Using CRISPR Interference as a Therapeutic Approach to Treat TGFβ2-Induced Ocular Hypertension and Glaucoma

Rayana, Naga Pradeep; Sugali, Chenna Kesavulu; Dai, Jiannong; Peng, Michael; Liu, Shaohui; Zhang, Yucheng; Wan, Jun; Mao, Weiming

doi:10.1167/iovs.62.12.7

Cited by 13 publications

(16 citation statements)

References 31 publications

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“…[122,123] The unexplained hyperacetylation status of transforming growth factor beta-2 (TGF-𝛽2) is one of the important cause of primary open-angle glaucoma (POAG). [124] Rayana et al successfully suppressed TGF-𝛽2 by deacetylating the promoter region of the TGF-𝛽2 gene, [125] Copyright 2021, Association for Research in Vision and Ophthalmology. B) Noggin knockdown therapy for bone fracture.…”

Section: Krüppel-associated Box (Krab) Inhibitionmentioning

confidence: 99%

Section: Preclinical Research Of Crispr‐cas9mentioning

confidence: 99%

mentioning

confidence: 99%

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Progress and Perspective of CRISPR‐Cas9 Technology in Translational Medicine

et al. 2023

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Translational medicine aims to improve human health by exploring potential treatment methods developed during basic scientific research and applying them to the treatment of patients in clinical settings. The advanced perceptions of gene functions have remarkably revolutionized clinical treatment strategies for target agents. However, the progress in gene editing therapy has been hindered due to the severe off‐target effects and limited editing sites. Fortunately, the development in the clustered regularly interspaced short palindromic repeats associated protein 9 (CRISPR‐Cas9) system has renewed hope for gene therapy field. The CRISPR‐Cas9 system can fulfill various simple or complex purposes, including gene knockout, knock‐in, activation, interference, base editing, and sequence detection. Accordingly, the CRISPR‐Cas9 system is adaptable to translational medicine, which calls for the alteration of genomic sequences. This review aims to present the latest CRISPR‐Cas9 technology achievements and prospect to translational medicine advances. The principle and characterization of the CRISPR‐Cas9 system are firstly introduced. The authors then focus on recent pre‐clinical and clinical research directions, including the construction of disease models, disease‐related gene screening and regulation, and disease treatment and diagnosis for multiple refractory diseases. Finally, some clinical challenges including off‐target effects, in vivo vectors, and ethical problems, and future perspective are also discussed.

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Section: Krüppel-associated Box (Krab) Inhibitionmentioning

confidence: 99%

Section: Preclinical Research Of Crispr‐cas9mentioning

confidence: 99%

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Progress and Perspective of CRISPR‐Cas9 Technology in Translational Medicine

et al. 2023

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“…Rayana et al . [51] reported histone hyperacetylation is associated with transforming growth factor beta 2 ( TGFβ2 ) elevation in the TM. Elevated TGFβ2 is associated with pathological changes in glaucomatous TM.…”

Section: Current Applications Of Crispr/cas9 In Understanding and Tre...mentioning

confidence: 99%

Therapeutic applications of CRISPR/Cas9 gene editing technology for the treatment of ocular diseases

et al. 2023

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Ocular diseases are a highly heterogeneous group of phenotypes, caused by a spectrum of genetic variants and environmental factors that exhibit diverse clinical symptoms. As a result of its anatomical location, structure and immune privilege, the eye is an ideal system to assess and validate novel genetic therapies. Advances in genome editing have revolutionized the field of biomedical science, enabling researchers to understand the biology behind disease mechanisms and allow the treatment of several health conditions, including ocular pathologies. The advent of clustered regularly interspaced short palindromic repeats (CRISPR)‐based gene editing facilitates efficient and specific genetic modifications in the nucleic acid sequence, resulting in permanent changes at the genomic level. This approach has advantages over other treatment strategies and is promising for the treatment of various genetic and non‐genetic ocular conditions. This review provides an overview of the CRISPR/CRISPR‐associated protein 9 (Cas9) system and summarizes recent advances in the therapeutic application of CRISPR/Cas9 for the treatment of various ocular pathologies, as well as future challenges.

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“…The reduction in OHT was linked with a diminished level of TGFβ2 and extracellular matrix proteins in the mouse eye. These findings suggest that CRISPR interference can be used as a tool for gene inhibition containing the therapeutic potential for the treatment of TGFβ2-induced OHT [ 77 ].…”

Section: Crispr/cas9 Is a Promising Strategy To Restore The Blindnessmentioning

confidence: 99%

CRISPR/Cas9—A Promising Therapeutic Tool to Cure Blindness: Current Scenario and Future Prospects

Ahmad

2022

IJMS

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CRISPR-based targeted genome editing is bringing revolutionary changes in the research arena of biological sciences. CRISPR/Cas9 has been explored as an efficient therapeutic tool for the treatment of genetic diseases. It has been widely used in ophthalmology research by using mouse models to correct pathogenic mutations in the eye stem cells. In recent studies, CRISPR/Cas9 has been used to correct a large number of mutations related to inherited retinal disorders. In vivo therapeutic advantages for retinal diseases have been successfully achieved in some rodents. Current advances in the CRISPR-based gene-editing domain, such as modified Cas variants and delivery approaches have optimized its application to treat blindness. In this review, recent progress and challenges of the CRISPR-Cas system have been discussed to cure blindness and its prospects.

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Using CRISPR Interference as a Therapeutic Approach to Treat TGFβ2-Induced Ocular Hypertension and Glaucoma

Cited by 13 publications

References 31 publications

Progress and Perspective of CRISPR‐Cas9 Technology in Translational Medicine

Progress and Perspective of CRISPR‐Cas9 Technology in Translational Medicine

Therapeutic applications of CRISPR/Cas9 gene editing technology for the treatment of ocular diseases

CRISPR/Cas9—A Promising Therapeutic Tool to Cure Blindness: Current Scenario and Future Prospects

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