Induced pluripotent stem cell-based modeling of mutant LRRK2-associated Parkinson's disease.

ABSTRACT: Recent advances in cell reprogramming have enabled assessment of disease-related cellular traits in patient-derived somatic cells, thus providing a versatile platform for disease modeling and drug development. Given the limited access to vital human brain cells, this technology is especially relevant for neurodegenerative disorders such as Parkinson's disease (PD) as a tool to decipher underlying pathomechanisms. Importantly, recent progress in genome-editing technologies has provided an ability to analyze isogenic induced pluripotent stem cell (iPSC) pairs that differ only in a single genetic change, thus allowing a thorough assessment of the molecular and cellular phenotypes that result from monogenetic risk factors. In this review, we summarize the current state of iPSC-based modeling o

SUBMITTER: Weykopf B 

PROVIDER: S-EPMC7114274 | biostudies-literature | 2019 Feb

REPOSITORIES: biostudies-literature