June 28, 2011

As previously reported, the scientists used German genetic engineering methods for the prevention of heart disease . Now, these methods can also be used against diseases of blood. American scientists have finally been able to achieve tangible results in the fight against incurable diseases of the blood, in particular, Hemophilia . The new technology allows you to remove genetic defects that trigger the disease in human blood, which can not cope with existing drugs. Scientific advances received called genome editing.

Experiments were carried out on mice. They deliberately infected hemophiliacs, destroys the body's ability to form red blood cells . With this disease, any damage or scratch cause severe bleeding, as a protein needed for the formation of platelets, blood thickening, is not produced. One boy of 30 000 born with the disease. For the normal life requires constant blood transfusion Comprising krovesvertyvayuschie components.

Katherine High Doctor of Children's Hospital of Philadelphia and director of the Center for Cellular and Molecular Therapy, in an interview to the journal Nature reported that editing of the genome   It allowed almost completely restore the normal function of blood, without causing obvious side effects.

In the first step, mice injected viral conductor which should cells liver Responsible for the production of protein. Reaching the goal, they introduced into the cells by enzymes that cause change the genetic sequences. Enzymes carry DNA fragments of healthy genes and replace them damaged areas responsible for hemophilia.

Level of development squirrel rises by 5%. This is sufficient to reduce the risk of hemophilia. With moderate bleeding of the disease can only occur during surgery or major trauma, as in completely healthy people.

Since such acts targeted molecular change and the likelihood of error is minimal, this will avoid the serious consequences, such as the emergence of leukemia and other cancers.