William French Anderson, Michael Blaise, and Ken Culver performed the first successful gene therapy on a human in 1990. They developed a protocol for treating Adenosine deaminase (ADA) deficiency, a severe combined immune deficiency, also known as the "Boy in the Bubble disease." ADA deficiency is a result of inheriting two copies of the defective ADA gene. Possession of a normal gene leads to the continuous, regular production of ADA in cells throughout the body. Without at least one properly functioning gene, children have no way of converting

Previous treatment options included bone marrow transplants, which worked well with matched donors. A major breakthrough occurred with the development of polyethylene glycol coated ADA (PEG-ADA). This treatment introduces coated ADA into the blood stream, although not into the cells. It requires expensive, painful shots on a weekly basis, but it succeeded in giving children with ADA deficiency a new lease on life. While their immune systems were far from normal, PEG-ADA allows some semblance of a normal life and a much-increased life span.

With the ever-increasing knowledge on gene therapy, many ethical and moral concerns have risen. Society as a whole shows both concern and apprehension on this contemporary issue.

Many argue that the morality of gene therapy is misguided, mainly because of the effort private companies and the NIH are doing to have certain DNA sequences patented. With the research going into decoding the human genome, there is an unusual demand for the patenting of useless DNA sequences, undermining the sole purpose of patents. Should DNA sequences should be patented at all or be treated as merchandise? Patenting any kind of DNA sequences is just like patenting life itself, and from history we know that the ownership of any human being is wrong. DNA or deoxyribonucleic acid sequences in an individual can thus be considered analogous to a piece of property.

Retroviruses have a limitation because they are unable to infect non-dividing cells. This drawback was circumvented by the so-called ex-vivo gene therapy. In this procedure, cells are removed from the target tissue and grown in vitro. During this period the virus is added and the cells become infected. Subsequently, the cells are transplanted back to the target tissue.

The next step to gene therapy is to find a vehicle to transport the new genes. This is done through the use of vectors. A vector is a DNA molecule into which a DNA fragment can be cloned and which can replicate in a suitable host organism. The majority of vectors used today are attenuated or modified viruses. "The modified viruses can not replicate in the patient but do retain the ability to efficiently deliver genetic material."9 There are two main modes used to deliver the genes of interest to the patient: non-viral and viral delivery vectors have been used. Both of them have advantages, but also a not-so-short list of disadvantages.

There is enormous money making potential in the future of gene therapy. It is estimated that by the year 2000 there will be at least 10 cancer gene therapies and that the gene therapy market will top $2 billion. "Over the past five years, estimated Michael Murphy, editor of the California Technology Stock Letter, companies have spent about $250 million on gene therapy research and development. When a product gets approved, he says the payoff in the annual sales will probably be ten times that."6

Some common words found in the essay are:
Genome Project, Patenting DNA, NGVL Gene, United Biologically, ARIAD Pharmaceuticals, Cornell University, Genesys Inc, Stock Letter, Ashanti DeSilva, Boy Bubble, gene therapy, parkinson's disease, human genome, cystic fibrosis, prostate cancer, dna sequences, gene vector, national gene, national gene vector, gene transfer, safety efficacy trials, safety efficacy, clinical safety efficacy, human genome project, efficacy trials united,
Approximate Word count = 3675
Approximate Pages = 15 (250 words per page double spaced)