Back

Cell type-focused compound screen in human organoids reveals molecules and pathways controlling cone photoreceptor death

Spirig, S. E.; Arteaga-Moreta, V. J.; Raics, Z.; Posada-Cespedes, S.; Chreng, S.; Galuba, O.; Galuba, I.; Claerr, I.; Renner, S.; Kleindienst, T.; Volak, A.; Imbach, J.; Malysheva, S.; Siwicki, R. A.; Hahaut, V.; Hou, Y.; Picelli, S.; Cattaneo, M.; Jüttner, J.; Cowan, C. S.; Duckely, M.; Baeschlin, D. K.; Renner, M.; Unterreiner, V.; Roska, B.

2023-10-10 neuroscience
10.1101/2023.10.09.561525 bioRxiv
Show abstract

Human organoids that mirror their corresponding organs in cell-type diversity present an opportunity to perform large-scale screens for compounds that protect disease-affected or damage healthy cell types. However, such screens have not yet been performed. Here, we generated 20,000 human retinal organoids with GFP-labeled cone photoreceptors. Since degeneration of cones is a leading cause of blindness, we induced cone death and screened 2,707 compounds with known targets, for those that saved cones or those that further damaged cones. We identified inhibitors of CK1 or MAPK11 that protected cones, HSP90 inhibitors that saved cones in the short term but damaged them in the longer term, and broad HDAC inhibition by many compounds that significantly damaged cones. This work provides a database for cone-damaging compounds and describes compounds that can be starting points to develop neuroprotection for cones in diseases such as macular degeneration.

Published in Neuron (predicted rank #9) · training set

Matching journals

The top 6 journals account for 50% of the predicted probability mass.

50% of probability mass above

"Similar papers" are the closest papers from that journal in the model's embedding space. They show what the match is built on, but the ranking comes mostly from a classifier over the whole training set, not from these examples alone.