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02/02/2018

A new strategy demonstrates its potential in the treatment of Parkinson's Disease in mice

vila_idibaps_884

02/02/2018

Although research on this treatment is still in preclinical phase, researchers emphasize how these new therapeutic strategies could slow the progression of the symptoms of Parkinson's Disease.

A study published in the journal http://www.cell.com/molecular-therapy-family/molecular-therapy/fulltext/S1525-0016(17)30574-9" Molecular Therapy demonstrates that a new therapeutic strategy reduces the expression of one of the major proteins involved in the progression of Parkinson's Disease, Alpha-synuclein. The study, conducted in mice, was directed by Miquel Vila, ICREA researcher and head of the group of http://en.vhir.org/portal1/grup-equip.asp?s=recerca&contentid=186912 Neurodegenerative Diseases at the Vall d'Hebron Research Institute (VHIR) and Analía Bortolozzi, researcher of the Neuropharmacology and Experimental Neuropathology team of the IDIBAPS and the IIBB-CSIC. The first signatories of the article are Diana Alarcon-Aris (IDIBAPS) and Ariadna Recasens (VH), pre-doctoral researchers in the research team.Parkinson's Disease is the second most common neurodegenerative disorder after Alzheimer's and affects more than 1% of the population. It is characterized by the accumulation of a protein, alpha-synuclein, in the substantia nigra dopaminergic neurons and in other areas of the nervous system. This causes alterations in the function of the dopaminergic circuit and the presence of motor symptoms such as tremors, slowness of movement, as well as also various non-motor symptoms such as sleep stage REM disorder, olfactory dysfunction, depression, cognitive impairments and dementia. Current treatments are symptomatic and most modulate the activity of the motor circuits.For the study, published in Molecular Therapy has been used an inhibitory oligonucleotide, ASO (antisense oligonucleotide) directed against alpha-synuclein to inhibit its synthesis. Oligonucleotides are molecules formed by short sequences of DNA or RNA which are used in gene therapy as a strategy for the silencing or inhibition of genes. Researchers have shown that the intranasal administration in mice of this oligonucleotide allows its accumulation in the brain and leads to the reduction of the gene expression of alpha-synuclein in a type of specific neurons. This decrease causes to increase the release of dopamine and serotonin with which neurotransmission enhances.Although research on this treatment is still in preclinical phase, researchers emphasize that "the results allow us to advance in the knowledge of the role of alpha-synuclein at a physiological level, but more importantly, they show how these new therapeutic strategies could slow the progression of the symptoms of Parkinson's Disease".

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