An investigation carried out by scientists from the Center for Genomic Regulation of Barcelona (CRG) has succeeded in delaying the progression of Huntington's disease (HD) in mice. To do this, researchers have used proteins called 'zinc fingers' to reduce the expression of the mutant gene involved in this pathology.
In Huntington's disease there is an alteration in the gene that encodes a protein called huntingtin, which is essential for the development and maintenance of neurons. This mutated gene is the cause of Huntington's symptoms, which consist of the appearance of involuntary movements that the patient can not control, in addition to changes in the character. As the disease progresses, there are also signs of dementia.
When the treatment was applied to mice with Huntington's disease, the appearance of the symptoms characteristic of this pathology was delayed.
Since HD is caused by the anomaly of a single gene, a therapy aimed at inhibiting that gene could achieve control of this disease for which, at present, there is no cure.
Researchers from the CGR have developed 'zinc fingers' that recognize and bind to certain DNA sequences, which allows regulating the genes to which they are attached. In the case of the mutant gene that causes Huntington's disease, the 'zinc fingers' prevent its expression, thus decreasing the production of the abnormal huntingtin protein.
The authors of the study, whose findings have published 'Proceedings of the National Academy of Sciences' (PNAS), observed that when they applied the treatment to mouse models with HD, the onset of symptoms of the disease was delayed. Now, the scientists intend to continue with the project to develop a treatment that is effective in humans.