{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,6,5]],"date-time":"2026-06-05T01:08:02Z","timestamp":1780621682689,"version":"3.54.1"},"reference-count":42,"publisher":"SAGE Publications","issue":"10","license":[{"start":{"date-parts":[[2024,12,1]],"date-time":"2024-12-01T00:00:00Z","timestamp":1733011200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/journals.sagepub.com\/page\/policies\/text-and-data-mining-license"}],"content-domain":{"domain":["journals.sagepub.com"],"crossmark-restriction":true},"short-container-title":["Journal of Ocular Pharmacology and Therapeutics"],"published-print":{"date-parts":[[2024,12,1]]},"abstract":"<jats:sec>\n                    <jats:title>Purpose:<\/jats:title>\n                    <jats:p>\n                      Leber congenital amaurosis (LCA) is a sight-threatening inherited retinal disorder (IRD) caused by numerous genetic mutations. Multi-characteristic opsin (MCO)-based optogenetic therapy allows the recruitment of residual cells of the retina in LCA for alternative vision transduction while being mutation-agnostic. Using\n                      <jats:italic toggle=\"yes\">rd12<\/jats:italic>\n                      mice, we investigated the\n                      <jats:italic toggle=\"yes\">in\u00a0vivo<\/jats:italic>\n                      efficacy of an adeno-associated virus2 (AAV2)-transduced ambient light-activatable MCO (MCO-010) containing a metabotropic glutamate receptor-6 bipolar cell-specific promoter\/enhancer.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods:<\/jats:title>\n                    <jats:p>Mice requiring &gt; 40 s to reach and board a dimly lit hidden platform in a water-maze were selected and randomly divided into 2 cohorts. These mice were intravitreally (IVT) injected with either 1.7E9 gene copies\/eye of MCO-010 or control AAV2 and re-tested in the water-maze. Spectral-domain optical coherence tomography (SD-OCT), hematoxylin and eosin staining of retinas, and electroretinographic (ERG) studies were also conducted.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results:<\/jats:title>\n                    <jats:p>\n                      Safety of MCO-010 in\n                      <jats:italic toggle=\"yes\">rd12<\/jats:italic>\n                      mice was confirmed by the lack of significant detrimental changes in the mouse behavior, b-wave amplitudes and in retinal thickness.\n                      <jats:italic toggle=\"yes\">rd12<\/jats:italic>\n                      control mice performed relatively poorly in the water-maze test requiring \u2265 30\u201360 s to find and board the platform. MCO-010-treated\n                      <jats:italic toggle=\"yes\">rd12<\/jats:italic>\n                      mice reached the platform much faster than the AAV2-treated\n                      <jats:italic toggle=\"yes\">rd12<\/jats:italic>\n                      mice, with some mice only requiring &lt; 5 s to achieve this goal (\n                      <jats:italic toggle=\"yes\">P<\/jats:italic>\n                      &lt; 0.01\u20130.0024).\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusions:<\/jats:title>\n                    <jats:p>\n                      IVT MCO-010 treatment was well tolerated by\n                      <jats:italic toggle=\"yes\">rd12<\/jats:italic>\n                      mice, and it prevented the decrease in retinal thickness, and preserved ERG parameters. It also significantly improved the vision in\n                      <jats:italic toggle=\"yes\">rd12<\/jats:italic>\n                      mice relative to control AAV2-injected mice. MCO-010 therefore represents a novel and efficacious optogenetic therapeutic to treat LCA and other IRDs irrespective of the genetic defect(s).\n                    <\/jats:p>\n                  <\/jats:sec>","DOI":"10.1089\/jop.2024.0084","type":"journal-article","created":{"date-parts":[[2024,10,23]],"date-time":"2024-10-23T11:27:44Z","timestamp":1729682864000},"page":"702-708","update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":6,"title":["Efficacy of Intravitreal Multi-Characteristic Opsin (MCO-010) Optogenetic Gene Therapy in a Mouse Model of Leber Congenital Amaurosis"],"prefix":"10.1177","volume":"40","author":[{"given":"Adnan","family":"Dibas","sequence":"first","affiliation":[{"name":"Nanoscope Technologies LLC, Bedford, Texas, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-0859-6641","authenticated-orcid":false,"given":"Subrata","family":"Batabyal","sequence":"additional","affiliation":[{"name":"Nanoscope Technologies LLC, Bedford, Texas, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sanghoon","family":"Kim","sequence":"additional","affiliation":[{"name":"Nanoscope Technologies LLC, Bedford, Texas, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michael","family":"Carlson","sequence":"additional","affiliation":[{"name":"Nanoscope Technologies LLC, Bedford, Texas, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Samarendra","family":"Mohanty","sequence":"additional","affiliation":[{"name":"Nanoscope Technologies LLC, Bedford, Texas, USA."},{"name":"Nanoscope Therapeutics Inc, Dallas, Texas, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Najam A.","family":"Sharif","sequence":"additional","affiliation":[{"name":"Nanoscope Therapeutics Inc, Dallas, Texas, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"179","published-online":{"date-parts":[[2024,12,2]]},"reference":[{"key":"e_1_3_4_2_2","doi-asserted-by":"publisher","DOI":"10.4103\/1673-5374.280303"},{"key":"e_1_3_4_3_2","doi-asserted-by":"publisher","DOI":"10.21037\/atm-20-4726"},{"key":"e_1_3_4_4_2","doi-asserted-by":"publisher","DOI":"10.3390\/biom13020271"},{"key":"e_1_3_4_5_2","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.1218933110"},{"key":"e_1_3_4_6_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.exer.2019.107834"},{"key":"e_1_3_4_7_2","doi-asserted-by":"publisher","DOI":"10.1007\/978-3-319-95046-4_26"},{"key":"e_1_3_4_8_2","doi-asserted-by":"publisher","DOI":"10.1002\/cne.21144"},{"issue":"1","key":"e_1_3_4_9_2","first-page":"143","article-title":"Morphometric analysis of the extramacular retina from postmortem eyes with retinitis pigmentosa","volume":"40","author":"Humayun MS","year":"1999","unstructured":"Humayun MS, , Prince M, , de Juan EJr, et al. 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