Owing to the promising therapeutic effect and one-time treatment advantage, gene therapy may completely change the management of eye diseases, especially retinal diseases. Adeno-associated virus (AAV) is considered on...Owing to the promising therapeutic effect and one-time treatment advantage, gene therapy may completely change the management of eye diseases, especially retinal diseases. Adeno-associated virus (AAV) is considered one of the most promising viral gene delivery tools because it can infect various types of tissues and is considered as a relatively safe gene delivery vector. The eye is one of the most popular organs for gene therapy, since its limited volume is suitable for small doses of AAV stably transduction. Recently, an increasing number of clinical trials of AAV-mediated gene therapy are underway. This review summarizes the biological functions of AAV and its application in the treatment of various ocular diseases, as well as the characteristics of different AAV delivery routes in clinical applications. Here, the latest research progresses in AAV-mediated gene editing and silencing strategies to modify that the genetic ocular diseases are systematically outlined, especially by base editing and prime editing. We discuss the progress of AAV in ocular optogenetic therapy. We also summarize the application of AAV-mediated gene therapy in animal models and the difficulties in its clinical transformation.展开更多
Artificial intelligence(AI)is an emerging field in which computerized systems are used to carry out complex tasks in place of humans.Medical AI algorithms have been developed for disease diagnosis and prediction and t...Artificial intelligence(AI)is an emerging field in which computerized systems are used to carry out complex tasks in place of humans.Medical AI algorithms have been developed for disease diagnosis and prediction and treatment recommendation across various clinical data types,e.g.,chest X-rays,electrocardiograms,and other radiological images.1 In ophthalmology,particularly,great progress has been made in AI systems over the past decade.Color fundus photography(CFP)and optical coherence tomography(OCT),which are readily available in routine clinical practice,are both mainstream and useful retinal imaging modalities in ophthalmology.In September 2023,the 2023 Lasker-Debakey Clinical Medical Research Award was awarded to three scientists for their work on OCT for accurate retinal disease detection.展开更多
基金the National Natural Science Foundation of China(82200961)the National Basic Science Center Program of China(82388101)+2 种基金the Science and Technology Commission of Shanghai(20DZ2270800)Shanghai Key Laboratory of Orbital Diseases and Ocular Oncology(2022SKLEKFKT004)the China Postdoctoral Science Foundation(2022M720091).
文摘Owing to the promising therapeutic effect and one-time treatment advantage, gene therapy may completely change the management of eye diseases, especially retinal diseases. Adeno-associated virus (AAV) is considered one of the most promising viral gene delivery tools because it can infect various types of tissues and is considered as a relatively safe gene delivery vector. The eye is one of the most popular organs for gene therapy, since its limited volume is suitable for small doses of AAV stably transduction. Recently, an increasing number of clinical trials of AAV-mediated gene therapy are underway. This review summarizes the biological functions of AAV and its application in the treatment of various ocular diseases, as well as the characteristics of different AAV delivery routes in clinical applications. Here, the latest research progresses in AAV-mediated gene editing and silencing strategies to modify that the genetic ocular diseases are systematically outlined, especially by base editing and prime editing. We discuss the progress of AAV in ocular optogenetic therapy. We also summarize the application of AAV-mediated gene therapy in animal models and the difficulties in its clinical transformation.
基金This work was supported by the National Natural Science Foundation of China(82200961)the Science and Technology Commission of Shanghai(20DZ2270800)+1 种基金the Shanghai Key Laboratory of Orbital Diseases and Ocular Oncology(2022SKLE-KFKT004)the China Postdoctoral Science Foundation(2022M720091,2023M741708,2023TQ0159,and GZC20233503)。
文摘Artificial intelligence(AI)is an emerging field in which computerized systems are used to carry out complex tasks in place of humans.Medical AI algorithms have been developed for disease diagnosis and prediction and treatment recommendation across various clinical data types,e.g.,chest X-rays,electrocardiograms,and other radiological images.1 In ophthalmology,particularly,great progress has been made in AI systems over the past decade.Color fundus photography(CFP)and optical coherence tomography(OCT),which are readily available in routine clinical practice,are both mainstream and useful retinal imaging modalities in ophthalmology.In September 2023,the 2023 Lasker-Debakey Clinical Medical Research Award was awarded to three scientists for their work on OCT for accurate retinal disease detection.
基金supported by the National Basic Science Center Program of China(82388101)the National Natural Science Foundation of China(82200961 and 82203260)+2 种基金the Science and Technology Commission of Shanghai(20DZ2270800)Shanghai Key Laboratory of Orbital Diseases and Ocular Oncology(2022SKLEKFKT004)China Postdoctoral Science Foundation(2022M720091)。
