NeurosciencebioRxivPreprint
A 3D iPSC retina model reveals non-cell-autonomous and non-neuronal mechanism of photoreceptor degeneration in a lysosomal storage disorder.
Jimin Han, Nathaniel Foley, Sonal Dalvi…·Ruchira Singh
This study uses a 3D iPSC retina model to show that acid ceramidase deficiency causes sphingolipid metabolism changes and photoreceptor degeneration in CLN3 disease, with therapeutic potential for rhAC.
DOI 10.1101/2025.07.10.664233