scholarly journals Derivation and Functional Analysis of Patient-Specific Induced Pluripotent Stem Cells as an In Vitro Model of Chronic Granulomatous Disease

Stem Cells ◽  
2012 ◽  
Vol 30 (4) ◽  
pp. 599-611 ◽  
Author(s):  
Yan Jiang ◽  
Sally A. Cowley ◽  
Ulrich Siler ◽  
Dario Melguizo ◽  
Katarzyna Tilgner ◽  
...  
Keyword(s):  
Stem Cells ◽  
Functional Analysis ◽  
Chronic Granulomatous Disease ◽  
Pluripotent Stem Cells ◽  
In Vitro Model ◽  
Patient Specific ◽  
Granulomatous Disease ◽  
Vitro Model ◽  
Induced Pluripotent

scholarly journals Modeling congenital cataract in vitro using patient-specific induced pluripotent stem cells

2021 ◽  
Vol 6 (1) ◽  
Author(s):  
Danni Lyu ◽  
Lifang Zhang ◽  
Zhenwei Qin ◽  
Shuang Ni ◽  
Jiayong Li ◽  
...  
Keyword(s):  
Stem Cells ◽  
Induced Pluripotent Stem Cells ◽  
Pluripotent Stem Cells ◽  
Congenital Cataract ◽  
In Vitro Model ◽  
Patient Specific ◽  
Congenital Cataracts ◽  
Vitro Model ◽  
Induced Pluripotent

AbstractCongenital cataracts are the leading cause of childhood blindness. To date, surgical removal of cataracts is the only established treatment, but surgery is associated with multiple complications, which often lead to visual impairment. Therefore, mechanistic studies and drug-candidate screening have been intrigued by the aims of developing novel therapeutic strategies. However, these studies have been hampered by a lack of an appropriate human-disease model of congenital cataracts. Herein, we report the establishment of a human congenital cataract in vitro model through differentiation of patient-specific induced pluripotent stem cells (iPSCs) into regenerated lenses. The regenerated lenses derived from patient-specific iPSCs with known causative mutations of congenital cataracts (CRYBB2 [p. P24T] and CRYGD [p. Q155X]) showed obvious opacification that closely resembled that seen in patients’ cataracts in terms of opacification severity and disease course accordingly, as compared with lentoid bodies (LBs) derived from healthy individuals. Increased protein aggregation and decreased protein solubility corresponding to the patients’ cataract severity were observed in the patient-specific LBs and were attenuated by lanosterol treatment. Taken together, the in vitro model described herein, which recapitulates patient-specific clinical manifestations of congenital cataracts and protein aggregation in patient-specific LBs, provides a robust system for research on the pathological mechanisms of cataracts and screening of drug candidates for cataract treatment.

Optimization of X-linked chronic granulomatous disease modelization by using patient-specific induced pluripotent stem cells

2013 ◽  
Vol 41 (8) ◽  
pp. S28
Author(s):  
Julie Brault ◽  
Erwan Goutagny ◽  
Tomo Saric ◽  
Karl-Heinz Krause ◽  
Marie-José Stasia ◽  
...  
Keyword(s):  
Stem Cells ◽  
Chronic Granulomatous Disease ◽  
Induced Pluripotent Stem Cells ◽  
Pluripotent Stem Cells ◽  
Patient Specific ◽  
Granulomatous Disease ◽  
Induced Pluripotent

scholarly journals 3D In Vitro Model of a Functional Epidermal Permeability Barrier from Human Embryonic Stem Cells and Induced Pluripotent Stem Cells

2014 ◽  
Vol 2 (5) ◽  
pp. 675-689 ◽  
Author(s):  
Anastasia Petrova ◽  
Anna Celli ◽  
Laureen Jacquet ◽  
Dimitra Dafou ◽  
Debra Crumrine ◽  
...  
Keyword(s):  
Stem Cells ◽  
Pluripotent Stem Cells ◽  
In Vitro Model ◽  
Embryonic Stem ◽  
Permeability Barrier ◽  
Epidermal Permeability Barrier ◽  
3D In Vitro Model ◽  
Vitro Model ◽  
Induced Pluripotent
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