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Tools to study congenital brain malformations and vision disorders in organoids

"It is an exciting time for personalized medicine. We expect there to be rapid innovations to further develop brain and retina organoid protocols for high-throughput screening, as well as toward near-physiological completeness, and single-cell genomic technologies are in place to resolve neurodevelopmental and vision disorders with extraordinary resolution."

In this perspective, Giovanna Brancati, PostDoc in our Human Retina and Organoid Development Group, headed by Gray Camp and her co-authors explain how stem cell-derived human organoids combined with single-cell genomic phenotyping provide an exciting new inroad to understanding neurodevelopmental and vision disorders, and discuss next steps in organoid engineering to address current deficiencies.

Explore human neurodevelopment and neurodegeneration in controlled culture environments

Human organoid models of the central nervous system, including the neural retina, are providing unprecedented opportunities to explore human neurodevelopment and neurodegeneration in controlled culture environments. The authors discuss how the single-cell multi-omic toolkit has been used to identify features and limitations of brain and retina organoids, and how these tools can be deployed to study congenital brain malformations and vision disorders in organoids. They also address how to improve brain and retina organoid protocols to revolutionize in vitro disease modeling.

Organoids can model disorders of the human central nervous system

"There are major concerns that organoids are less physiological than the animal models that have so far been instrumental for our understanding of brain and retina development. However, organoids are a complementary new technology that, in addition to being human derived, have the added advantage of being amenable to high-throughput manipulations in controlled culture settings. With its high throughput and the rich amount of information provided, single-cell RNA sequencing (scRNA-seq)—and generally single-cell technologies—has, in our opinion, established itself as a technique of choice to reveal and help quantify the heterogeneity typical of organoids, laying the foundation for their use in disease modeling, drug discovery, and personalized medicine", the authors write.

Original publication

Resolving neurodevelopmental and vision disorders using organoid single-cell multi-omics
Giovanna Brancati, Barbara Treutlein, J. Gray Camp
Neuron, Sep 23, 2020, Volume 107Issue 6p989-1238, doi: