Use of Zebrafish Models to Boost Research in Rare Genetic Diseases.

ABSTRACT: Rare genetic diseases are a group of pathologies with often unmet clinical needs. Even if rare by a single genetic disease (from 1/2000 to 1/more than 1,000,000), the total number of patients concerned account for approximatively 400 million peoples worldwide. Finding treatments remains challenging due to the complexity of these diseases, the small number of patients and the challenge in conducting clinical trials. Therefore, innovative preclinical research strategies are required. The zebrafish has emerged as a powerful animal model for investigating rare diseases. Zebrafish combines conserved vertebrate characteristics with high rate of breeding, limited housing requirements and low costs. More than 84% of human genes responsible for diseases present an orthologue, suggesting that the ma

SUBMITTER: Crouzier L 

PROVIDER: S-EPMC8706563 | biostudies-literature | 2021 Dec

REPOSITORIES: biostudies-literature