Gene Therapy Concept

The general concept behind gene therapy is explained below. Almost all human diseases are rooted in genetics, from cancer to autoimmune diseases and even hair loss. Already in the 1980s, researchers were able to prove by animal testing that disease-bearing genes can be corrected or replaced with therapeutic genes. This process is called gene therapy. So gene therapy treats the “real” causes of disease (the diseased cells) and not just the symptoms. Up to now, more than 4000 people have been treated with gene therapy in the context of clinical studies, mostly for AIDS or cancer.

The method is always similar or identical: The healing gene is brought to the desired cells using a “gene taxi” (scientifically, a gene vector), for instance to a tumour cells.

The greatest problem in gene therapy is the transport, i.e. how to route the healing gene to the desired cells most efficiently. Currently, viruses modified to be non-dangerous (e.g. the common cold) or liposomes are the most common ways to achieve this. Thousands of scientists work ceaselessly to improve these means of transport. Some, for instance, try to merge viruses and liposomes or develop new “gene taxis” altogether.

Gene therapy is starting to fulfil its promises to mankind. It is safe to assume that the treatment of various other diseases can be accomplished with little or no side effects using gene therapy since it is highly specific and only affects the genes that cause the disease targeted. At the moment, side effects of medication are among the most frequent causes of nosocomial deaths – in the treatment of lung infections and diabetes they are placed on Slots 4 and 6 of the statistics on the cause of death (refer to the Bates’ Study). This could change drastically with the approval of highly specific, gene-based medication. As therapeutic genes cannot be transmitted via semen or ova, it is impossible to pass the genes on to one’s offspring.

The concept of gene therapy has the potential to revolutionize medicine entirely. There are virtually no diseases that cannot be treated using gene therapy.

Some examples:

1. Cancer
   Multiple clinical studies are currently testing the p53-gene which is able to kill cancer cells. Its only side effects are a slight increase in body temperature, a feeling of warmth and a slight tingling in the fingers, but it is basically free of side effects. Gene-therapeutic vaccination against prostate cancer is now also possible.
2. AIDS
   There are different approaches for the use of gene therapy against AIDS. Particularly elegant is the concept of DNA-Vaccines, a special form of gene therapy. These DNA vaccines are more efficient and have fewer side effects than regular vaccines and are even able to protect from incurable diseases. Vaxgen, a biotechnical company, has already begun Phase III of the clinical testing process of an anti-AIDS vaccine. DNA vaccines against cancer and malaria are also being developed.
3. Chronic Pain
   Using animal testing, chronic pain can be cured successfully while the ability to feel pain remains intact.
4. Erectile Dysfunction
   US researchers are currently working on a gene-therapeutic treatment of erectile dysfunction. In 1999, the process was successfully tested in rats. Based on the results, it is safe to assume that treatment is forthcoming in half-year intervals.
5. Alzheimer
   The University of California is currently in Phase I of testing a very promising gene therapy.
6. Hearing problems
   It could be possible to grow hair cells, thus making it possible to protect people from going deaf.
7. Parkinson’s Disease
   Here, too, promising therapies are being developed. Three monkeys were cured almost entirely in animal testing. Scientists hope to be able to apply the therapy to humans within the next three to five years.