Genome editing in the treatment of ocular diseases
Emran M. Abu-Taleb, 서수지, Avery E. Sears, Rafał Hołubowicz, Sanjay R. Kedhar, Andrew W. Browne, Krzysztof Palczewski,
소속 상세정보
( Emran M. Abu-Taleb )
서수지 ( Suh Su-Sie )
( Avery E. Sears )
( Rafał Hołubowicz )
( Sanjay R. Kedhar )
( Andrew W. Browne )
( Krzysztof Palczewski )
Abstract
Genome-editing technologies have ushered in a new era in gene therapy, providing novel therapeutic strategies for a wide range of diseases, including both genetic and nongenetic ocular diseases. These technologies offer new hope for patients suffering from previously untreatable conditions. The unique anatomical and physiological features of the eye, including its immune-privileged status, size, and compartmentalized structure, provide an optimal environment for the application of these cutting-edge technologies. Moreover, the development of various delivery methods has facilitated the efficient and targeted administration of genome engineering tools designed to correct specific ocular tissues. Additionally, advancements in noninvasive ocular imaging techniques and electroretinography have enabled real-time monitoring of therapeutic efficacy and safety. Herein, we discuss the discovery and development of genome-editing technologies, their application to ocular diseases from the anterior segment to the posterior segment, current limitations encountered in translating these technologies into clinical practice, and ongoing research endeavors aimed at overcoming these challenges.
키워드
Genetic engineering; Targeted gene repair
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