{"doi":"10.1126/scitranslmed.aax9106","title":"Gene-edited human stem cell–derived β cells from a patient with monogenic diabetes reverse preexisting diabetes in mice","abstract":") in iPSCs derived from a patient with Wolfram syndrome (WS). After differentiation to β cells with our recent six-stage differentiation strategy, corrected WS SC-β cells performed robust dynamic insulin secretion in vitro in response to glucose and reversed preexisting streptozocin-induced diabetes after transplantation into mice. Single-cell transcriptomics showed that corrected SC-β cells displayed increased insulin and decreased expression of genes associated with endoplasmic reticulum stress. CRISPR-Cas9 correction of a diabetes-inducing gene variant thus allows for robust differentiation of autologous SC-β cells that can reverse severe diabetes in an animal model.","journal":"Science Translational Medicine","year":2020,"id":50648,"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":184,"citer_count":0,"citers_with_citation_signal":0,"citers_with_endowment":0,"datacite_reuse_total":0,"is_dataset":false,"is_dataset_confidence":0.9402,"is_data_producer":false,"deposit_databanks":null,"is_oa":true,"file_count":0,"downloads":0,"has_version_chain":false,"published_date":"2020-01-01","fair_score":null,"fair_percentile":null,"algorithm_id":"datarank_citation_only_1hop_v6","ranking_scope":"data_only","authors":[{"id":246839,"name":"Punn Augsornworawat","orcid":"0000-0001-8602-5433","position":1,"is_corresponding":false},{"id":246841,"name":"Leonardo Velazco-Cruz","orcid":"0000-0003-4664-3936","position":2,"is_corresponding":false},{"id":250007,"name":"Michelle H. Kim","orcid":"0000-0003-0096-8276","position":3,"is_corresponding":false},{"id":252815,"name":"Rie Asada","orcid":null,"position":4,"is_corresponding":false},{"id":250008,"name":"Nathaniel J. Hogrebe","orcid":"0000-0002-6282-2056","position":5,"is_corresponding":false},{"id":250009,"name":"Shuntaro Morikawa","orcid":"0000-0001-5547-9491","position":6,"is_corresponding":false},{"id":250010,"name":"Fumihiko Urano","orcid":"0000-0002-9944-6293","position":7,"is_corresponding":false},{"id":7381,"name":"Jeffrey R. Millman","orcid":"0000-0003-0426-3492","position":8,"is_corresponding":false},{"id":246840,"name":"Kristina G. Maxwell","orcid":"0000-0003-0869-1662","position":0,"is_corresponding":true}],"reference_count":75,"raw_metadata":null,"created_at":"2026-07-18T20:39:29.006085Z","pmid":"32321868","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":[]}