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Until recently, no real thought was given to a treating a genetic disease except for traditional methods of reducing the symptoms of the diseases. With recent developments in gene therapy, the possibility of “correcting” mutated or altered genes has come into being.
For example, hemophilia is a disease causes by a defect in the gene that code for clotting factor. Hemophilia is a disease that causes people to clot blood incorrectly and thus bleed for a possibly dangerous period of time.
The way hemophilia used to be treated was by a complete blood transfusion, which had many side effects, including HIV infection. Now physicians are able to give the clotting factors individually without giving whole blood and without HIV.
The main objective of gene therapy is to take cells that have either an “incorrect” gene or a mutated gene, and correct the gene to what is believed to be the normal form of the gene. It is also believed that, even in a partial sense, a human can be created that has the corrected gene.
As far as genetic diseases are concerned today, there is no traditional method available that can provide an effective cure for such a disease. Creating transgenic animals and mice, as you have seen, is becoming pretty commonplace today. The question comes when we are trying to introduce new genes, or at least copies of normal genes, into humans. This is theoretically possible, but many technical and ethical boundaries do exist.
The main problem is the fact that when transgenic animals are created, many individual animals receive the foreign DNA and only the few that survive and display the appropriate qualities are kept.
In other words, it’s a game of numbers, and it becomes very difficult when you’re dealing with an animal that only produces on, maybe two (but there ARE exceptions) offspring at a time. So the bottom line here is that we need to have more effective methods of gene delivery into humans, or else the chance of gene therapy becoming a success in humans becomes very low.
Also, there is a fundamental difference between gene therapy for recessive diseases and gene therapy for dominant diseases. Recessive diseases are caused by the lack of production of a specific product, so gene therapy must provide a specific function.
Dominant diseases are caused by an altered function, for which gene therapy must wither return the defective gene to the normal state or inactivate it, along with providing the normal gene.
Current technology is far from being able to replace a particular gene, or even to alter the expression of a gene, so we will discuss the treatment of recessive disorders. The possibilities of gene therapy approaches to curing disease are exciting, but are loaded with risks and challenges. However, the prospect of widespread gene therapy brings about many risks, social issues, and ethical issues.