{"database":"biostudies-literature","file_versions":[],"scores":null,"additional":{"submitter":["Hirayama M"],"funding":["Ministry of Health, Labour and Welfare","Japan Agency for Medical Research and Development","Ministry of Education, Culture, Sports, Science and Technology","Ministry of Economy, Trade and Industry"],"pagination":["101847"],"full_dataset_link":["https://www.ebi.ac.uk/biostudies/studies/S-EPMC11866429"],"repository":["biostudies-literature"],"omics_type":["Unknown"],"volume":["6(1)"],"pubmed_abstract":["A first-in-human investigator-initiated clinical study of a corneal endothelial cell substitute (CLS001) derived from a clinical-grade induced pluripotent stem cell (iPSC) line shows improvement of visual acuity and corneal stromal edema, with no adverse events for up to 1 year after surgery for the treatment of bullous keratopathy. While preclinical tests, including multiple whole-genome analysis and tumorigenicity tests adhering to the Food and Drug Administration (FDA) draft guidelines, are negative, an additional whole-genome analysis conducted on transplanted CLS001 cells reveals a de novo in-frame deletion of exon22 in the EP300 gene. No adverse events related to the mutation are observed. Our study demonstrates the feasibility of using iPSC-derived cells to replace donor transplant "],"journal":["Cell reports. Medicine"],"pubmed_title":["A first-in-human clinical study of an allogenic iPSC-derived corneal endothelial cell substitute transplantation for bullous keratopathy."],"pmcid":["PMC11866429"],"funding_grant_id":["JP21bk0104123"],"pubmed_authors":["Aso K","Dohi H","Negishi K","Okano H","Shimmura S","Hatou S","Sayano T","Hokama R","Hirayama OI","Nomura M","Takasu N","Inagaki E","Hanatani T","Hirayama M"],"additional_accession":[]},"is_claimable":false,"name":"A first-in-human clinical study of an allogenic iPSC-derived corneal endothelial cell substitute transplantation for bullous keratopathy.","description":"A first-in-human investigator-initiated clinical study of a corneal endothelial cell substitute (CLS001) derived from a clinical-grade induced pluripotent stem cell (iPSC) line shows improvement of visual acuity and corneal stromal edema, with no adverse events for up to 1 year after surgery for the treatment of bullous keratopathy. While preclinical tests, including multiple whole-genome analysis and tumorigenicity tests adhering to the Food and Drug Administration (FDA) draft guidelines, are negative, an additional whole-genome analysis conducted on transplanted CLS001 cells reveals a de novo in-frame deletion of exon22 in the EP300 gene. No adverse events related to the mutation are observed. Our study demonstrates the feasibility of using iPSC-derived cells to replace donor transplant ","dates":{"release":"2025-01-01T00:00:00Z","publication":"2025 Jan","modification":"2026-06-02T08:09:36.039Z","creation":"2025-04-04T02:12:34.345Z"},"accession":"S-EPMC11866429","cross_references":{"pubmed":["39809262"],"doi":["10.1016/j.xcrm.2024.101847"]}}