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oai:repisalud.isciii.es:20.500.12105/126062024-09-27T09:27:54Zcom_20.500.12105_19604com_20.500.12105_2051col_20.500.12105_19605

00925njm 22002777a 4500 dc Murillo-Cuesta, Silvia author Artuch, Rafael author Asensio, Fernando author de la Villa, Pedro author Dierssen, Mara author Enriquez, Jose Antonio author Fillat, Cristina author Fourcade, Stéphane author Ibáñez, Borja author Montoliu, Lluis author Oliver, Eduardo author Pujol, Aurora author Salido, Eduardo author Vallejo, Mario author Varela-Nieto, Isabel author 2020-10-14 Animal models are invaluable for biomedical research, especially in the context of rare diseases, which have a very low prevalence and are often complex. Concretely mouse models provide key information on rare disease mechanisms and therapeutic strategies that cannot be obtained by using only alternative methods, and greatly contribute to accelerate the development of new therapeutic options for rare diseases. Despite this, the use of experimental animals remains controversial. The combination of respectful management, ethical laws and transparency regarding animal experimentation contributes to improve society's opinion about biomedical research and positively impacts on research quality, which eventually also benefits patients. Here we present examples of current advances in preclinical research in rare diseases using mouse models, together with our perspective on future directions and challenges. Front Genet. 2020; 11:583932 10.3389/fgene.2020.583932 1664-8021 Frontiers in genetics 33173540 http://hdl.handle.net/20.500.12105/12606 Orphan diseases Animal models Preclinical research Novel therapies Ethics Transparency The Value of Mouse Models of Rare Diseases: A Spanish Experience.