Important terms explained simply
A virus used in research to deliver genes into cells. It is not dangerous because it no longer functions properly.
In certain tissues (e.g., bone marrow). Can only develop into certain cells.
Cells from another person (donor). They are sometimes used in transplants. This can be quick and inexpensive, but the immune system might recognize the cells as foreign and reject them.
Cells from the patient’s own body. This is personalized, but expensive and time-consuming. Sometimes they are used so that the body does not reject them.
A sign in the body that indicates whether something is wrong or whether a treatment is helping.
An immune cell (T cell) produced by the body that is modified in the laboratory so that it can better recognize and eliminate cancer cells.
A “bundle” of genetic information. Humans have 46 chromosomes.
A kind of “gene scissors.” It can be used to cut out and replace a defective gene. Very precise.
YouTube video from the Max Planck Society: Gene editing with CRISPR/Cas9 (English subtitles)
A cell becomes a specific type of cell (e.g., nerve cell or muscle cell)
The cell is not functioning properly—for example, because a gene is defective.
Cells from a very young embryo. They can develop into almost any type of cell. However, they come from an embryo—which is difficult for some people to accept.
These are changes that “turn off” or “turn on” the gene—without the DNA itself changing.
The cells are removed from the body, modified in the laboratory, and then reintroduced into the body.
A component in your body that determines your appearance and how your cells function. Sometimes a gene is defective, which can lead to a disease.
A person’s entire genetic information.
A method for specifically modifying a gene—like a “text editor” for DNA. CRISPR is an example of this.
A treatment in which a defective gene in the body is “repaired” or replaced with a healthy gene. Goal: to cure or prevent diseases.
A gene is introduced into a cell.
Cells that form all blood cells (red blood cells, white blood cells, platelets).
Cells of a different type (e.g., animal cells). Not suitable for treating humans.
One gene is defective, the other is normal.
Both copies of a gene are defective.
Skin cells or other cells are converted back into stem cells—without an embryo.
The treatment takes place in the laboratory (e.g., cells are treated in a test tube).
The treatment takes place directly inside the body (e.g., an injection)—without removing cells.
A research study in which new treatments are tested on people to see if they are safe and effective.
Cells that multiply uncontrollably. Sometimes they are combated with special cells.
DNA and RNA—these are the building blocks of genetic information.
A cell can transform into almost any other cell.
A component in the body that performs many functions (e.g., enzymes, receptors).
A type of medicine that aims to replace diseased or damaged cells.
DNA composed of different sources (e.g., a human gene inserted into a bacterium).
An “antenna” on the cell that detects something (e.g., a drug or a virus).
Genetic modification in somatic cells. These changes are not passed on to the children.
A cell that can reproduce itself and transform into other cells (e.g., a nerve cell or a blood cell). It helps heal tissue.
A gene that has been introduced from another species or through research.
A cell, tissue, or organ is transferred from one person to another.
A collection of abnormal cells. May be benign or malignant.
An immune cell. In CAR-T therapy, it is modified to recognize cancer cells.
A “transporter” (e.g., a virus) that delivers a gene into a cell.
In gene therapy, a harmless virus is used to deliver the healthy gene into the cells. The virus is not dangerous because it no longer functions properly. The virus is merely a “delivery service.”
Viruses that have been modified so that they only transport genes into cells. The virus does not cause illness. It is merely a "delivery service."
A cell that can transform into a specific type of cell.
Cells are cultured and multiplied in the laboratory so that there are enough of them to perform a therapy.
A treatment in which healthy cells are introduced into the body to replace or support diseased cells.