Recreating pathophysiology of CLN2 disease and demonstrating reversion by TPP1 gene therapy in hiPSC-derived retinal organoids and retina-on-chip.
在hiPSC衍生的视网膜类器官和芯片视网膜中重现CLN2疾病的病理生理，并证明TPP1基因治疗可逆转该疾病
期刊:Cell Reports Medicine
影响因子:10.6
doi:10.1016/j.xcrm.2025.102244
Corti Serena, Kim Kwi Hye, Chen Ting, Botezatu Adelina, Cora Virginia, Ma Ke, Pashkovskaia Natalia, Bernal Vergara Anamaria, Sperlich Denise, Dave Kaushambee, Tolone Arianna, Reddinger Ryan M, Tully Christopher B, Higgins Mikayla, Kleger Alexander, Breunig Markus, Lopatta Paul, Wingerter Svenja, Cipriano Madalena, Bolz Sylvia, Ueffing Marius, Buss Nicholas, Loskill Peter, Liebau Stefan, Achberger Kevin
