Open access
Jul 2026
Restoration of impaired lysosomal function mitigates drusen-like deposit formation and cell death in Malattia Leventinese
Induced pluripotent stem cell–derived retinal pigment epithelial cells from a patient with MAL showed therapeutic potential for MAL and other drusen-related diseases, including age-related macular degeneration, and pharmacological restoration of lysosomal function alleviated defects.
Yumi Inoue, H. Ikeda, Masayuki Hata et al.
· JCI Insight · 0 citations