a) Harmless viruses A favorable method for "gene transfer" would be using harmless viruses as carriers of genes into cells. This method is not... ... middle of paper ... ...s, designed personalities, master races, or Frankenstein monsters will be created can be given no credence in the light of what is presently known. The best insurance against possible abuse is well-informed public. With proper safeguards offered by the scientific society, this powerful therapeutic procedure would provide great benefits reducing the sufferings and death caused by genetic disorders Works Cited [1] Aiuti A, Cattaneo F, Galimberti S et al. (2009) Gene therapy for immunodeficiency due to adenosine deaminase deficiency. New England [2] Journal of Medicine 360:447-458. [3] The Gene Therapy Research Unit, The Children’s Hospital, Westmead, Australia [online]. [4] Available from: http://www.chw.edu.au/prof/services/genetherapy/ [Accessed Feb 2014]. [5] http://www.extremetech.com/ [Accessed Mar 2014]. [6] http://ghr.nlm.nih.gov/handbook/therapy/ethics
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.
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.
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.
In September 14, 1990, an operation, which is called gene therapy, was performed successfully at the National Institutes of Health in the United States. The operation was only a temporary success because many problems have emerged since then. Gene therapy is a remedy that introduces genes to target cells and replaces defective genes in order to cure the diseases which cannot be cured by traditional medicines. Although gene therapy gives someone who is born with a genetic disease or who suffers cancer a permanent chance of being cured, it is high-risk and sometimes unethical because the failure rate is extremely high and issues like how “good” and “bad” uses of gene therapy can be distinguished still haven’t been answered satisfactorily.
So Gene Therapy, being a process solely aiming to heal genetic issues in human beings, is a technology which is desirable for our modern society. If a method can be produced, which cause...
Rubanyi, G. (2001). The Future of Human Gene Therapy. Molecular Aspects of Medicine. 22,3, pp. 113-142.
Every year, the rate of mortality increasing because most diseases may lead to death if not treated early. One of the methods that can be used to cure some diseases is by using the treatment known as gene therapy. Based on Pruitt’s (2008) study, numbers of inherited and acquired diseases were reduced since gene therapy has the ability to provide new treatments to cure them. According to Shi and Zou (2008), gene therapy is defined as expression of protein or interrupts the synthesis of protein in cell by transferring the genetic material into a host in order to treat or prevent a disease. Besides that, Kelly (2007) stated that an “abnormal” hereditary disease-causing gene in an individual’s cells and tissues is treated and used gene therapy by to replace them with a “normal” gene. Around 1970’s, idea to use “genes” as “drugs” for human therapy was originally from United States (Giacca, 2010). Moreover, there are some objectives in using the gene therapy as a treatment. First, gene therapy is used to cure or slow the progression of disease by introducing the genetic material into target cells and next objective is to aim at the direct correction of endogenous genetic defects by delivered some additional copies of a gene (Pruitt, 2008; Giacca, 2010). Furthermore, Yadav and Tyagi (2008) found that there are two types of gene therapy which are germline gene therapy and somatic cell therapy. As stated by Shi and Zou (2008), therapy that involved modification of any cells in a patient’s body is called as somatic cell gene therapy while germ line gene therapy is therapy that involved modifying of human eggs or sperms that pass genes on to future generations. Other than that, animal tissue culture is used to test the effective...
Gene therapy is a method used by doctors that ‘uses genes as medicine’. It repairs faulty or damaged gene copies by transferring a therapeutic or working gene into specific parts of faulty gene copy. This means that gene therapy can be used to replace a faulty gene or to introduce a new gene whose function is to modify the clinical course of a condition or to cure a problem in the gene (Refer to image 2). Image 1 shows how gene therapy is completed using an adenovirus vector while image 2 explains the method of gene therapy and shows how cells are removed, altered and injected back into the patient. Gene therapy has potential to treat and cure many medical conditions such as various types of cancer, degenerative diseases and viral infections such as AIDS. Research into gene therapy still needs to be done before this approach to the treatment of certain conditions will realise its full potential. Trials are being conducted in the United States and Europe and a modest number initiated in other countries, including Australia. The majority of these trials are focussing on treating acquired conditions such as cancer (European Society of Gene & Cell therapy, 2011).
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.
Gene therapy has become an exciting and controversial issue on the scientific and medical horizon. Science offers new technologies that, in the future, will be able to treat and cure common genetically passed diseases. However, as it is an extremely broad subject, some time must be dedicated to its interpretation and explanation. First, a general definition of gene therapy is required. Genethics, the Clash between the New Genetics and Human Values, by David T. Suzuki and Peter Knudtson, defines gene therapy as "the medical replacement or repair of defective or faulty genes in living human cells." It is not really so elementary as the definition would imply. Within gene therapy there lie certain aspects, some more controversial than others, some more achievable and probable than others. The ethical question must be addressed at each turn. However, all of this will be discussed at greater length subsequently.
Gene therapies necessity, stems from the fact that it can cure, life threatening gene based diseases, with minimal after effects. Although there are some ethical and physical issues that can occur through this technology, when comparing it to the other genetic technologies, it’s quite reliable. Although it is in its infancy, the technology has still been present for several years, with the first case of gene therapy being done in 1990. Sure, there have been some complications, but its still a growing technology with a strong future in front of
Genes are what keep your DNA, which is a genetic code that controls how you look and the way your body works (“What is Gene Therapy”). Each person has two copies of each gene, one passed down from mum and the other from dad (“What Is a Gene?”). However sometimes certain genes may become mutated or there are too many or too little of that gene and that may cause disorders; ADA-SCID is an example of such a disorder. ADA, adenosine deaminase deficiency, is one of the forms of SCID, severe combined immunodeficiency, which weakens the patient’s immune system. ADA-SCID has a few forms of treatment but one of the most promising ones is gene therapy. Gene therapy is a process that can manipulate one’s genes to cure a disease. Gene therapy has both
Genes are made of DNA – the code of life (Gene Therapy- The Great Debate!). The changes in genes may cause serious problems, which we called genetic disorder. In theory, the only method to cure genetic disorders is gene therapy, which basically means the replacement of genes in order to correct the loss or change in people’s DNA. Although gene therapy gives patients with genetic disorders a permanent cure, it is controversial because it has safety and efficacy problems, and raises ethical issues.
The opportunity to decide which genes will be passed on to future generations certainly would be tempting for many people. A method by which we could prevent genetic diseases and customize our offspring’s gender, physical traits and the intelligence is not as impossible as it may seem at first. The debate over the designer children revived recently with the discovery of the new technique allowing scientists to alter DNA.