Establishment and characterization of human pluripotent stem cells-derived brain organoids to model cerebellar diseases.

ABSTRACT: The establishment of robust human brain organoids to model cerebellar diseases is essential to study new therapeutic strategies for cerebellum-associated disorders. Machado-Joseph disease (MJD) is a cerebellar hereditary neurodegenerative disease, without therapeutic options able to prevent the disease progression. In the present work, control and MJD induced-pluripotent stem cells were used to establish human brain organoids. These organoids were characterized regarding brain development, cell type composition, and MJD-associated neuropathology markers, to evaluate their value for cerebellar diseases modeling. Our data indicate that the organoids recapitulated, to some extent, aspects of brain development, such as astroglia emerging after neurons and the presence of ventricular-like zones

SUBMITTER: Bras J 

PROVIDER: S-EPMC9307606 | biostudies-literature | 2022 Jul

REPOSITORIES: biostudies-literature