{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,17]],"date-time":"2026-09-17T19:03:07Z","timestamp":1789671787685,"version":"4.0.1"},"reference-count":49,"publisher":"SAGE Publications","license":[{"start":{"date-parts":[[2026,9,14]],"date-time":"2026-09-14T00:00:00Z","timestamp":1789344000000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"},{"start":{"date-parts":[[2026,9,14]],"date-time":"2026-09-14T00:00:00Z","timestamp":1789344000000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/journals.sagepub.com\/page\/policies\/text-and-data-mining-license"}],"funder":[{"DOI":"10.13039\/501100001659","name":"Deutsche Forschungsgemeinschaft","doi-asserted-by":"crossref","award":["CRC1690; Project A01, Project No. 528760423"],"award-info":[{"award-number":["CRC1690; Project A01, Project No. 528760423"]}],"id":[{"id":"10.13039\/501100001659","id-type":"DOI","asserted-by":"crossref"}]},{"DOI":"10.13039\/501100001659","name":"Deutsche Forschungsgemeinschaft","doi-asserted-by":"crossref","award":["EXC 2067\/1-390729940"],"award-info":[{"award-number":["EXC 2067\/1-390729940"]}],"id":[{"id":"10.13039\/501100001659","id-type":"DOI","asserted-by":"crossref"}]}],"content-domain":{"domain":["journals.sagepub.com"],"crossmark-restriction":true},"short-container-title":["Human Gene Therapy"],"abstract":"<jats:p>\n                    Advancing the development of specific gene therapies for rare genetic eye disorders is a major challenge in modern translational vision research. It requires integrated clinical, molecular, mechanistic, and regulatory expertise, yet these aspects are often addressed in isolation. Combining complementary skills, knowledge, and expertise within an integrated, highly interactive research framework has the capacity to drive and advance therapeutic development. Moreover, early-stage involvement of patients and targeted clinical observation, alongside molecular and therapeutic research, potentially enable clinical trial readiness and support the translation of preclinical therapeutic innovations into clinical care. In this Perspective article, we present the InsightRP2 framework, an integrated translational strategy that incorporates clinical data, artificial intelligence\u2013supported imaging analysis, experimental disease modeling, and adeno-associated virus design with the aim to facilitate the development of a targeted gene therapy for\n                    <jats:italic toggle=\"yes\">RP2<\/jats:italic>\n                    -associated retinitis pigmentosa.\n                  <\/jats:p>","DOI":"10.1177\/10430342261480906","type":"journal-article","created":{"date-parts":[[2026,9,15]],"date-time":"2026-09-15T06:16:47Z","timestamp":1789453007000},"update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":0,"title":["InsightRP2\u2014An Interdisciplinary Approach Toward Therapy Development in\n                    <i>RP2<\/i>\n                    -Associated Retinopathy"],"prefix":"10.1177","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-4300-8224","authenticated-orcid":false,"given":"Nina","family":"B\u00f6gershausen","sequence":"first","affiliation":[{"name":"Institute of Human Genetics, University Medical Center G\u00f6ttingen, G\u00f6ttingen, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"G\u00f6khan","family":"Yigit","sequence":"additional","affiliation":[{"name":"Institute of Human Genetics, University Medical Center G\u00f6ttingen, G\u00f6ttingen, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alisa","family":"Fedorenko","sequence":"additional","affiliation":[{"name":"Institute of Human Genetics, University Medical Center G\u00f6ttingen, G\u00f6ttingen, Germany"},{"name":"Stem