iBB

Endogenous human brain tissue is not easily available for studying neurodevelopment and neurodegenerative diseases. However, human pluripotent stem cells (PSCs) have been used to generate a variety of glial and neuronal cells of the central nervous system. Still, reproducible protocols for generating in vitro models of the human cerebellum are scarce. In this context, Silva et al. describe the scalable production of human PSC-derived cerebellar organoids using single-use vertical-wheel bioreactors. The transcriptomic profile of cerebellar organoids derived under dynamic conditions demonstrates a faster cerebellar differentiation combined with significant enrichment of extracellular matrix and upregulation of transcripts involved in angiogenesis when compared with the static protocol. The authors anticipate that large-scale production of cerebellar organoids may help developing models for drug screening, toxicological tests and studying pathological pathways involved in cerebellar degeneration.

Evguenia Bekman and Tiago Fernandes from SCERG-iBB together with Simão Teixeira da Rocha from IMM-FMUL have been awarded by the Amélia de Mello Foundation (FAM) with the Pedro Maria José de Mello Costa Duarte Scholarship to carry out the project “Exploring new therapeutic strategies for Angelman syndrome: A disease modeling approach based on 3D cerebellar organoids derived from patient iPSCs”. The Angelman Syndrome is a genetic-neurological disease discovered in the 60s of the last century by the English pediatrician Harry Angelman, which is estimated to affect 1 in every 15,000 live births and which is characterized by developmental delay, difficulty in speech, sleep disorders, convulsions, disconnected movements and a frequent smile.