Lancaster MA, Renner M, Martin CA, Wenzel D, Bicknell LS, Hurles ME, Homfray T, Penninger JM, Jackson AP, Knoblich JA.
Cerebral organoids model human brain development and microcephaly.
Nature. 2013 Aug 28;
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Although there is a long way to go to phenotypically build a real model for human brain in 3D cultures, this strategy could potentially revolutionize research on neurodevelopment disorders, particularly Down syndrome.
The model would potentially allow us to dissect the effects of over-expressing genes one by one, and identify those that are indeed real players in causing cognitive dysfunction in people with Down syndrome. We have been doing this for the last decade using mice. However, no mouse model can perfectly model human conditions. In addition, the new strategy could be used for therapy, allowing us to test the effects of different drugs on human trisomic neurons' structure and function in vitro.