Cell Unit, Clinic for Cardiology and Pneumology, University Medical Center G\u00f6ttingen, G\u00f6ttingen, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rozhin","family":"Bayati","sequence":"additional","affiliation":[{"name":"Institute of Human Genetics, University Medical Center G\u00f6ttingen, G\u00f6ttingen, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-4767-7759","authenticated-orcid":false,"given":"Patricia","family":"D\u00f6ring","sequence":"additional","affiliation":[{"name":"Department of Ophthalmology, University Medical Center G\u00f6ttingen, G\u00f6ttingen, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Maximilian","family":"Gerhardt","sequence":"additional","affiliation":[{"name":"Department of Ophthalmology, LMU University Hospital, LMU Munich, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Carolin","family":"Strohmeyer","sequence":"additional","affiliation":[{"name":"Department of Ophthalmology, University Medical Center G\u00f6ttingen, G\u00f6ttingen, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Oscar","family":"Guti\u00e9rrez-Guti\u00e9rrez","sequence":"additional","affiliation":[{"name":"Stem Cell Unit, Clinic for Cardiology and Pneumology, University Medical Center G\u00f6ttingen, G\u00f6ttingen, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9317-6114","authenticated-orcid":false,"given":"Behnam","family":"Javanmardi","sequence":"additional","affiliation":[{"name":"Institute for Genomic Statistics and Bioinformatics, University Hospital Bonn, Bonn, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marianne","family":"Gaubert","sequence":"additional","affiliation":[{"name":"Institute of Human Genetics, University Medical Center G\u00f6ttingen, G\u00f6ttingen, Germany"},{"name":"German Center for Child and Adolescent Health (DZKJ), Partner Site G\u00f6ttingen, G\u00f6ttingen, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Emrah","family":"Kaygusuz","sequence":"additional","affiliation":[{"name":"Institute of Human Genetics, University Medical Center G\u00f6ttingen, G\u00f6ttingen, Germany"},{"name":"Department of Molecular Biology and Genetics, Faculty of Science, Bilecik \u015eeyh Edebali University, Bilecik, T\u00fcrkiye"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Peter","family":"Krawitz","sequence":"additional","affiliation":[{"name":"Institute for Genomic Statistics and Bioinformatics, University Hospital Bonn, Bonn, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marius","family":"Ueffing","sequence":"additional","affiliation":[{"name":"Institute for Ophthalmic Research, Centre for Ophthalmology, University Hospital Tubingen, T\u00fcbingen, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hans","family":"Hoerauf","sequence":"additional","affiliation":[{"name":"Department of Ophthalmology, University Medical Center G\u00f6ttingen, G\u00f6ttingen, Germany"},{"name":"Else Kr\u00f6ner Fresenius Center for Optogenetic Therapies (EKFZ), University Medical Center G\u00f6ttingen, G\u00f6ttingen, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nicolas","family":"Feltgen","sequence":"additional","affiliation":[{"name":"Eye Hospital, University of Basel, Basel, Switzerland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Stylianos","family":"Michalakis","sequence":"additional","affiliation":[{"name":"Department of Ophthalmology, LMU University Hospital, LMU Munich, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-3365-6933","authenticated-orcid":false,"given":"Hildegard","family":"B\u00fcning","sequence":"additional","affiliation":[{"name":"Else Kr\u00f6ner Fresenius Center for Optogenetic Therapies (EKFZ), University Medical Center