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Biotechnology with gene technology is a key factor in medical progress. Because medicine is a special domain of life science, research progress in the two domains is often undistinguishable, and of profit to both.

Many diseases are caused because a specific protein from the human body is lacking or not functioning well. Administering this protein as a drug can allow complete or partial recovery. The protein can be isolated from human cells or tissue. This procedure can have severe consequences: preparations of proteins from human blood for example, are not always safe. In the beginning of the '80's many haemophilia patients lacking a blood cloth factor have been infected with HIV. The blood factors administered to these patients, contained remains of the HIV-virus from seropositive blood donors. Currently, the human blood cloth factor is produced in the lab, by means of gene technology.

Today, many drugs are produced in a lab, using biotech tools. Bacteria, yeast and animal cells produce human proteins that are available as drugs. Transgenic plants producing e.g. edible vaccines are under development. The first biotech drug insulin was commercially available in 1982. Since 1995, 150 new biotech drugs have been developed, including treatments for serious maladies like HIV infection, Alzheimer's disease, psoriasis, asthma and multiple myeloma. Many of them are targeted to previously untreated diseases.

Technical progress in life sciences has led to:

The many applications of genomics:

Link to listing of: biotech drugs approved in 2003
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More knowledge on the genome, can lead to the cause of a disease - malfunctioning of a protein and hence a mutant gene - more easily. In addition, knowing this gene will allow screening for the disease. Genetic diagnosis will allow assessing the individual genetic predisposition. The understanding of how genes influence whether and how a medication works and whether it causes side effects could lead to personalized drug treatment. To achieve this, genetic tests would have to become a part of standard diagnosis.
Knowing more about the molecular causes of a disease not only accelerates progress of research towards diagnosis, but also towards prevention and therapy of the disease. Since diseases have very rarely a single cause, the way is not straightforward. Nevertheless, the efficacy of the new treatments of diseases such as cancer, Alzheimer or cystic fibrosis is improving steadily.
Knowledge about bacterial and viral genomes helps to identify more easily the mechanisms of infections and thus improve their prevention and treatment. The benefits of this technique joined to efficient international collaborations were illustrated during the outbreak of SARS in 2003; the genome was deciphered within a week after the pathogenic agent had been identified. Already a few months later, promising vaccines are under development.

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Stem cells are very special body cells. Serving as a sort of repair system, they have the potential to develop into many different cell types in the body. Theoretically they can divide without limits to renew themselves, or divide and differentiate into other, specialised cells such as a muscle cell, a red blood cell or a brain cell. Stem cells have the ability to form different types of tissues and organs, and they are present in every adult as well as in embryos.
Embryonic stem cells, as their name suggests, are present in embryos. They can develop in (almost) all body cells (see "Read more"). For research purposes, embryonic stem cells are derived from embryos that develop from eggs that have been fertilized in vitro - in an in vitro fertilization clinic - and then donated for research purposes with informed consent of the donors. However, some countries have strong legal restrictions on research with human embryonic stem cells.
An adult stem cell is an undifferentiated cell found among differentiated cells in a tissue or organ, can renew itself, and can differentiate to yield the major specialized cell types of the tissue or organ. The primary roles of adult stem cells in a living organism are to maintain and repair the tissue in which they are found. Some scientists now use the term somatic stem cell instead of adult stem cell.
Stem cells are very interesting for medical situations, in which tissues have to be regenerated (e.g. burns, multiple sclerosis, Parkinson's disease).
In addition, the specific factors and conditions that allow stem cells to remain unspecialized are of great interest to scientists. It has taken scientists many years of trial and error to learn to grow stem cells in the laboratory without them spontaneously differentiating into specific cell types. For example, it took 20 years to learn how to grow human embryonic stem cells in the laboratory following the development of conditions for growing mouse stem cells. Therefore, an important area of research is understanding the signals in a mature organism that cause a stem cell population to proliferate and remain unspecialized until the cells are needed for repair of a specific tissue. Such information is critical for scientists to be able to grow large numbers of unspecialized stem cells in the laboratory for further experimentation.

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Genetic diagnosis is a test performed to identify the specific genetic features of a person. Just a small amount of his/her biological material (e.g. blood, hair, sperm,…) is necessary for this test.
In medicine, it is used:

Presently, the diagnostic possibilities are usually more advanced than available appropriate therapeutic solutions. Genetic diagnosis could in future help to design strategies of disease prevention or tailor-made therapy treatments and therefore contribute to avoid non-adapted or overdosed medical treatments.
The DNA fingerprinting used for crime investigations or paternity tests probe regions of DNA which are unrelated to known functional genes.

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The aim of a cell therapy is to repair diseased or injured body parts with healthy new cells. These could be (derived from) stem cells that are able to differentiate into the targeted cell type and tissue. Bone marrow transplantation is a kind of stem cell therapy, which is already widely applied to treat leukaemia and other blood disorders. Bone marrow stem cells from a donor are injected into the patient's blood and form healthy blood cells. Rejection of the foreign donor cells by the immune system still poses a problem.
To avoid rejection, scientists want to use the patient's own stem cells to regenerate tissues. This is still in an experimental phase, but is seen as promising with great potential. The hope is to treat a wide range of human disorders, such as neurological diseases (e.g. Parkinson's disease), spinal cord injuries, skin burns, diabetes or cancer.

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Today the word 'cloning' is used to describe several things. But in essence, the technique is used to produce identical organisms. Clones are genetically identical descendants derived asexually from a single individual. Plants are cloned for many years; we sometimes do it ourselves in our garden. Or it occurs spontaneous: shoots or sprouts can develop in genetically identical plants. Also microorganisms can multiply by cloning.

