U.S.A. scientists have used a new genome-modifying technique to treat thalassemia (Mediterranean anemia) in mice, according to data published in the journal Nature Communications.
Mediterranean anemia is an inherited blood disease, which reduces the production of hemoglobin and thus causes a lack of oxygen in the body, which has a variety of consequences (pale skin, weakness, fatigue and more serious complications).
Researchers at Yale and Carnegie Mellon University, led by Professor Peter Glazer, have developed a new minimally invasive strategy for correcting the mutations that cause thalassemia. They used an innovative combination of nanoparticles, synthetic DNA fragments and intravenous infusion and thus treated the animals. Specifically, they took a protein from the bone marrow that activates blood stem cells and combined that protein with synthetic molecules (nucleic acid peptides, known as DNA) that mimic DNA and attach to the defective HB target gene.
This triggers a process in the blood-producing cell, a process of self-correction of the mutant pathogen. DNA molecules are transported through biocompatible nanoparticles, which are introduced into the body by intravenous injection. Although the success rate was around 7%, in terms of successful genetic correction in hematopoietic stem cells, this was enough to eliminate the anemia. The mice no longer had any symptoms of the disease and after five months the hemoglobin levels in their blood were normal.
The achievement paves the way for the application of the new genetic technique to humans. Doctors hope that with the help of genetics they will be able to cure hereditary blood disorders, such as thalassemia and sickle cell disease.
According to Dr. Glazer, the new method is superior to alternative genetic techniques such as competing CRISPR, which can have side effects, such as mutating an unrelated gene. If the technique proves to be effective in future clinical trials in other animals, it could eventually lead to the treatment of Mediterranean anemia in humans – and possibly other haematological disorders, such as sickle cell disease.
But it will take years until a final conclusion is reached.
SOURCE: www.health.in.gr