G\u00f6ttingen, G\u00f6ttingen, Germany"},{"name":"Laboratory for Experimental Gene and Cell Therapy, Institute of Experimental Hematology, Hannover Medical School, Hannover, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lukas","family":"Cyganek","sequence":"additional","affiliation":[{"name":"Stem Cell Unit, Clinic for Cardiology and Pneumology, University Medical Center G\u00f6ttingen, G\u00f6ttingen, Germany"},{"name":"Else Kr\u00f6ner Fresenius Center for Optogenetic Therapies (EKFZ), University Medical Center G\u00f6ttingen, G\u00f6ttingen, Germany"},{"name":"Translational Neuroinflammation and Automated Microscopy, Fraunhofer Institute for Translational Medicine and Pharmacology (ITMP), G\u00f6ttingen, Germany"},{"name":"Cluster of Excellence \u201cMultiscale Bioimaging: From Molecular Machines to Networks of Excitable Cells\u201d (MBExC), University of G\u00f6ttingen, G\u00f6ttingen, Germany."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Bernd","family":"Wollnik","sequence":"additional","affiliation":[{"name":"Institute of Human Genetics, University Medical Center G\u00f6ttingen, G\u00f6ttingen, Germany"},{"name":"German Center for Child and Adolescent Health (DZKJ), Partner Site G\u00f6ttingen, G\u00f6ttingen, Germany"},{"name":"Else Kr\u00f6ner Fresenius Center for Optogenetic Therapies (EKFZ), University Medical Center G\u00f6ttingen, G\u00f6ttingen, Germany"},{"name":"Cluster of Excellence \u201cMultiscale Bioimaging: From Molecular Machines to Networks of Excitable Cells\u201d (MBExC), University of G\u00f6ttingen, G\u00f6ttingen, Germany."}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"179","published-online":{"date-parts":[[2026,9,14]]},"reference":[{"key":"e_1_3_3_2_1","doi-asserted-by":"publisher","DOI":"10.3390\/jcm11236877"},{"key":"e_1_3_3_3_1","doi-asserted-by":"publisher","DOI":"10.1167\/iovs.12-11025"},{"key":"e_1_3_3_4_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ophtha.2022.11.015"},{"key":"e_1_3_3_5_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(06)69740-7"},{"key":"e_1_3_3_6_1","doi-asserted-by":"publisher","DOI":"10.1038\/nrg2717"},{"key":"e_1_3_3_7_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.stemcr.2020.05.007"},{"key":"e_1_3_3_8_1","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/ddv354"},{"key":"e_1_3_3_9_1","doi-asserted-by":"publisher","DOI":"10.1136\/bjo-2021-320897"},{"key":"e_1_3_3_10_1","doi-asserted-by":"publisher","DOI":"10.1038\/s42256-025-01040-8"},{"key":"e_1_3_3_11_1","doi-asserted-by":"publisher","DOI":"10.1186\/s12911-025-03119-z"},{"key":"e_1_3_3_12_1","unstructured":"11.Morelle O. EyeLab: Zenodo 2022."},{"key":"e_1_3_3_13_1","doi-asserted-by":"publisher","DOI":"10.1038\/s41598-023-35230-4"},{"key":"e_1_3_3_14_1","doi-asserted-by":"publisher","DOI":"10.1364\/BOE.8.003627"},{"key":"e_1_3_3_15_1","doi-asserted-by":"publisher","DOI":"10.1093\/nar\/gkv1222"},{"key":"e_1_3_3_16_1","doi-asserted-by":"publisher","DOI":"10.1038\/gim.2015.30"},{"key":"e_1_3_3_17_1","doi-asserted-by":"publisher","DOI":"10.1038\/s41586-021-03819-2"},{"key":"e_1_3_3_18_1","doi-asserted-by":"publisher","DOI":"10.1038\/s41588-025-02400-1"},{"key":"e_1_3_3_19_1","doi-asserted-by":"publisher","DOI":"10.1126\/science.adg7492"},{"key":"e_1_3_3_20_1","doi-asserted-by":"publisher","DOI":"10.1186\/s40246-023-00451-1"},{"key":"e_1_3_3_21_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.gene.2019.05.006"},{"key":"e_1_3_3_22_1","doi-asserted-by":"publisher","DOI":"10.1038\/nsmb.1396"},{"key":"e_1_3_3_23_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.str.2005.11.008"},{"key":"e_1_3_3_24_1","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0021379"},{"key":"e_1_3_3_25