Scalable production of RPE cells from induced pluripotent stem cell under GMP conditions for cellular replacement therapy of the dry form of AMD
在 GMP 条件下从诱导多能干细胞大规模生产 RPE 细胞,用于干型 AMD 的细胞替代疗法
基本信息
- 批准号:MR/L022842/1
- 负责人:
- 金额:$ 202.39万
- 依托单位:
- 依托单位国家:英国
- 项目类别:Research Grant
- 财政年份:2014
- 资助国家:英国
- 起止时间:2014 至 无数据
- 项目状态:已结题
- 来源:
- 关键词:
项目摘要
Age related macular degeneration (AMD) is the leading cause of blindness in people over the age of 60 in the western world and each year the problem increases. AMD results in the central portion of vision being lost making it impossible to appreciate fine detail. AMD is associated with defects of the retinal support cells - the retinal pigment epithelial cells (RPE). The rods and cones (the photoreceptors) in the retina, which are the light sensitive cells, depend for their survival on the normal functioning of these cells, and so failure of these cells leads to progressive loss of vision and eventual blindness. There is currently no treatment which prevents the development of AMD. However there has been some success for certain forms of AMD with a range of interventions, but these approaches are only suitable for certain patients and are often only temporary. Our aim is to produce a cell replacement therapy from stem cells derived from patients themselves which are effective in replacing dysfunctional RPE found in AMD which will lead to a surgical therapy capable of stabalising and restoring vision in the vast majority of patients. We plan to develop, over three years, the necessary process of producing stem cells derived from patient skin samples and to turn these stem cells into RPE. We will test the safety and efficacy of these cells ready for use in patients. The goal is to submit a clinical trial application at the end of this project for these patient derived eye cells to initiate a Phase I/II clinical trial.
在西方国家,年龄相关性黄斑变性(AMD)是导致60岁以上人群失明的主要原因,而且这个问题每年都在增加。AMD导致中央部分的视力丧失,使其无法欣赏细节。AMD与视网膜支持细胞-视网膜色素上皮细胞(RPE)的缺陷有关。视网膜上的视杆细胞和视锥细胞(光感受器)是感光细胞,它们的生存依赖于这些细胞的正常功能,因此这些细胞的衰竭会导致视力逐渐丧失,最终失明。目前还没有任何治疗方法可以预防AMD的发展。然而,通过一系列干预措施,某些形式的AMD已经取得了一些成功,但这些方法只适用于某些患者,而且往往只是暂时的。我们的目标是利用来自患者自身的干细胞开发一种细胞替代疗法,这种疗法可以有效地替代AMD中发现的功能失调的RPE,这将导致一种能够稳定和恢复绝大多数患者视力的手术治疗。我们计划在三年内开发出从患者皮肤样本中提取干细胞的必要过程,并将这些干细胞转化为RPE。我们将测试这些准备用于患者的细胞的安全性和有效性。目标是在项目结束时提交临床试验申请,用于这些患者来源的眼细胞,以启动I/II期临床试验。
项目成果
期刊论文数量(7)
专著数量(0)
科研奖励数量(0)
会议论文数量(0)
专利数量(0)
Mbd3 Promotes Reprogramming of Primary Human Fibroblasts.
- DOI:10.15283/ijsc18036
- 发表时间:2018-11-30
- 期刊:
- 影响因子:2.3
- 作者:Jaffer S;Goh P;Abbasian M;Nathwani AC
- 通讯作者:Nathwani AC
Stemming the Tide of Age-Related Macular Degeneration: New Therapies for Old Retinas.
