Supporting data for “ CONTINUOUS SECRETION OF GLIAL CELL- DERIVED NEUROTROPHIC FACTOR (GDNF) INTO THE VITREOUS BY TERMINABLE CELL- ENCAPSULATING COLLAGEN-ALGINATE COMPOSITE (CAC) GEL AS A THERAPY FOR RETINAL DEGENERATIVE DISEASES”

Retinal degenerative diseases (RDDs) including age related macular degeneration (AMD), and retinitis pigmentosa (RP) can lead to irreversible vision impairment and blindness. Management of RDDs remains elusive. Encapsulated cell therapy (ECT) has shown its promising potential to provide continuous and sustained delivery of therapeutics by encapsulating drug-secreting cells into gel matrix/semipermeable membrane to treat RDDs. Previous work by our team developed collagen-alginate composite (CAC) encapsulated cell therapy (ECT)gels in which glial cell-derived neurotrophic factor (GDNF)-secreting human embryonic kidney (HEK) cells were enclosed to provide continuous GDNF supply in the rat eye. Aiming to translate CAC ECT gel to clinical application, this study investigated (1) in vivo safety of CAC ECT gel, (2) in vivo performance of CAC ECT gel, and (3) in vivo termination of CAC ECT gels by oral administration of doxycycline (Dox) in healthy New Zealand White (NZW) rabbits, and (4) in vivo therapeutical potential of CAC ECT gel in NZW rabbits with retinal degeneration induced by intravenous injection of sodium iodate (SI).

视网膜退行性疾病（RDDs），包括年龄相关性黄斑变性（AMD）和色素性视网膜炎（RP），可能导致不可逆的视力损害和失明。RDDs的管理至今尚无良策。封装细胞疗法（ECT）通过将药物分泌细胞封装于凝胶基质/半透膜中以提供持续的药物治疗，已显示出其巨大的应用潜力。本研究团队先前开发的胶原蛋白-海藻酸盐复合材料（CAC）封装细胞疗法（ECT）凝胶，其中包含分泌神经营养因子（GDNF）的人胚胎肾（HEK）细胞，旨在为鼠眼提供持续的GDNF供应。本研究旨在将CAC ECT凝胶应用于临床，调查了以下内容：（1）CAC ECT凝胶的体内安全性；（2）CAC ECT凝胶的体内性能；（3）通过口服强力霉素（Dox）诱导新西兰白兔体内CAC ECT凝胶的终止；（4）CAC ECT凝胶在由静脉注射碘酸钠（SI）诱导的视网膜退变新西兰白兔中的体内治疗潜力。