Abstract
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.
Highlights
Animal research has contributed greatly to advance human health and quality of life
Animal experimentation is essential for understanding the pathogenic mechanisms of rare disease (RD) and developing new, safe and effective treatments (Garattini and Grignaschi, 2017)
This is especially true for RDs whose low prevalence is associated with a lack of knowledge, delays in diagnosis, and absence of effective treatments in most cases (European Commission, 2017)
Summary
Animal research has contributed greatly to advance human health and quality of life. The use of laboratory animals increased exponentially in the 20th century and they are currently employed in almost every field of biomedical research. Rare Disease Mouse Models culture, and computational methods like data mining/generation, may help to predict clinical outcomes and reduce animal use (Cronin, 2016), but they cannot mimic the complexity of human biology.
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