{"id":7913,"date":"2020-09-10T09:31:02","date_gmt":"2020-09-10T09:31:02","guid":{"rendered":"https:\/\/website.prod.unilu.spikeseed.cloud\/en\/news\/new-approach-against-parkinsons-disease-through-stem-cell-research\/"},"modified":"2020-09-10T09:31:02","modified_gmt":"2020-09-10T09:31:02","slug":"new-approach-against-parkinsons-disease-through-stem-cell-research","status":"publish","type":"news","link":"https:\/\/www.uni.lu\/en\/news\/new-approach-against-parkinsons-disease-through-stem-cell-research\/","title":{"rendered":"New approach against Parkinson\u2019s disease through stem cell research"},"content":{"rendered":"

In a seven-year research effort, an international team of scientists has clarified the cause for certain genetic forms of Parkinson\u2019s disease, and has identified potential pharmacological treatments. The interdisciplinary research team, led by Prof. Rejko Kr\u00fcger, of the Luxembourg Centre for Systems Biomedicine (LCSB) of the University of Luxembourg, experimented on patient-based cell cultures in the laboratory. The new combination of active substances they identified will have to undergo clinical trials before they can be used to treat patients. The research team published its results today in the prestigious scientific journal Science Translational Medicine.<\/p>\n

\nLack of protein DJ-1 makes you sick<\/h3>\n

A protein called DJ-1 plays a crucial role in keeping nerve cells functioning. If the body is unable to produce ample amounts of DJ-1, important nerve cells die. The result is the onset of neuro-degenerative diseases such as Parkinson\u2019s. The production of important proteins like DJ-1 can be disrupted or halted permanently if the genetic blueprints or the production processes they encode are defective.<\/p>

Now, Prof. Rejko Kr\u00fcger<\/a>\u2019s research team in Luxembourg has succeeded in identifying for the first time the importance of an error in the production process known as \u2018splicing\u2019 in the development of a certain form of Parkinson\u2019s disease. \u201cIn the patients, an essential tool for the assembly of the protein DJ-1 fails to dock properly,\u201d Kr\u00fcger explains. \u201cIn scientific terms, we call that exon skipping. As a result of this defect, the protein doesn\u2019t get built at all.\u201d The research result offers an entirely new point of attack for treating this malfunction of protein synthesis with drugs. \u201cThis insight fundamentally changes our view of the causes of the disease and presents entirely new possibilities for treatment,\u201d says Dr Ibrahim Boussaad<\/a>, LCSB scientist and first author of the scientific paper. \u201cWe could only gain this new understanding thanks to the skin cells from the patients,\u201d Boussaad emphasises.<\/p>\n

\nCell donation enables progress<\/h3>\n

The Luxembourg Parkinson\u2019s Study, initiated in 2015 (see also www.parkinson.lu<\/a>), includes a group of 800 Parkinson\u2019s patients and 800 healthy control subjects. Thanks to the donation of skin cells taken by small biopsies, the researchers in Luxembourg were able to reprogram these cells to grow into nerve cells in vitro<\/i>. These nerve cells are very similar to the neurons in affected regions of the donor\u2019s brain and can be used for analyses and tests in the laboratory. Because it is not possible to take neurons directly from the brain of patients, for health and ethical reasons, reprogramming is the only way to examine the clinical features of the patient\u2019s neurons in vitro<\/i>. In scientific jargon, this is called a patient-based in vitro<\/i> model, and is an important step in personalised medicine. Using this method, Prof. Kr\u00fcger\u2019s team was able to explain the cause of the genetic form of Parkinson\u2019s disease in which the PARK7 gene is mutated. Prof. Thomas Gasser, a medical director at the T\u00fcbingen University Hospital and co-author of the paper, adds, \u201cWe are proud to have been able to contribute our expertise in the reprogramming of patient cells to this stem cell work of our colleagues in Luxembourg.\u201d Institutions from Germany, Italy and the USA collaborated in the research project.<\/p>

\n \n\"\"\n <\/figure>\n

\nLuxembourg\u2019s interdisciplinarity is a key to this success<\/h3>\n

Precise bioinformatics algorithms developed at the LCSB allowed the research team to immediately carry out an automated search for potential active substances for drug treatment. This yielded a hit in the form of the active compounds phenylbutyric acid and RECTAS (RECTifier of Aberrant Splicing). Administered in combination, these two active substances allow the cells in the test tube to effectively reactivate the production of the important protein DJ-1. \u201cOnly by combining numerous disciplines \u2013 from medical practice, to laboratory research, to computer science \u2013 could we understand the cause and at the same time identify active substances for a potential treatment,\u201d Prof. Rejko Kr\u00fcger explains. He adds, \u201cThis kind of scientific progress \u2018Made in Luxembourg\u2019 is possible because all the necessary disciplines have been unified in Luxembourg for several years now.\u201d This work represents the high point to date of the PEARL program of the Luxembourg National Research Fund (FNR), through which the research of Prof. Kr\u00fcger and his team is funded. The team of scientists especially expresses its gratitude to the people who are participating in the Luxembourg Parkinson\u2019s Study and who have made this research possible in the first place.<\/p>

More information about the Luxembourg Parkinson\u2019s Study, including participation in the study, can be found at www.parkinson.lu<\/a>.<\/p>

Reference: Boussaad et al., A patient-based model of RNA mis-splicing uncovers treatment targets in Parkinson\u2019s disease<\/a>, Science Translational Medicine<\/i>, 9 September 2020. DOI: 10.1126\/scitranslmed.aau3960<\/p>\n

\n\u00a9 University of Luxembourg<\/h5>\n<\/div><\/section>","protected":false},"excerpt":{"rendered":"

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The research team published its results today in the prestigious scientific journal Science Translational Medicine.<\/p>\n","protected":false},"author":0,"featured_media":7914,"template":"","format":"standard","meta":{"featured_image_focal_point":[],"show_featured_caption":false,"ulux_newsletter_groups":"","uluxPostTitle":"","uluxPrePostTitle":"","_trash_the_other_posts":false,"_price":"","_stock":"","_tribe_ticket_header":"","_tribe_default_ticket_provider":"","_tribe_ticket_capacity":"0","_ticket_start_date":"","_ticket_end_date":"","_tribe_ticket_show_description":"","_tribe_ticket_show_not_going":false,"_tribe_ticket_use_global_stock":"","_tribe_ticket_global_stock_level":"","_global_stock_mode":"","_global_stock_cap":"","_tribe_rsvp_for_event":"","_tribe_ticket_going_count":"","_tribe_ticket_not_going_count":"","_tribe_tickets_list":"[]","_tribe_ticket_has_attendee_info_fields":false},"news-category":[11,10],"news-topic":[26],"organisation":[209,227,233],"authorship":[],"acf":[],"yoast_head":"\nNew approach against Parkinson\u2019s disease through stem cell research - University of Luxembourg<\/title>\n<meta name=\"description\" content=\"In a seven-year research effort, an international team of scientists has clarified the cause for certain genetic forms of Parkinson\u2019s disease, and has identified potential pharmacological treatments. 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