{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,17]],"date-time":"2026-10-17T02:03:12Z","timestamp":1792202592566,"version":"4.4.0"},"reference-count":98,"publisher":"MDPI AG","issue":"9","license":[{"start":{"date-parts":[[2025,5,6]],"date-time":"2025-05-06T00:00:00Z","timestamp":1746489600000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["JCM"],"abstract":"<jats:p>Diabetic retinopathy (DR) is one of the leading causes of preventable blindness worldwide. It is characterized by a spectrum of disease that spans mild non-proliferative diabetic retinopathy (NPDR) all the way to neovascular glaucoma and tractional retinal detachment secondary to proliferative diabetic retinopathy (PDR). Most eyes with DR remain asymptomatic unless vision-threatening complications, such as diabetic macular edema (DME) and\/or PDR, develop. Current treatment options include laser photocoagulation and\/or anti-VEGF intravitreal injections. Patients under treatment with anti-VEGF agents usually require constant monitoring and multiple injections to optimize outcomes. This treatment burden plays a key role in suboptimal adherence to treatment in many patients, compromising their outcomes. Gene therapy has emerged as a promising therapeutic option for DR. The mechanism for current trials evaluating gene therapies for DR consists of delivering transgenes to the retina that express anti-angiogenic proteins that inhibit VEGF. Preliminary results from the SPECTRA (4D-150) and ALTITUDE (ABBV-RGX-314) studies are promising, demonstrating an improvement in the diabetic retinopathy severity score and a reduction in the treatment burden. In contrast, the INFINITY (ADVM-022) trial was complicated by several cases of severe inflammation and hypotony that led the sponsor to discontinue further development of this product for DME.<\/jats:p>","DOI":"10.3390\/jcm14093205","type":"journal-article","created":{"date-parts":[[2025,5,6]],"date-time":"2025-05-06T05:54:43Z","timestamp":1746510883000},"page":"3205","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":22,"title":["Gene Therapy in Diabetic Retinopathy and Diabetic Macular Edema: An Update"],"prefix":"10.3390","volume":"14","author":[{"given":"Maricruz","family":"Odio-Herrera","sequence":"first","affiliation":[{"name":"Asociados de M\u00e1cula Vitreo y Retina de Costa Rica, San Jos\u00e9 60612, Costa Rica"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0008-6936-532X","authenticated-orcid":false,"given":"Gloriana","family":"Orozco-Loaiza","sequence":"additional","affiliation":[{"name":"Asociados de M\u00e1cula Vitreo y Retina de Costa Rica, San Jos\u00e9 60612, Costa Rica"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-3387-7058","authenticated-orcid":false,"given":"Lihteh","family":"Wu","sequence":"additional","affiliation":[{"name":"Asociados de M\u00e1cula Vitreo y Retina de Costa Rica, San Jos\u00e9 60612, Costa Rica"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1968","published-online":{"date-parts":[[2025,5,6]]},"reference":[{"key":"ref_1","unstructured":"IDF (2025, April 19). 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