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The approach of molecular medicine to the treatment of Gaucher disease

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dc.contributor.author ROTARU, Ludmila
dc.contributor.author SIDORENKO, Ludmila
dc.date.accessioned 2024-04-26T09:18:02Z
dc.date.available 2024-04-26T09:18:02Z
dc.date.issued 2024
dc.identifier.citation ROTARU, Ludmila, SIDORENKO, Ludmila. The approach of molecular medicine to the treatment of Gaucher disease. In: Quo Vadis – Ethics of the Scientific Research: conf. NANO-2024, event devoted to the 60th anniversary of the Technical University of Moldova, 15-18 April 2024, Chişinău, Republica Moldova: Program and proceedings of the conference, Chişinău 2024, pp. 96-97. ISBN 978-9975-64-422-8. en_US
dc.identifier.isbn 978-9975-64-422-8
dc.identifier.uri http://repository.utm.md/handle/5014/26950
dc.description.abstract Gaucher disease presents a metabolic rare disease with the autosomal recessive transmission. The cause of this monogenic disease is the mutation of the gene GBA. As a consequence of this mutation, a deficiency of synthesis of the enzyme β- gucocerebrosidase occurs. As a result, glucocerebrosides accumulate throughout the body, especially in the bone marrow, spleen and liver. Three different forms of Gaucher disease have been determined, distinguished by the absence or presence and extent of neurological complications. Currently, five therapies for the treatment of Gaucher disease are described. This review study provides the most recent information on the therapies approved for the treatment of the disease. en_US
dc.language.iso en en_US
dc.publisher Technical University of Moldova en_US
dc.relation.ispartofseries NANO-2024 "Quo Vadis – Ethics of the Scientific Research", event devoted to the 60th anniversary of the Technical University of Moldova;15-18 April 2024, Chişinău, Republica Moldova
dc.rights Attribution-NonCommercial-NoDerivs 3.0 United States *
dc.rights.uri http://creativecommons.org/licenses/by-nc-nd/3.0/us/ *
dc.subject Gaucher disease en_US
dc.subject metabolic disease en_US
dc.subject autosomal recessive transmission en_US
dc.title The approach of molecular medicine to the treatment of Gaucher disease en_US
dc.type Article en_US


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