한빛사논문
- 조회337
Elliot H. Choi1, Susie Suh1, Avery E. Sears1, Rafał Hołubowicz1, Sanjay R. Kedhar1, Andrew W. Browne1 and Krzysztof Palczewski1,2,3,4,*
1Gavin Herbert Eye Institute, Department of Ophthalmology, University of California, Irvine, CA, USA.
2Department of Physiology and Biophysics, University of California, Irvine, CA, USA.
3Department of Chemistry, University of California, Irvine, CA, USA.
4Department of Molecular Biology and Biochemistry, University of California, Irvine, CA, USA.
*Corresponding author: correspondence to 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.
논문정보
- Review Paper
- 2023년 08월 (BRIC 등록일 2023-08-07)
- 국외 연구진
- 의약학 > 유전 및 유전체의학
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