期刊
SCIENCE TRANSLATIONAL MEDICINE
卷 11, 期 475, 页码 -出版社
AMER ASSOC ADVANCEMENT SCIENCE
DOI: 10.1126/scitranslmed.aat5580
关键词
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资金
- NEI IRP funds
- Common Fund Therapeutic Challenge Award
- NATIONAL EYE INSTITUTE [ZIAEY000456, ZICEY000461, ZIAEY000530, ZICEY000503] Funding Source: NIH RePORTER
- NATIONAL EYE INSTITUTE
- COMMON FUND, OFFICE OF THE DIRECTOR [ZIAEY000542] Funding Source: NIH RePORTER
Considerable progress has been made in testing stem cell-derived retinal pigment epithelium (RPE) as a potential therapy for age-related macular degeneration (AMD). However, the recent reports of oncogenic mutations in induced pluripotent stem cells (iPSCs) underlie the need for robust manufacturing and functional validation of clinical-grade iPSC-derived RPE before transplantation. Here, we developed oncogenic mutation-free clinical-grade iPSCs from three AMD patients and differentiated them into clinical-grade iPSC-RPE patches on biodegradable scaffolds. Functional validation of clinical-grade iPSC-RPE patches revealed specific features that distinguished transplantable from nontransplantable patches. Compared to RPE cells in suspension, our biodegradable scaffold approach improved integration and functionality of RPE patches in rats and in a porcine laser-induced RPE injury model that mimics AMD-like eye conditions. Our results suggest that the in vitro and in vivo preclinical functional validation of iPSC-RPE patches developed here might ultimately be useful for evaluation and optimization of autologous iPSC-based therapies.
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