Literature DB >> 30205877

CRISPR/Cas9 genome surgery for retinal diseases.

Christine L Xu1, Karen Sophia Park1, Stephen H Tsang2.   

Abstract

Retinal diseases that impair vision can impose heavy physical and emotional burdens on patients' lives. Currently, clustered regularly interspaced short palindromic repeats (CRISPR) is a prevalent gene-editing tool that can be harnessed to generate disease model organisms for specific retinal diseases, which are useful for elucidating pathophysiology and revealing important links between genetic mutations and phenotypic defects. These retinal disease models are fundamental for testing various therapies and are indispensible for potential future clinical trials. CRISPR-mediated procedures involving CRISPR-associated protein 9 (Cas9) may also be used to edit genome sequences and correct mutations. Thus, if used for future therapies, CRISPR/Cas9 genome surgery could eliminate the need for patients with retinal diseases to undergo repetitive procedures such as drug injections. In this review, we will provide an overview of CRISPR/Cas9, discuss the different types of Cas9, and compare Cas9 to other endonucleases. Furthermore, we will explore the many ways in which researchers are currently utilizing this versatile tool, as CRISPR/Cas9 may have far-reaching effects in the treatment of retinal diseases.
Copyright © 2018 Elsevier Ltd. All rights reserved.

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Year:  2018        PMID: 30205877      PMCID: PMC6136254          DOI: 10.1016/j.ddtec.2018.05.001

Source DB:  PubMed          Journal:  Drug Discov Today Technol        ISSN: 1740-6749


  75 in total

1.  Double nicking by RNA-guided CRISPR Cas9 for enhanced genome editing specificity.

Authors:  F Ann Ran; Patrick D Hsu; Chie-Yu Lin; Jonathan S Gootenberg; Silvana Konermann; Alexandro E Trevino; David A Scott; Azusa Inoue; Shogo Matoba; Yi Zhang; Feng Zhang
Journal:  Cell       Date:  2013-08-29       Impact factor: 41.582

Review 2.  Leber congenital amaurosis.

Authors:  I Perrault; J M Rozet; S Gerber; I Ghazi; C Leowski; D Ducroq; E Souied; J L Dufier; A Munnich; J Kaplan
Journal:  Mol Genet Metab       Date:  1999-10       Impact factor: 4.797

3.  Gene Augmentation Therapy Restores Retinal Function and Visual Behavior in a Sheep Model of CNGA3 Achromatopsia.

Authors:  Eyal Banin; Elisha Gootwine; Alexey Obolensky; Raaya Ezra-Elia; Ayala Ejzenberg; Lina Zelinger; Hen Honig; Alexander Rosov; Esther Yamin; Dror Sharon; Edward Averbukh; William W Hauswirth; Ron Ofri
Journal:  Mol Ther       Date:  2015-06-19       Impact factor: 11.454

4.  Effect of genome size on AAV vector packaging.

Authors:  Zhijian Wu; Hongyan Yang; Peter Colosi
Journal:  Mol Ther       Date:  2009-11-10       Impact factor: 11.454

Review 5.  ZFN, TALEN, and CRISPR/Cas-based methods for genome engineering.

Authors:  Thomas Gaj; Charles A Gersbach; Carlos F Barbas
Journal:  Trends Biotechnol       Date:  2013-05-09       Impact factor: 19.536

Review 6.  New and emerging technologies for the treatment of inherited retinal diseases: a horizon scanning review.

Authors:  J Smith; D Ward; M Michaelides; A T Moore; S Simpson
Journal:  Eye (Lond)       Date:  2015-06-26       Impact factor: 3.775

7.  TALEN and CRISPR/Cas Genome Editing Systems: Tools of Discovery.

Authors:  A A Nemudryi; K R Valetdinova; S P Medvedev; S M Zakian
Journal:  Acta Naturae       Date:  2014-07       Impact factor: 1.845

8.  Single-cell analysis of long non-coding RNAs in the developing human neocortex.

Authors:  Siyuan John Liu; Tomasz J Nowakowski; Alex A Pollen; Jan H Lui; Max A Horlbeck; Frank J Attenello; Daniel He; Jonathan S Weissman; Arnold R Kriegstein; Aaron A Diaz; Daniel A Lim
Journal:  Genome Biol       Date:  2016-04-14       Impact factor: 13.583

9.  Editing VEGFR2 Blocks VEGF-Induced Activation of Akt and Tube Formation.

Authors:  Xionggao Huang; Guohong Zhou; Wenyi Wu; Gaoen Ma; Patricia A D'Amore; Shizuo Mukai; Hetian Lei
Journal:  Invest Ophthalmol Vis Sci       Date:  2017-02-01       Impact factor: 4.799

10.  In Vivo CRISPR/Cas9 Gene Editing Corrects Retinal Dystrophy in the S334ter-3 Rat Model of Autosomal Dominant Retinitis Pigmentosa.

Authors:  Benjamin Bakondi; Wenjian Lv; Bin Lu; Melissa K Jones; Yuchun Tsai; Kevin J Kim; Rachelle Levy; Aslam Abbasi Akhtar; Joshua J Breunig; Clive N Svendsen; Shaomei Wang
Journal:  Mol Ther       Date:  2015-12-15       Impact factor: 11.454

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  5 in total

Review 1.  Viral Delivery Systems for CRISPR.

Authors:  Christine L Xu; Merry Z C Ruan; Vinit B Mahajan; Stephen H Tsang
Journal:  Viruses       Date:  2019-01-04       Impact factor: 5.048

2.  Targeted Krüppel-Like Factor 4 Gene Knock-Out in Retinal Ganglion Cells Improves Visual Function in Multiple Sclerosis Mouse Model.

Authors:  Venu Talla; Rajeshwari Koilkonda
Journal:  eNeuro       Date:  2020-04-02

Review 3.  Microphysiological Neurovascular Barriers to Model the Inner Retinal Microvasculature.

Authors:  Thomas L Maurissen; Georgios Pavlou; Colette Bichsel; Roberto Villaseñor; Roger D Kamm; Héloïse Ragelle
Journal:  J Pers Med       Date:  2022-01-24

Review 4.  CRISPR/Cas9 gene editing: New hope for Alzheimer's disease therapeutics.

Authors:  Shanu Bhardwaj; Kavindra Kumar Kesari; Mahesh Rachamalla; Shalini Mani; Ghulam Md Ashraf; Saurabh Kumar Jha; Pravir Kumar; Rashmi K Ambasta; Harish Dureja; Hari Prasad Devkota; Gaurav Gupta; Dinesh Kumar Chellappan; Sachin Kumar Singh; Kamal Dua; Janne Ruokolainen; Mohammad Amjad Kamal; Shreesh Ojha; Niraj Kumar Jha
Journal:  J Adv Res       Date:  2021-07-06       Impact factor: 12.822

Review 5.  CRISPR/Cas9-A Promising Therapeutic Tool to Cure Blindness: Current Scenario and Future Prospects.

Authors:  Irshad Ahmad
Journal:  Int J Mol Sci       Date:  2022-09-29       Impact factor: 6.208

  5 in total

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