12 million for rare hereditary diseases
The EU has allocated 12 million euros for the development of new methods of diagnosis and treatment
rare genetic diseases
Weekly Pharmacy www.apteka.ua based on the materials of NEUROMICS and the European Commission:
World's top researchers collaborate to diagnose and treat rare genetic diseasesAccording to the press service of the European Commission, the EU has allocated a grant of 12 million euros for the development of new methods for the diagnosis and treatment of rare genetic diseases under the Seventh Framework Program of the EU (Seventh Framework Program for research and technological development).
To achieve the stated goals, a 5-year NEUROMICS project (Integrated European Project on Omics Research of Rare Neuromuscular and Neurodegenerative Diseases – an integrated European project for innovative Research of Rare Neuromuscular and Neurodegenerative Diseases) was launched in October 2012. It was headed by Olaf Riess, Professor of the Department of Medical Genetics at the Institute of Human Genetics at the University of Tübingen.
The project will bring together leading research groups in Europe, 5 most innovative small and medium-sized enterprises (SME enterprises) working in the field of R&D, and a number of research institutions from the USA and Australia. The main task of the project will be the use of innovative Omics technologies (Omics is an English professional neologism formed from the suffix of terms such as "genomics", "proteomics", "metabolomics". Omics technologies are usually understood as innovations created as a result of advanced research on the structure and functions of nucleic acids, proteins and metabolic processes). The research of the project participants will focus on the diagnosis and creation of pathomechanism-oriented methods of treatment of the 10 most common neuromuscular and neurodegenerative diseases (Huntington's disease, frontotemporal dementia, hereditary spastic paraplegia, ataxia, spinal muscular atrophy, hereditary motor-sensory neuropathies, congenital myasthenic syndrome, congenital dystrophy and myopathy, muscular dystrophy, neuromuscular channelopathy).
One of the priority tasks of the project is the development of new drugs working on the principle of antisense therapy (antisense therapy is a therapy technique based on stopping the synthesis of a protein involved in the development of the disease by inhibiting the translation of its matrix RNA using short nucleotide sequences complementary to it) and histone deacetylase inhibitors (a class of drugs that, by influencing the "packaging" of genetic material in the chromosome reduces the availability of DNA for transcription factors and thereby inhibits the synthesis of a certain protein).
Neurodegenerative and neuromuscular disorders are among the most common among orphan diseases, which are noted in more than 500 thousand patients in Europe.
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