Subretinal Transplantation of Human Embryonic Stem Cell-Derived Retinal Tissue in a Feline Large Animal Model.
Subretinal Transplantation of Human Embryonic Stem Cell-Derived Retinal Tissue in a Feline Large Animal Model.
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DOI：
10.3791/61683
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发表时间：
2021-08-05
期刊：
影响因子：
--
通讯作者：
Petersen-Jones SM
中科院分区：
文献类型：
--
作者：
Occelli LM;Marinho F;Singh RK;Binette F;Nasonkin IO;Petersen-Jones SM
视网膜退行性疾病（RD）与光感受器丧失相关，如老年性黄斑变性（AMD）、色素性视网膜炎（RP）和莱伯先天性黑内障（LCA）会导致进行性和衰弱性视力丧失。一旦光感受器丧失，对恢复视力的治疗方法的需求尚未得到满足。将人类多能干细胞（hPSC）衍生的视网膜组织（类器官）移植到患有晚期RD的眼睛的视网膜下空间，可以带来具有数千个健康无突变光感受器的视网膜组织片，并且有可能通过一种批准的方案治疗大多数/所有与光感受器变性相关的致盲疾病。胚胎视网膜组织移植到动物模型和晚期RD患者的视网膜下空间已经成功开发，但由于伦理问题和组织供应有限，不能作为常规治疗方法。利用先进的手术方法将视网膜细胞/组织移植到视网膜下间隙，大眼遗传性视网膜变性（IRD）动物模型对于开发视力恢复疗法具有重要价值。球体大小和光感受器分布的相似性（例如，黄斑样区域中央区域的存在）以及IRD模型的可用性密切再现了人类IRD，将有助于将有希望的治疗方法快速转化为临床。本文介绍了一种将hpsc来源的视网膜组织移植到大型动物模型的视网膜下空间的手术技术，允许在动物模型中评估这种有前途的方法。
Retinal degenerative (RD) conditions associated with photoreceptor loss such as age-related macular degeneration (AMD), retinitis pigmentosa (RP) and Leber Congenital Amaurosis (LCA) cause progressive and debilitating vision loss. There is an unmet need for therapies that can restore vision once photoreceptors have been lost. Transplantation of human pluripotent stem cell (hPSC)-derived retinal tissue (organoids) into the subretinal space of an eye with advanced RD brings retinal tissue sheets with thousands of healthy mutation-free photoreceptors and has a potential to treat most/all blinding diseases associated with photoreceptor degeneration with one approved protocol. Transplantation of fetal retinal tissue into the subretinal space of animal models and people with advanced RD has been developed successfully but cannot be used as a routine therapy due to ethical concerns and limited tissue supply. Large eye inherited retinal degeneration (IRD) animal models are valuable for developing vision restoration therapies utilizing advanced surgical approaches to transplant retinal cells/tissue into the subretinal space. The similarities in globe size, and photoreceptor distribution (e.g., presence of macula-like region area centralis) and availability of IRD models closely recapitulating human IRD would facilitate rapid translation of a promising therapy to the clinic. Presented here is a surgical technique of transplanting hPSC-derived retinal tissue into the subretinal space of a large animal model allowing assessment of this promising approach in animal models.
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影响因子：
5.9
作者：
Assawachananont, Juthaporn;Mandai, Michiko;Okamoto, Satoshi;Yamada, Chikako;Eiraku, Mototsugu;Yonemura, Shigenobu;Sasai, Yoshiki;Takahashi, Masayo
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作者：
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12.4
作者：
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通讯作者：
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