阻止与年龄相关的黄斑变性的浪潮:旧视网膜的新疗法。
- DOI:10.1167/iovs.15-18643
- 发表时间:2016
- 期刊:
- 影响因子:4.4
- 作者:Ramsden CM
- 通讯作者:Ramsden CM
{{
item.title }}
{{ item.translation_title }}
- DOI:
{{ item.doi }} - 发表时间:
{{ item.publish_year }} - 期刊:
- 影响因子:{{ item.factor }}
- 作者:
{{ item.authors }} - 通讯作者:
{{ item.author }}
数据更新时间:{{ journalArticles.updateTime }}
{{ item.title }}
- 作者:
{{ item.author }}
数据更新时间:{{ monograph.updateTime }}
{{ item.title }}
- 作者:
{{ item.author }}
数据更新时间:{{ sciAawards.updateTime }}
{{ item.title }}
- 作者:
{{ item.author }}
数据更新时间:{{ conferencePapers.updateTime }}
{{ item.title }}
- 作者:
{{ item.author }}
数据更新时间:{{ patent.updateTime }}
Peter Coffey其他文献
Risk factors for the occurrence of Escherichia coli virulence genes eae, stx1 and stx2 in wild bird populations
野生鸟类中大肠杆菌毒力基因eae、stx1和stx2出现的危险因素
- DOI:
10.1017/s0950268809002507 - 发表时间:
2009 - 期刊:
- 影响因子:4.2
- 作者:
Laura A. Hughes;Malcolm J. Bennett;Peter Coffey;J. Elliott;Trevor R. Jones;Richard C. Jones;A. Lahuerta;K. McNiffe;David J. Norman;Nicola J. Williams;Julian Chantrey - 通讯作者:
Julian Chantrey
Multi-population analysis reveals spatial consistency in drivers of population dynamics of a declining migratory bird
多种群分析揭示了候鸟数量下降的种群动态驱动因素的空间一致性
- DOI:
- 发表时间:
- 期刊:
- 影响因子:0
- 作者:
C. R. Nater;Malcolm D. Burgess;Peter Coffey;Bob Harris;Frank Lander;David Price;Mike Reed;Robert A. Robinson - 通讯作者:
Robert A. Robinson
RETRACTED ARTICLE: Equestrian injuries presenting to a regional trauma centre in Ireland
- DOI:
10.1007/s11845-018-1952-5 - 发表时间:
2018-12-18 - 期刊:
- 影响因子:1.600
- 作者:
Ali Abdulkarim;Fiachra Richard Power;Peter Coffey;Eoin Sheehan - 通讯作者:
Eoin Sheehan
Peter Coffey的其他文献
{{
item.title }}
{{ item.translation_title }}
- DOI:
{{ item.doi }} - 发表时间:
{{ item.publish_year }} - 期刊:
- 影响因子:{{ item.factor }}
- 作者:
{{ item.authors }} - 通讯作者:
{{ item.author }}
{{ truncateString('Peter Coffey', 18)}}的其他基金
Stem cell based treatment strategy for Age-related Macular Degeneration (AMD)
基于干细胞的年龄相关性黄斑变性(AMD)治疗策略
- 批准号:
G1000730/1 - 财政年份:2010
- 资助金额:
$ 202.39万 - 项目类别:
Research Grant
相似国自然基金
交货期敏感的单件模式产品供应链的协调优化
- 批准号:70871060
- 批准年份:2008
- 资助金额:24.0 万元
- 项目类别:面上项目
供应链中生产和配送联合排序和调度的模型、算法及应用
- 批准号:70372058
- 批准年份:2003
- 资助金额:14.0 万元
- 项目类别:面上项目
相似海外基金
Dysregulation of PPARα in RPE degeneration
RPE 变性中 PPARα 的失调
- 批准号:
10736062 - 财政年份:2023
- 资助金额:
$ 202.39万 - 项目类别:
Damage-Free, Ultrasonic Cell Isolation from Retinal Pigment Epithelium (RPE) Monolayers
从视网膜色素上皮 (RPE) 单层中进行无损伤超声波细胞分离
- 批准号:
10717828 - 财政年份:2023
- 资助金额:
$ 202.39万 - 项目类别:
AMD Mitochondria Modulate Expression of microRNA 135b-5p and 148a-3p in RPE Cybrids: Implications for Age-related Macular Degeneration
AMD 线粒体调节 RPE Cybrids 中 microRNA 135b-5p 和 148a-3p 的表达:对年龄相关性黄斑变性的影响
- 批准号:
10433610 - 财政年份:2022
- 资助金额:
$ 202.39万 - 项目类别:
Metabolic dysfunction from ECM remodeling in diseases of human RPE
人类 RPE 疾病中 ECM 重塑的代谢功能障碍
- 批准号:
10537228 - 财政年份:2022
- 资助金额:
$ 202.39万 - 项目类别:
AMD Mitochondria Modulate Expression of microRNA 135b-5p and 148a-3p in RPE Cybrids: Implications for Age-related Macular Degeneration
AMD 线粒体调节 RPE Cybrids 中 microRNA 135b-5p 和 148a-3p 的表达:对年龄相关性黄斑变性的影响
- 批准号:
10597239 - 财政年份:2022
- 资助金额:
$ 202.39万 - 项目类别:
Metabolic dysfunction from ECM remodeling in diseases of human RPE
人类 RPE 疾病中 ECM 重塑的代谢功能障碍
- 批准号:
10680561 - 财政年份:2022
- 资助金额:
$ 202.39万 - 项目类别:
Promoting RPE repair through modulation of Hippo signaling
通过调节 Hippo 信号传导促进 RPE 修复
- 批准号:
10206803 - 财政年份:2021
- 资助金额:
$ 202.39万 - 项目类别:
Altered RPE matrix in Sorsby Fundus Dystrophy leads to metabolic dysfunction
索尔斯比眼底营养不良症中 RPE 基质的改变导致代谢功能障碍
- 批准号:
10696149 - 财政年份:2021
- 资助金额:
$ 202.39万 - 项目类别:
Promoting RPE repair through modulation of Hippo signaling
通过调节 Hippo 信号传导促进 RPE 修复
- 批准号:
10385789 - 财政年份:2021
- 资助金额:
$ 202.39万 - 项目类别: