{"doi":"10.1056/nejmoa2309915","title":"Gene Editing for <i>CEP290</i> -Associated Retinal Degeneration","abstract":"BACKGROUND: (IVS26 variant). METHODS: -associated inherited retinal degeneration caused by a homozygous or compound heterozygous IVS26 variant received a subretinal injection of EDIT-101 in the worse (study) eye. The primary outcome was safety, which included adverse events and dose-limiting toxic effects. Key secondary efficacy outcomes were the change from baseline in the best corrected visual acuity, the retinal sensitivity detected with the use of full-field stimulus testing (FST), the score on the Ora-Visual Navigation Challenge mobility test, and the vision-related quality-of-life score on the National Eye Institute Visual Function Questionnaire-25 (in adults) or the Children's Visual Function Questionnaire (in children). RESULTS: of the minimum angle of resolution (range, 3.9 to 0.6). No serious adverse events related to the treatment or procedure and no dose-limiting toxic effects were recorded. Six participants had a meaningful improvement from baseline in cone-mediated vision as assessed with the use of FST, of whom 5 had improvement in at least one other key secondary outcome. Nine participants (64%) had a meaningful improvement from baseline in the best corrected visual acuity, the sensitivity to red light as measured with FST, or the score on the mobility test. Six participants had a meaningful improvement from baseline in the vision-related quality-of-life score. CONCLUSIONS: and other genetic causes. (Funded by Editas Medicine and others; BRILLIANCE ClinicalTrials.gov number, NCT03872479.).","journal":"New England Journal of Medicine","year":2024,"id":416231,"datarank":3.309750393451642,"base_score":5.3230099791384085,"endowment":5.3230099791384085,"self_citation_contribution":0.7984514968707613,"citation_network_contribution":2.5112988965808807,"self_endowment_contribution":0.7984514968707613,"citer_contribution":2.5112988965808807,"corpus_percentile":null,"corpus_rank":null,"citation_count":204,"citer_count":100,"citers_with_citation_signal":100,"citers_with_endowment":100,"datacite_reuse_total":0,"is_dataset":false,"is_dataset_confidence":0.9545,"is_data_producer":true,"deposit_databanks":{"ClinicalTrials.gov":["NCT03872479"]},"is_oa":true,"file_count":0,"downloads":0,"has_version_chain":false,"published_date":"2024-01-01","fair_score":null,"fair_percentile":null,"algorithm_id":"datarank_citation_only_1hop_v6","ranking_scope":"data_only","authors":[{"id":368842,"name":"Tomás S. Alemán","orcid":"0000-0001-5709-0236","position":1,"is_corresponding":false},{"id":1200441,"name":"Kanishka T. Jayasundera","orcid":null,"position":2,"is_corresponding":false},{"id":382736,"name":"Bright S. Ashimatey","orcid":"0000-0002-1319-1008","position":3,"is_corresponding":false},{"id":1199324,"name":"Keunpyo Kim","orcid":"0000-0002-7103-924X","position":4,"is_corresponding":false},{"id":1199325,"name":"Alia Rashid","orcid":"0000-0001-7563-7442","position":5,"is_corresponding":false},{"id":658841,"name":"Michael Jaskolka","orcid":"0000-0003-2341-0750","position":6,"is_corresponding":false},{"id":19951,"name":"R Myers","orcid":"0000-0001-9701-676X","position":7,"is_corresponding":false},{"id":1200442,"name":"Byron L Lam","orcid":null,"position":8,"is_corresponding":false},{"id":295377,"name":"Steven T. Bailey","orcid":"0000-0003-4949-1464","position":9,"is_corresponding":false},{"id":226909,"name":"Jason Comander","orcid":"0000-0002-4257-7145","position":10,"is_corresponding":false},{"id":473532,"name":"Andreas Lauer","orcid":"0000-0001-7582-9030","position":11,"is_corresponding":false},{"id":896476,"name":"Albert M. Maguire","orcid":"0000-0001-7030-2301","position":12,"is_corresponding":false},{"id":249764,"name":"Mark E. Pennesi","orcid":"0000-0002-4943-6599","position":13,"is_corresponding":false},{"id":280705,"name":"Eric A. Pierce","orcid":"0000-0002-2354-4102","position":0,"is_corresponding":true}],"reference_count":43,"raw_metadata":null,"created_at":"2026-07-19T01:56:20.964471Z","pmid":"38709228","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":[]}