What is it about?

As a genetic malignancy, retinoblastoma (Rb) is caused by RB1 mutations. We have shown an innovative Rb organoid model derived from human stem cells with a biallelic mutagenesis of the RB1 gene. The tumorigenic growth was observed in dish and SYK inhibitors display a significant therapeutic effect.

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Why is it important?

This elegant in-dish Rb organoid model can be used to efficiently and effectively dissect the origin of Rb and mechanisms of Rb tumorigenesis, as well as screen novel therapies.

Perspectives

I hope this article provides a useful tool of medical and research explorations for biological dissection and therapeutic development of the most prevalent intraocular malignancy in children.

Zi-Bing Jin

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This page is a summary of: Human embryonic stem cell-derived organoid retinoblastoma reveals a cancerous origin, Proceedings of the National Academy of Sciences, December 2020, Proceedings of the National Academy of Sciences,
DOI: 10.1073/pnas.2011780117.
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