Gene Therapy: Facts, Pros and Cons, Basic Information, Objectives, Genetic Mapping, Treatments, Patents

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The objective of Gene Therapy is to identify the defective genes responsible for disease and utilizing this knowledge for treatment, cure, and prevention of disease. The intent is not to change physical characteristics but to help the physically impaired to overcome their problem.


Many genes remain undiscovered. These undiscovered genes might be the cure for many of today's incurable diseases. Uncovering the mystery of genetics and using the knowledge for the correction of disease and/or defective genetic material is just a part of gene therapy.

Gene Sequencing

After discovery of a new gene, the next step is sequencing. The process involves documenting the exact chemical sequence of the gene. As mentioned in the previous section, the chemical disposition determines the gene's function. After the sequencing is found, the gene is then mapped. This process is similar to putting the pieces of a jig saw puzzle together. Information of this type is extremely valuable to pharmaceutical companies looking for new miracle drugs. Chemical sequence will tell exactly what drugs are effective or ineffective for treatment of diseases. Therefore, pharmaceutical companies can customize drugs to fit the patient. Adverse drug reactions will be history.

How does Gene Therapy Work?

After identifying the responsible genes, treatment of the disease begins:

Gene Therapy for Treating disease is accomplished by replacing defective with healthy genetic material. This corrects the problem at its source. One of the major obstacles in this process has been the method of gene delivery. A common method for gene delivery is the use of an altered virus carrying the gene. The assumption is that the virus can break through the body's immune system allowing the genetic material to pass as well. The virus is altered to prevent it from reproducing and invading the body, as it normally would do. However, the body's immune system still recognizes the virus as a virus. Rejection takes place-causing inflammation, which in turn damages other organs in the process. In September of 1999, an eighteen-year-old man died when he participated in research for gene therapy. His liver was injected with an altered virus carrying corrective genetic material. Four days after treatment began; inflammation developed and the man's vital organs cease to function. This death has caused many to reconsider the methods used for gene delivery. A new approach being considered is the use of the outer shells of viruses has come into play. The shells can carry the gene into the body without triggering the immune system.

Computer Systems in mapping Genetics Genes

Computer systems are being built to aid in the genetic mapping. Venter is building a huge computer system capable of storing 20 trillion bytes to work on detailing gene maps. IBM is working on a computer system that will have over a million processors and capable of doing a billion tasks per second. The IBM computer is called BlueGene. It is intended to detail the protein folds of genes. This knowledge is necessary for determining the role of a gene. The computer will take this knowledge and use it to analyze tissue samples, identify illnesses and prescribe treatments best suited to an individual's genetic material. Furthermore, these computers will dramatically speed up sequencing process. Presently, the machine used for mapping is made by the PE Corporation. This machine arranges the genes in the proper sequence.

Celera Company has mapped 90% of the genes mapped. Ironically, this company added the government's genes data base to their own database, but will not allow the government researchers access to Celera's data. This has given Celera the lead in the race. Celera sells the genetic information to drug companies.

President Clinton and the British prime minister have said that genetic information is available to everybody and therefore is not patentable.

Genetics Genes Patents

Government researchers, other researchers worldwide and private companies are in the process of mapping all the genes that make up the human body. As mentioned earlier, the genetic map is the blueprint for the creation of all organisms. Government researchers are racing to complete this task before private companies finish. Among the companies involved in the race are Venter, IBM, Celera, Geron Corporation, Novartis, and Glaxo. As these companies discover the genes for various ailments and human tissue, patents are granted. Patents impose many restrictions on the use of the genetic maps. Unquestionably, patents make gene therapy very expensive. To illustrate the potential monetary gains of gene therapy, Bill Gates is a major investor in Darwin Molecular Corporation. This company owns the patent for a premature aging gene. Bill Gates has said, the fusion of information and biological technology will bring about a change in the human condition that will make anything we have done to date seem infinitesimal by comparison. (15:14) The U.S. patent office has granted for genetic maps of sheep, fish, mice, rabbits, pigs, cows. There is a patent for human ears, which is grown on the backs of mice. Other patents granted include the genes for Alzheimer's, Obesity, Arthritis, Melanoma, Blindness, Breast Cancer, and Ovarian Cancer.

First successful use of Gene Therapy

The first successful use of gene therapy was correcting heart damage. This was accomplished by injecting the heart with a corrective gene. The gene excreted an enzyme initiating a response from the damaged cell. After which, the excreted enzyme produced a specific response from the chemical composition of the cells DNA. All defective cells can be corrected in this manner.

written May 2000