Eye on genome editing

Du, Samuel W.; Palczewski, Krzysztof

doi:10.1084/jem.20230146

Journal of Experimental Medicine

2023

DOI: 10.1084/jem.20230146

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Eye on genome editing

Samuel W. Du

Krzysztof Palczewski

Abstract: CRISPR/Cas9 genome editing techniques have the potential to treat previously untreatable inherited genetic disorders of vision by correcting mutations that cause these afflictions. Using a prime editor, Qin et al. (2023. J. Exp. Med.https://doi.org/10.1084/jem.20220776) restored visual functions in a mouse model (rd10) of retinitis pigmentosa.

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Genome editing in the treatment of ocular diseases

Choi,

Suh,

Sears

et al. 2023

Exp Mol Med

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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.

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Genome editing in the treatment of ocular diseases

Choi,

Suh,

Sears

et al. 2023

Exp Mol Med

Self Cite

View full text Add to dashboard Cite

show abstract

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Eye on genome editing

Cited by 1 publication

References 18 publications

Genome editing in the treatment of ocular diseases

Genome editing in the treatment of ocular diseases

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