Stem cell sources and characterization in the development of cell-based products for treating retinal disease: An NEI Town Hall report

被引:9
作者
Fortress, Ashley M. [1 ]
Miyagishima, Kiyoharu J. [2 ]
Reed, Amberlynn A. [1 ]
Temple, Sally [3 ]
Clegg, Dennis O. [4 ]
Tucker, Budd A. [5 ]
Blenkinsop, Timothy A. [6 ]
Harb, George [7 ]
Greenwell, Thomas N. [1 ]
Ludwig, Tenneille E. [8 ]
Bharti, Kapil [9 ]
机构
[1] NEI, NIH, Bethesda, MD 20892 USA
[2] NEI, Retinal Neurophysiol Sect, NIH, Bethesda, MD 20892 USA
[3] Neural Stem Cell Inst, Rensselaer, NY USA
[4] Univ Calif Santa Barbara, Ctr Stem Cell Biol & Engn, Santa Barbara, CA USA
[5] Univ Iowa, Inst Vis Res, Dept Ophthalmol & Visual Sci, Carver Coll Med, Iowa City, IA USA
[6] Icahn Sch Med Mt Sinai, Black Family Stem Cell Inst, Ophthalmol Cell Dev & Regenerat Biol, New York, NY USA
[7] Cellino Biotech, Cambridge, MA USA
[8] WiCell Res Inst, Madison, WI USA
[9] NEI, Ocular & Stem Cell Translat Res, NIH, Bethesda, MD 20892 USA
关键词
Embryonic stem cells; Induced pluripotent stem cells; Clinical manufacturing; Cell replacement therapy; Retinal degeneration; Autologous; Allogeneic; Translational research; Cell sources; Cell characterization; PIGMENT EPITHELIUM; HUMAN RPE; MACULAR DEGENERATION; GANGLION-CELLS; TRANSPLANTATION; SURVIVAL; IPSCS; PHOTORECEPTORS; FIBROBLASTS; GENERATION;
D O I
10.1186/s13287-023-03282-y
中图分类号
Q813 [细胞工程];
学科分类号
摘要
National Eye Institute recently issued a new Strategic Plan outlining priority research areas for the next 5 years. Starting cell source for deriving stem cell lines is as an area with gaps and opportunities for making progress in regenerative medicine, a key area of emphasis within the NEI Strategic Plan. There is a critical need to understand how starting cell source affects the cell therapy product and what specific manufacturing capabilities and quality control standards are required for autologous vs allogeneic stem cell sources. With the goal of addressing some of these questions, in discussion with the community-at-large, NEI hosted a Town Hall at the Association for Research in Vision and Ophthalmology annual meeting in May 2022. This session leveraged recent clinical advances in autologous and allogeneic RPE replacement strategies to develop guidance for upcoming cell therapies for photoreceptors, retinal ganglion cells, and other ocular cell types. Our focus on stem cell-based therapies for RPE underscores the relatively advanced stage of RPE cell therapies to patients with several ongoing clinical trials. Thus, this workshop encouraged lessons learned from the RPE field to help accelerate progress in developing stem cell-based therapies in other ocular tissues. This report provides a synthesis of the key points discussed at the Town Hall and highlights needs and opportunities in ocular regenerative medicine.
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页数:13
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