• Xi Chen, Yang Yu, Huiling Nie, Xun Qin, Wen Bai, Junsong Ren, Jin Yao, Juxue Li, and Qin Jiang.
• Affiliated Eye Hospital, Nanjing Medical University, Nanjing, Jiangsu, China.
• Medicine (Baltimore). 2023 Jun 16; 102 (24): e34043e34043.

BackgroundAdeno-associated virus (AAV) plays a vital role in ocular gene therapy and has been widely studied since 1996. This study summarizes and explores the publication outputs and future research trends of AAV-based ocular gene therapy.MethodsPublications and data about AAV-based ocular gene therapy were downloaded from the Web of Science Core Collection or ClinicalTrials.gov database. The publications and data were analyzed by Microsoft Excel, CiteSpace, VOS viewer, and a free online platform (http://bibliometric.com).ResultsTotally 832 publications from the Web of Science Core Collection relevant to AAV-based ocular gene therapy were published from 1996 to 2022. These publications were contributed by research institutes from 42 countries or regions. The US contributed the most publications among these countries or regions, notably the University of Florida. Hauswirth WW was the most productive author. "Efficacy" and "safety" are the main focus areas for future research according to the references and keywords analysis. Eighty clinical trials examined AAV-based ocular gene therapy were registered on ClinicalTrials.Gov. Institutes from the US and European did the dominant number or the large proportion of the trials.ConclusionsThe research focus of the AAV-based ocular gene therapy has transitioned from the study in biological theory to clinical trialing. The AAV-based gene therapy is not limited to inherited retinal diseases but various ocular diseases.Copyright © 2023 the Author(s). Published by Wolters Kluwer Health, Inc.

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