Knockout mouse models as a resource for rare diseases studies

Abstract excerpt

<title>Abstract</title><p>Rare diseases (RDs) are a challenge for medicine due to their heterogeneous clinical manifestation and low prevalence. There is a lack of specific treatments and only a few hundred of the approximately 7.000 RDs have an approved regime. Rapid technological development in genome sequencing enables the mass identification of potential candidates that in their mutated form could trigger dise...

Topics

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• Animals, Genetically Modified
• Disease Models, Animal
• Gene Knockout Techniques
• Genetic Predisposition to Disease
• Mice
• Mice, Knockout
• Phenotype
• Rare Diseases

Identifiers and source

Literature Corpus work

939e5f86-22c2-5a2a-93f9-e256acd61931

DOI

10.21203/rs.3.rs-1956287/v1