Treatment of Macular Degeneration Using Embryonic Stem Cell-Derived Retinal Pigment Epithelium: Preliminary Results in Asian Patients

被引:268
作者
Song, Won Kyung [1 ]
Park, Kyung-Mi [2 ]
Kim, Hyun-Ju [2 ]
Lee, Jae Ho [3 ]
Choi, Jinjung [4 ]
Chong, So Young [5 ]
Shim, Sung Han [6 ]
Del Priore, Lucian V. [7 ]
Lanza, Robert [8 ]
机构
[1] CHA Univ, CHA Bundang Med Ctr, Dept Ophthalmol, Songnam 463712, Gyeonggi Do, South Korea
[2] CHA Biotech Co Ltd, Dev Div, Seoul 135907, South Korea
[3] CHA Biotech Co Ltd, CHA Stem Cell Inst, Seoul 135907, South Korea
[4] CHA Univ, CHA Bundang Med Ctr, Dept Internal Med, Div Rheumatol, Songnam 463712, Gyeonggi Do, South Korea
[5] CHA Univ, CHA Bundang Med Ctr, Dept Internal Med, Div Hematol Oncol, Songnam 463712, Gyeonggi Do, South Korea
[6] CHA Univ, Dept Biomed Sci, Seoul 135081, South Korea
[7] Med Univ S Carolina, Albert Florens Storm Eye Inst, Charleston, SC 29425 USA
[8] Ocata Therapeut, Marlborough, MA 01752 USA
来源
STEM CELL REPORTS | 2015年 / 4卷 / 05期
关键词
FACTOR-H GENE; POLYPOIDAL CHOROIDAL VASCULOPATHY; GEOGRAPHIC ATROPHY; VISUAL FUNCTION; POLYMORPHISMS; RPE; ASSOCIATION; TRANSPLANTATION; CHINESE; SAFETY;
D O I
10.1016/j.stemcr.2015.04.005
中图分类号
Q813 [细胞工程];
学科分类号
摘要
Embryonic stem cells hold great promise for various diseases because of their unlimited capacity for self-renewal and ability to differentiate into any cell type in the body. However, despite over 3 decades of research, there have been no reports on the safety and potential efficacy of pluripotent stem cell progeny in Asian patients with any disease. Here, we report the safety and tolerability of subretinal transplantation of human embryonic-stem-cell (hESC)-derived retinal pigment epithelium in four Asian patients: two with dry age-related macular degeneration and two with Stargardt macular dystrophy. They were followed for 1 year. There was no evidence of adverse proliferation, tumorigenicity, ectopic tissue formation, or other serious safety issues related to the transplanted cells. Visual acuity improved 9-19 letters in three patients and remained stable (+1 letter) in one patient. The results confirmed that hESC-derived cells could serve as a potentially safe new source for regenerative medicine.
引用
收藏
页码:860 / 872
页数:13
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