_1","doi-asserted-by":"publisher","DOI":"10.1186\/s13073-019-0690-2"},{"key":"e_1_3_3_26_1","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/ddu509"},{"key":"e_1_3_3_27_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.stem.2023.11.004"},{"key":"e_1_3_3_28_1","doi-asserted-by":"publisher","DOI":"10.1007\/s12015-025-10884-x"},{"key":"e_1_3_3_29_1","doi-asserted-by":"publisher","DOI":"10.1007\/s40265-025-02237-2"},{"key":"e_1_3_3_30_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ejpb.2015.01.009"},{"key":"e_1_3_3_31_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.pharmthera.2025.108862"},{"key":"e_1_3_3_32_1","doi-asserted-by":"publisher","DOI":"10.1074\/jbc.M116.760314"},{"key":"e_1_3_3_33_1","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/10.11.1177"},{"key":"e_1_3_3_34_1","doi-asserted-by":"publisher","DOI":"10.1038\/s41467-022-29490-3"},{"key":"e_1_3_3_35_1","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.2416827121"},{"key":"e_1_3_3_36_1","doi-asserted-by":"publisher","DOI":"10.1038\/s41591-024-03422-8"},{"key":"e_1_3_3_37_1","doi-asserted-by":"publisher","DOI":"10.1038\/s41551-020-00632-6"},{"key":"e_1_3_3_38_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.omtn.2024.102123"},{"key":"e_1_3_3_39_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(23)01952-9"},{"key":"e_1_3_3_40_1","doi-asserted-by":"publisher","DOI":"10.1038\/s41551-025-01461-1"},{"issue":"4","key":"e_1_3_3_41_1","first-page":"1467","article-title":"Emerging approaches for restoration of hearing and vision","volume":"100","author":"Kleinlogel S","year":"2020","unstructured":"40.Kleinlogel S, , Vogl C, , Jeschke M, et al. Emerging approaches for restoration of hearing and vision. Physiol Rev 2020;100(4):1467\u20131525. Available from: https:\/\/pubmed.ncbi.nlm.nih.gov\/32191560\/","journal-title":"Physiol Rev"},{"key":"e_1_3_3_42_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.omtm.2019.01.008"},{"key":"e_1_3_3_43_1","doi-asserted-by":"publisher","DOI":"10.15252\/emmm.202013392"},{"key":"e_1_3_3_44_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymthe.2025.03.064"},{"key":"e_1_3_3_45_1","doi-asserted-by":"publisher","DOI":"10.1038\/s41467-024-50033-5"},{"key":"e_1_3_3_46_1","doi-asserted-by":"publisher","DOI":"10.1007\/s00018-019-03283-2"},{"key":"e_1_3_3_47_1","doi-asserted-by":"publisher","DOI":"10.7554\/eLife.57245"},{"key":"e_1_3_3_48_1","doi-asserted-by":"publisher","DOI":"10.1161\/CIRCULATIONAHA.125.074155"},{"key":"e_1_3_3_49_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa2513525"},{"key":"e_1_3_3_50_1","doi-asserted-by":"publisher","DOI":"10.1038\/s41586-024-08463-0"}],"container-title":["Human Gene Therapy"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/journals.sagepub.com\/doi\/pdf\/10.1177\/10430342261480906","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/journals.sagepub.com\/doi\/full-xml\/10.1177\/10430342261480906","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/journals.sagepub.com\/doi\/pdf\/10.1177\/10430342261480906","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,9,17]],"date-time":"2026-09-17T18:16:44Z","timestamp":1789669004000},"score":1,"resource":{"primary":{"URL":"https:\/\/journals.sagepub.com\/doi\/10.1177\/10430342261480906"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2026,9,14]]},"references-count":49,"alternative-id":["10.1177\/10430342261480906"],"URL":"https:\/\/doi.org\/10.1177\/10430342261480906","relation":{},"ISSN":["1043-0342","1557-7422"],"issn-type":[{"value":"1043-0342","type":"print"},{"value":"1557-7422","type":"electronic"}],"subject":[],"published":{"date-parts":[[2026,9,14]]},"article-number":"10430342261480906"}}