Tuesday, 23 April 2013


Gene Therapy has made important medical advances in less than two decades, but it is in its infancy, and current therapies are primarily experimental. It consists on transferring a gene to an individual´s cell or tissue to treat, cure or prevent a specific disease. It can be used with somatic and germinal cells, but right now, it´s only being developed with somatic cells. In “in vivo” gene therapy, the genetic material is introduced directly into the body of the affected person. Through “ex vivo” gene therapy, cells are extracted from the body and modified through in vitro transfer. Then they are expanded and introduced again in the body of the affected person. 



Gene Therapy can help to treat genetic disorders and acquired diseases:

  1. Gene therapy for genetic disorders:

  • Severe combined immune deficiency: it can be also called ADA-SCID, and affects the correct working of the immune system. It was used in Italy with six patients, without noticeable side effects. The altered gene in this illness is the  adenosine deaminase.
  •     Chronic granulomatus disorder: It is the inability to fight bacterial and fungal infections. Two patients were treated in Germany. Their reconstituted immune systems protected them for two years.
  •   HemophiliaPeople born with Hemophilia can´t induce blood clots and suffer from external and internal bleeding. The therapeutic gene was introduced on the liver of some patients. Then they acquired the ability to clot normally, but those cells introduced before were rejected by patient’s immune system. In Hemophilia A, the modified gene is the Factor VIII. In Hemophilia B, the modified gene is the Factor IX

  •  Other genetic disorders:  A number of clinical trials will soon be launched for various disorders that include congenital blindness, lysosomal storage disease and muscular dystrophy (distrophine is altered).

  1. Gene therapy for acquired diseases

  •       Cancer: Multiple gene therapy strategies have been developed to treat a wide variety of cancers. Two-thirds of all gene therapy trials are for cancer and many of these are very developed, entering the advanced stage.
  •  Neurodegenerative diseases:  Those are Parkinson´s and Huntington´s disease. Some exciting treatment results have been obtained in animals. Clinical trials for those disorders will be soon launched.
  •  Other acquired diseases: Gene therapeutic techniques have been applied to treat HIV, hepatitis, influenza, heart disease and diabetes. 

The human genome project and its applications

The human genome project started in 1990 under the direction of doctor Francis Collins, who led a group of scientists from United States. It´s based on the elaboration of a genetic map of human species and the knowledge of the huge amounts of genes knowing its function and location. In other words, it´s the characteristic  genetic material of human beings, the sequence of DNA unique to humans.
In haploid cells it is compound of 3.000 million bases present in the 23 chromosomes from cellular nucleus.

The Genome Project has meant an extraordinary development for the classification and determination of the sequence from almost all the chromosome bases. At the beginning of the investigation it was needed to create genetic and physical maps with a very high definition, due to the small size of the chromosomes.

Almost all its applications are related to medicine.
With the human genome, we are able to discover  mutations responsible for particular diseases. In some cases, they are hereditary. With that, we can change it and cure people suffering from it.
Using the genome project in a wrong way, could be very harmful, so it was prohibited to use it.
Some people, think that it´ll be useful to use it to make the new population being as perfect as we can, changing genetically the weaker genes and using those techniques to make our babies fit and healthy.
It´s real application now a day is very scarce, because there is a lot of argumentation about it. If we use the Genome in a proportionate and correct way, it´ll be very useful but if it´s used in a wrong way, it could be disastrous and very harmful. When the study finishes, we´ll be able to choose the number of children we want to have and almost all their characteristics, we would be able to cure lots of chronic diseases through individual and calculated doses and drugs.

No comments:

Post a Comment