a Division of Genetics , Brigham & Women's Hospital, Harvard Medical School , Boston , MA , USA.
b Mass Eye & Ear, Schepens Eye Research Institute, Harvard Medical School , Boston , MA , USA.
Expert Opin Biol Ther. 2018 May;18(5):505-513. doi: 10.1080/14712598.2018.1443442. Epub 2018 Mar 6.
The corneal epithelium is maintained by limbal stem cells (LSCs) that reside in the basal epithelial layer of the tissue surrounding the cornea termed the limbus. Loss of LSCs results in limbal stem cell deficiency (LSCD) that can cause severe visual impairment. Patients with partial LSCD may respond to conservative therapies designed to rehabilitate the remaining LSCs. However, if these conservative approaches fail or, if complete loss of LSCs occurs, transplantation of LSCs or their alternatives is the only option. While a number of clinical studies utilizing diverse surgical and cell culture techniques have shown favorable results, a universal cure for LSCD is still not available. Knowledge of the potential risks and benefits of current approaches, and development of new technologies, is essential for further improvement of LSCD therapies.
This review focuses on cell-based LSCD treatment approaches ranging from current available clinical therapies to preclinical studies of novel promising applications.
Improved understanding of LSC identity and development of LSC expansion methods will influence the evolution of successful LSCD therapies. Ultimately, future controlled clinical studies enabling direct comparison of the diverse employed approaches will help to identify the most effective treatment strategies.
角膜上皮由位于角膜周围组织基底层的角膜缘干细胞（LSCs）维持，该组织称为角膜缘。LSCs 的丧失会导致 LIM 干细胞缺陷（LSCD），从而导致严重的视力损害。部分 LSCD 患者可能对旨在恢复剩余 LSCs 的保守治疗有反应。但是，如果这些保守方法失败，或者 LSCs 完全丧失，则 LSCs 或其替代品的移植是唯一的选择。虽然许多利用不同的手术和细胞培养技术的临床研究已经显示出良好的结果，但 LSCD 仍然没有通用的治疗方法。了解当前方法的潜在风险和益处，并开发新技术，对于进一步改善 LSCD 治疗至关重要。
本综述重点介绍了基于细胞的 LSCD 治疗方法，从现有的临床治疗方法到新的有前途的应用的临床前研究。
对 LSC 特性和 LSC 扩增方法的深入了解将影响成功的 LSCD 治疗方法的发展。最终，未来的对照临床试验能够直接比较所采用的各种方法，将有助于确定最有效的治疗策略。
