Gene therapy

Background and need of Gene therapy

Gene therapy is a form of genetic engineering and it is a form of treatment for a certain type of disease. It is a revolutionary treatment method as no previous treatment methods have been used by similar methods. Gene therapy treats diseases caused by single defective genes that lead to mutations and thus various types of diseases such as cystic fibrosis, hemorrhagic disease and muscular dystrophy. Also diseases caused by the lack of certain types of genes such as SCID. As technology, research and our curiosity progress, the need for new treatment methods increases, the more problems we discover the more solutions we seek, the more new diseases we discover the more new methods we need. Our technology in healthcare and research has been revolutionized somewhat unbelievably when it comes to the human body, which has also given us several explanations for the emergence of various diseases. In the past, under the direction of the church’s board, the only explanation for illnesses was that, according to God, it was worth it and that you had to ask forgiveness to be healthy, which fortunately, knowledge has increased and today we know about the rise to many more diseases, which means that you can also counteract these emergencies that you do through gene therapy, among other things. The need for alternative treatment methods has also increased as more diseases have been discovered that require alternative treatment methods. Today, there are approximately 2,800 diseases caused by single defective genes and many researchers believe that gene therapy could be the solution to them. These diseases are due to mutations in our genetic material that can be affected by our environment (epigenetics) or occur spontaneously.


It is common for people to ”inherit” diseases or at least tend to diseases of their parents through the hereditary mass. A total of 23 chromosomes are inherited from their parents, par 23 are the sex chromosomes, two x chromosomes for women and one x chromosome and one Y chromosome for men. The other 22 chromosomes are very similar and they are called autosomes. A gene found on one of the autosomes is called the autosomal gene. Various genetic abnormalities occur, such as an extra chromosome 21 in all the cells of the body which causes down syndrome or an extra chromosome 18 that causes Edwards syndrome. Note, however, these diseases do not result from progeny, but from germ cell division when the chromosomes are broken down incorrectly.


Most of the genes are in the cell nucleus and the genes are in turn in the 23 chromosome pairs we inherit. We thus inherit genes from our parents, genes with a tendency for certain traits. Different chromosomes carry different genes, different properties, which is why an extra chromosome 18 causes Edwards Syndrome, it becomes too much chromosome with the same kind of genes, properties. When the body gets an extra chromosome, they receive the same information several times, which means that the body’s development does not happen as it should. Diseases are usually due to the lack of something like a protein, but it can also be due to, for example, having too much set of something, like a chromosome here. One could compare it to the fact that an uneven cell division, of which too many cells in the same place, leads to tumors / nodules. The picture shows symptoms in children with Edwards syndrome, also called trisomy 18.


A man inherits a predisposition in biology means that he inherits a ”trait”, a predisposition that he inherits can be a predisposition to a disease. The tendency one inherits can either be dominant or a recessive one, depending on how much these tendencies control when it comes to different properties in the body. By characteristics, I do not mean whether a person is funny or nice, but characteristics in the body that you inherit from their parents or even earlier generations. That is why you usually have the same features as your mom or maybe the same nose as your dad, you usually have the same or similar hair color. A dominant trait can pass on certain traits with only one inheritance, that is, it is enough to get a dominant inheritance grant for a trait to go through. In contrast, recessive traits “give up” for dominant traits and it is necessary to inherit inheritance from both parents for the trait to pass through.