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Induced to cure: Using iPS cells to develop autologous cell therapy for degenerative eye diseases

机译:诱导治愈:利用iPS细胞开发自体细胞疗法治疗眼部退行性疾病

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??The recent success with embryonic stem (ES) cell derived retinal pigment epithelium (RPE) has provided hope for a treatment for degenerative eye diseases. Induced pluripotent stem (iPS) cells are an alternate and an autologous source of stem cells potentially with fewer immune-challenges as compared to ES cells. Using a developmentally guided differentiation protocol we have developed fully polarized RPE tissue from iPS cells. The RPE monolayer along with its secreted ECM and the scaffold form a tissue that well mimics the native tissue. This tissue has been functionally authenticated in vitro for its ability to perform several key RPE functions and is being tested in animal models. We have begun manufacturing for a Phase I Investigational New Drug (IND) to transplant autologous iPS cell derived RPE in patients in advanced Geographic Atrophy stage of age-related macular degeneration (AMD), one of the leading blinding diseases in the US. We propose to develop National Institutes of Health Clinical Center as an a??alpha-stem cell clinica?? with the capability to recruit patients, manufacture clinical-grade autologous iPS cell derived RPE tissue, perform transplantation, do patient care, and be able to transfer technology to academic and private sectors. We suggest that our open access model with complete access to the entire IND package will reduce redundant efforts in the field, foster public-private partnerships, and help move ocular regenerative medicine field forward.
机译:胚胎干(ES)细胞源性视网膜色素上皮(RPE)的最新成功为治疗退行性眼病提供了希望。诱导多能干(iPS)细胞是干细胞的替代来源和自体来源,与ES细胞相比,可能具有更少的免疫挑战。使用开发指导的分化方案,我们已经从iPS细胞开发了完全极化的RPE组织。 RPE单层及其分泌的ECM和支架形成了一个很好地模仿天然组织的组织。该组织具有执行多种关键RPE功能的能力,已在体外进行了功能验证,并且正在动物模型中进行测试。我们已经开始制造第一阶段研究用新药(IND),以将自体iPS细胞衍生的RPE移植到年龄相关性黄斑变性(AMD)的晚期地理萎缩阶段的患者中,AMD是美国主要的致盲疾病之一。我们建议将国立卫生研究院临床中心发展为一个“α-干细胞临床诊所”。具有招募患者,制造临床级自体iPS细胞来源的RPE组织,进行移植,进行患者护理以及将技术转移到学术和私人领域的能力。我们建议,我们的开放获取模式可完全访问整个IND软件包,这将减少该领域的重复工作,促进公私合作,并有助于推动眼再生医学领域的发展。

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