The present invention provides ocular cells, genetically modified by a CRISPR system targeting the expression of B2M for ocular cell therapy. The invention further provides methods of generating an expanded population of genetically modified ocular cells, for example limbal stem cells (LSCs) or corneal endothelial cells (CECs), wherein the cells are expanded involving the use of a LATS inhibitor and the expression of B2M in the cells has been reduced or eliminated. The present invention also provides a cell populations, preparations, uses and methods of therapy comprising said cells.
本发明提供了通过CRI
SPR系统靶向B2M表达进行
基因修饰的眼部细胞,用于眼部细胞治疗。该发明进一步提供了生成扩增的
基因修饰眼部细胞群体的方法,例如角膜边缘干细胞(LSCs)或角膜内皮细胞(C
ECs),其中细胞的扩增涉及使用
LATS抑制剂,并且细胞中的B2M表达已经降低或消除。本发明还提供了细胞群体、制剂、用途和包括所述细胞的治疗方法。