Aug 2026
A 3D human retina model reveals a non-cell-autonomous and non-neuronal mechanism of photoreceptor loss in a lysosomal storage disorder.
Treatment with recombinant human acid ceramidase (rhAC) ameliorated photoreceptor degeneration in both the CLN3 disease RO-RPE model and CLN3 miniswine eyes, suggesting that rhAC could be a therapeutic approach for retinal degeneration in CLN3 disease.
Jimin Han, Nathaniel Foley, Sonal Dalvi et al.
· Science Translational Medici... · 0 citations