Nowadays, the definition of cloning is extended to the process used to obtain identical copies of biological material such as DNA molecules, cells, organs or tissues (molecular cloning).

Cloning became widely known with the production of the first cloned mammal, the sheep Dolly, which was obtained by transferring the nucleus of a somatic cell into an enucleated egg. The modified egg was then implanted into the uterus to grow. The procedure when used to create duplicates of an existing animal is also called reproductive cloning.

Therapeutic cloning is somewhat different. It uses the technology of nucleus transfer to regenerate organs or tissues for medicinal purposes. Embryonic stem cells can be triggered to develop into specific body cells, ready for transplantation. Unfortunately these cells are often rejected by the patients' immune system. If stem cells are prepared from an embryo which results from the transfer of the nucleus from a patient's body cell in an enucleated oocyte, the stem cells contain the genetic material of the patient. The cells or tissues made out from them can be transplanted in the same patient without risk of rejection. At this moment the organ or tissue will exhibit the patients' immunology markers.

Molecular cloning is used to produce large quantities of a specific DNA sequence. The process includes the isolation of the DNA sequence of interest, the production of multiple copies either enzymatically or in an organism, for example a bacterium that is capable of growing over extended periods. The ability to generate a nearly endless number of copies (clones) of a particular sequence is the basis of recombinant DNA technology.
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Reproductive cloning is the generation of individuals with identical genetic information. Two types of reproductive cloning are known. One technique generates clones from embryos while the other requires transfer of the cell nucleus from an adult cell. The generation of clones from embryo cells has been used successfully to clone laboratory mice, goat, sheep, cattle and other mammals.
The most well-known work on cloning by nuclear transfer was the production of the sheep Dolly in 1997. In this experiment, the nucleus from an egg cell (oocyte) was removed and replaced by the nucleus of a somatic cell from an adult ewe (female sheep). Thus, the egg cell now contained the nuclear genetic material (DNA) of the adult cell. The resulting 'fertilized' oocyte developed into an embryo which was implanted in the uterus of another adult ewe. It divided like a normal embryo and the lamb which was born from it was a clone, i.e. a genetic copy of the original donor adult animal. Presently, the probability with this method of producing severe genetic defects in the clone is very high and the rate of successful cloning experiments with mammals is usually very poor.
Reproductive cloning of humans is banned by law in most countries.

Therapeutic cloning is a cell therapy strategy that uses stem cells to produce healthy copies of a sick person's cells or tissues. Stem cells are prepared from an embryo which results from the transfer of the nucleus from a patient's body cell in an enucleated oocyte. In this way the stem cells contain the genetic material of the patient. Thus, the resulting modified stem cells can grow and be transplanted to repair damaged tissue without causing an immune reaction.
In this strategy, the nucleus is removed from an egg cell and replaced by the nucleus of a cell that was obtained from an adult person (the patient). The modified stem cell thus contains the genetic material of the patient. It is stimulated to divide and form an embryo. This embryo is not transferred into a uterus as it is done with reproductive cloning, but serves as a source of embryonic stem cells. The objective is to obtain specialised cells and tissues that are genetically identical to those of the patient's cells. Potential medical applications are the treatment of degenerative diseases like Parkinson's disease, stroke, liver damage, diabetes, burns and many more.
Clinical application of therapeutic cloning is still far away. Therapeutic cloning is the subject of heated debate as embryos are created and destroyed during the process, thus posing ethical problems. In some countries, therapeutic cloning of human cells is forbidden.

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The term xenotransplantation (from the greek word "xenos" = foreign) stands for the transplantation of animal organs, cells or tissues into human patients. This new approach is being taken to overcome the shortage of human organs available for transplantation. This deficit is expected to increase in both the developed and developing world in the years to come.
Currently, xenotransplantation is still experimental but has potential for clinical applications. One of the major hurdles to xenotransplantation is the rejection of the animal tissues or organs by the patient. Recent advances to overcome this problem include: Furthermore,

For further information see:
Xenotransplantation on the Web
and the Report of WHO Consultation on Xenotransplantation

Just as every person in the world is unique, each of our genomes also exists only once. Gene diagnosis, as a part of modern medicine, is based on analysing special characteristics of individual genomes. Therefore, every result of a test is unique and applies only to a single patient.
Drug response and side-effects of a treatment can be due to genetic factors of a patient. That is why treatment can be individualized. Personalized medicine would mean that every patient receives a tailor-made treatment. Gene diagnosis could be used to select the best treatment that is the most effective one with the least side-effects. It could spare unnecessary inefficient treatment and thus save suffering as well as money.

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Molecular medicine employs diagnostic methods to analyse the genetic makeup of a person. Genetic data of a patient are particularly sensitive information. They describe invariable features, they can enable long-term predictions about a person's health and, in most cases, they also have implications for the patient's relatives.
Some genes or gene variants have been found to be associated with higher risks of developing a certain disease. Data from predictive genetic tests are thus interesting for health insurance companies and employers who may like to know whether their client or worker is prone to developing a severe illness. Discrimination of a person on the basis of her/his genetic data would then be possible. In some countries, the use of genetic data by insurance companies and employers is forbidden by law.
Medical science and the development of new treatments rely on the progress in genomics and on the relevance of genetic data for diseases. On the other hand, patients have a right to the privacy of their medical data. Thus, data protection is an essential and complex issue.

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