Human iPSCs as Model Systems for BMP-Related Rare Diseases.

ABSTRACT: Disturbances in bone morphogenetic protein (BMP) signalling contribute to onset and development of a number of rare genetic diseases, including Fibrodysplasia ossificans progressiva (FOP), Pulmonary arterial hypertension (PAH), and Hereditary haemorrhagic telangiectasia (HHT). After decades of animal research to build a solid foundation in understanding the underlying molecular mechanisms, the progressive implementation of iPSC-based patient-derived models will improve drug development by addressing drug efficacy, specificity, and toxicity in a complex humanized environment. We will review the current state of literature on iPSC-derived model systems in this field, with special emphasis on the access to patient source material and the complications that may come with it. Given the essentia

SUBMITTER: Sanchez-Duffhues G 

PROVIDER: S-EPMC10487005 | biostudies-literature | 2023 Sep

REPOSITORIES: biostudies-literature