Research work Genomic Regulation Center in Barcelona, Spain, discovered new molecular mechanisms of Huntington's disease. The results of this study, published in the «Journal of Clinical Investigation» magazine, questioned the methods of treating a disease that is currently practiced. The scientists also noted that the messenger RNA is a key component of the pathogen, which will help identify new therapeutic strategies.
Huntington's disease - a neurodegenerative disease, which is currently considered incurable. Researchers from around the world are working to determine the cause of the disease and the molecular processes in order to find the right medicine.
Huntington's disease is caused by excessive repetition nucleotide triplet (CAG) in a gene disease. The number of CAG repeats can be different. Healthy people have up to 36 repetitions, but the pathology develops with 36 repetitions. A direct consequence of this excess of repetitions is the synthesis of the mutated protein, which is considered the main cause of the disease in the last 20 years.
A new study on mice showed that the mutated fragment - messenger RNA - plays a key role in the pathogenesis. Scientists have suggested that blocking its activity may negate the changes associated with the disease.
The results are of great importance for translational research aimed at the development of new therapies. However, scientists have to conduct a more in-depth study of the molecular mechanisms.
The following studies will show whether it is possible to reverse the effects of Huntington's disease in patients as well, as was demonstrated in laboratory animals. In addition, scientists will investigate whether blocking the activity of messenger RNA to help in the prevention of disease. But, despite the gaps of research, this work is of great importance in the knowledge of the mechanisms of this incurable neurodegenerative disease.