{"doi":"10.1084/jem.20230913","title":"Identifying biomarkers of heterogeneity and transplantation efficacy in retinal pigment epithelial cells","abstract":"Transplantation of retinal pigment epithelial (RPE) cells holds great promise for patients with retinal degenerative diseases, such as age-related macular degeneration. In-depth characterization of RPE cell product identity and critical quality attributes are needed to enhance efficacy and safety of replacement therapy strategies. Here, we characterized an adult RPE stem cell-derived (RPESC-RPE) cell product using bulk and single-cell RNA sequencing (scRNA-seq), assessing functional cell integration in vitro into a mature RPE monolayer and in vivo efficacy by vision rescue in the Royal College of Surgeons rats. scRNA-seq revealed several distinct subpopulations in the RPESC-RPE product, some with progenitor markers. We identified RPE clusters expressing genes associated with in vivo efficacy and increased cell integration capability. Gene expression analysis revealed lncRNA (TREX) as a predictive marker of in vivo efficacy. TREX knockdown decreased cell integration while overexpression increased integration in vitro and improved vision rescue in the RCS rats.","journal":"The Journal of Experimental Medicine","year":2023,"id":328729,"datarank":0.0,"base_score":0.0,"endowment":0.0,"self_citation_contribution":0.0,"citation_network_contribution":0.0,"self_endowment_contribution":0.0,"citer_contribution":0.0,"corpus_percentile":null,"corpus_rank":null,"citation_count":22,"citer_count":0,"citers_with_citation_signal":0,"citers_with_endowment":0,"datacite_reuse_total":0,"is_dataset":false,"is_dataset_confidence":0.9483,"is_data_producer":false,"deposit_databanks":null,"is_oa":true,"file_count":0,"downloads":0,"has_version_chain":false,"published_date":"2023-01-01","fair_score":null,"fair_percentile":null,"algorithm_id":"datarank_citation_only_1hop_v6","ranking_scope":"data_only","authors":[{"id":994184,"name":"Justine D. Manos","orcid":"0000-0002-4265-4016","position":1,"is_corresponding":false},{"id":1050706,"name":"Yue Wang","orcid":"0009-0007-6647-3379","position":2,"is_corresponding":false},{"id":994185,"name":"Anne L. Williams","orcid":"0000-0002-2491-5294","position":3,"is_corresponding":false},{"id":1050707,"name":"Cuiping Zhao","orcid":"0009-0003-3885-7093","position":4,"is_corresponding":false},{"id":994187,"name":"Susan Borden","orcid":"0009-0001-2361-1686","position":5,"is_corresponding":false},{"id":318193,"name":"Nazia M. Alam","orcid":"0000-0002-1941-2190","position":6,"is_corresponding":false},{"id":318194,"name":"Glen T. Prusky","orcid":"0000-0001-9658-8598","position":7,"is_corresponding":false},{"id":40564,"name":"Sally Temple","orcid":"0000-0001-7301-783X","position":8,"is_corresponding":false},{"id":788721,"name":"Jeffrey H. Stern","orcid":"0000-0002-5272-5025","position":9,"is_corresponding":false},{"id":626239,"name":"Nathan C. Boles","orcid":"0000-0002-9253-9411","position":10,"is_corresponding":false},{"id":505255,"name":"Farhad Farjood","orcid":"0000-0002-8826-6721","position":0,"is_corresponding":true}],"reference_count":44,"raw_metadata":null,"created_at":"2026-07-19T01:08:56.940647Z","pmid":"37728563","pmcid":null,"fwci":null,"citation_percentile":null,"influential_citations":0,"oa_status":null,"license":null,"views":0,"total_file_size_bytes":0,"version_count":0,"fair_f":null,"fair_a":null,"fair_i":null,"fair_r":null,"fair_zscore":null,"fair_rationale":null,"fair_model":null,"fair_agent_version":null,"fair_fulltext_source":null,"fair_has_llm":null,"fair_computed_at":null,"clinical_trials":[],"software_tools":[],"db_accessions":[],"linked_datasets":[],"topics":[]}