1. GENE THERAPY
Introduction:
Gene therapy is a technique by which scientists and doctors can modify the genome of a
living being in order to treat a disease. Genetic engineering has been used to investigate
on the human genome but the most important application is in the medicine fields such
as the development of drugs and also the curing and treatment of diseases in order to
save lives.
Basically gene therapy consists on insert, eliminate or modify the genes which are the
cause of this disease.
Some examples of diseases that can be treated by gene therapy are cancer and
hemophilia, so we see that either acquired diseases or the ones caused by a genetic
disorder can be treated in this way.
Development:
Hemophilia is a very dangerous disease caused by a genetic disorder, and which affects
lots of people.
Hemophilia consists on the body of a person not being able to induce blood clots, this
meaning that the person can suffer an internal or external bleeding, which is really
dangerous and probably will put in risk this person’s life.
It is caused by the lack of the protein in charge of the clotting of blood which works
with the platelets (the ones who actually stick together in order to plug the hole), and
with the absence of this protein the wound cannot be stopped from bleeding.
What genetic engineering does is to, for example in Hemophilia B (as there are two
types of hemophilia) which there is a mutation in their clotting Factor IX gene, is cured
by using an adenovirus which will send a proper copy of Factor IX.

2. Benefits and limitations:
The obvious benefits of this therapy applied in the curing of hemophilia, is that as
hemophilia is a very dangerous disease which may mean the end of the person’s life,
and it actually affects a lot of people around the world, the treatment and curing of this
disease has an enormous benefit for humanity.
Also the fact that this disease is caused by a single disorder on one gene is really “easy”
to cure.
Always the development of these diseases means directly an advance of medicine,
especially on the field of the human genome.
The main limitation just now is the fact that the experiments done for now in the
medical trials haven’t proven a definitive solution or results, so for now it doesn’t have
a direct effect. Also this treatment may have side effects caused by antibodies produced
because of the viral vector introduced in the person’s body.
Conclusion:
To conclude I want to say that I think this is a really important advance on curing a very
dangerous disease as hemophilia. Even if the results are not yet definitive, the
development of this treatment is great, and little by little medicine will save some of the
people who suffer from it.
Bibliography:
http://www.iptv.org/exploremore/ge/uses/use2_gene.cfm
http://scienceblogs.com/erv/2012/09/25/gene-therapy-for-hemophilia-a/
http://www.asgct.org/about_gene_therapy/diseases.php