{"id":20749,"date":"2024-07-11T08:59:36","date_gmt":"2024-07-11T06:59:36","guid":{"rendered":"https:\/\/ela-international.preprod.melisagency.fr\/?post_type=news_research&p=20749"},"modified":"2024-07-11T10:42:34","modified_gmt":"2024-07-11T08:42:34","slug":"study-of-gpr65-as-a-new-krabbe-disease-drug-target","status":"publish","type":"news_research","link":"https:\/\/elainternational.eu\/en\/actualites_recherche\/study-of-gpr65-as-a-new-krabbe-disease-drug-target\/","title":{"rendered":"Study of GPR65 as a new Krabbe disease drug target"},"content":{"rendered":"\n\n
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Study of GPR65 as a new Krabbe disease drug target<\/strong><\/h3>\n <\/div>\n <\/div>\n<\/section>\n\n\n
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Sara Carpi – Department of Health Sciences, University \u2018 Magna Gr\u00e6cia\u2019 of Catanzaro, Catanzaro, Italy and Marco Cecchini – NEST, Istituto Nanoscienze \u2010 CNR, Pisa, Italy – ELA 2023\u2010006C4<\/em><\/p>\n <\/div>\n<\/section>\n\n\n\n\n\n

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Description of the project<\/strong><\/h5>\n <\/div>\n
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Krabbe disease (KD, or Globoid cell leukodystrophy) is a neurodegenerative disease caused by a deficiency of the lysosomal enzyme GALC. The early childhood form represents 85\u201090% of cases and the onset of symptoms is between the 3rd and the 6th month after birth. During the advanced state, blindness and deafness show up; then a vegetative state arises which ends with death within the first 1\u20102 years after birth. Unfortunately, for infants who have already developed symptoms of KD, there is no treatment that can change the course of the disease. Treatment, therefore, focuses on managing symptoms and providing supportive care.<\/p>\n

Currently, the literature indicates that GALC\u2010deficiency correction might not be sufficient to cure the patients with KD, suggesting that not yet identified players could be implicated in KD pathogenesis. In this context, we identified some clear evidence pointing to the protein named GPR65 as a key factor in the pathogenesis of the disease.<\/p>\n

Here, we propose a deep investigation of GPR65 and its evaluation as a druggable target in KD. In particular, we will evaluate GPR65 expression in patients with KD and test drugs to correct GPR65 deficiency in KD cell models. Moreover, we will explore the combination of a GPR65 mRNA\u2010based therapy with the GALC correction.<\/p>\n

In case of a successful project outcome and in a forward\u2010looking vision, GPR65 mRNA\u2010based therapy might be used in combination with a main GALC\u2010deficiency correcting therapy, to help in achieving the complete KD phenotype rescue.<\/p>\n<\/div>\n<\/div>\n<\/div>\n<\/div>\n<\/div>\n<\/div>\n

Project financed by ELA up to: 60 000 \u20ac<\/p>\n <\/div>\n <\/div>\n<\/section>","protected":false},"excerpt":{"rendered":"","protected":false},"author":2,"featured_media":0,"parent":0,"template":"","news_research_categories":[127],"news_research_tags":[129,114],"class_list":["post-20749","news_research","type-news_research","status-publish","hentry","news_research_categories-project","news_research_tags-krabbe-disease","news_research_tags-leukodystrophy"],"acf":[],"yoast_head":"\nStudy of GPR65 as a new Krabbe disease drug target - ELA International<\/title>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/elainternational.eu\/en\/actualites_recherche\/study-of-gpr65-as-a-new-krabbe-disease-drug-target\/\" \/>\n<meta property=\"og:locale\" content=\"en_US\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"Study of GPR65 as a new Krabbe disease drug target - 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