CoChat
Sign Up
Cited by 14

The DOI matches its Crossref record; 6 of the 6 references it cites were found in a registry or an index.

CRISPR/Cas9 genome surgery for retinal diseases

Xu, Christine L., Park, Karen Sophia, Tsang, Stephen H.·2018·Drug Discovery Today: Technologies
DOI 10.1016/j.ddtec.2018.05.001PMC PMC6136254
Open this paper in CoChat — free account

Overview

The claims and statistics of this paper haven’t been checked yet.

Run the check — free, no account needed. It reads the open-access text and checks the sources it cites.

A few free checks a day · papers already verified open instantly · results are public

Integrity signals

Registry

Crossref · 10.1016/j.ddtec.2018.05.001

Cites

6 of 6 references verified0 citation errors · 0 not checkable

Statistics

Verification

Claims the paper makes

The claims and statistics of this paper haven’t been checked yet.

Run the check — free, no account needed. It reads the open-access text and checks the sources it cites.

A few free checks a day · papers already verified open instantly · results are public

Claims other papers make about this one

Each is checked against this paper’s own text: does it say what the citing paper says it does?

Self-check

Percentages, sums and reported differences re-computed from the paper’s own numbers.

The self-check needs the paper’s full text.

Citations

Cites

Show all 6 references

Research works printed but not linked: 72

  1. Sengillo, 2016, Gene and cell-based therapies for inherited retinal disorders: an update, Am J Med Genet C Semin Med Genet, 366, 349, 10.1002/ajmg.c.31534 · DOI
  2. Sengillo, 2017, Correction of monogenic and common retinal disorders with gene therapy, Genes (Basel), 8, 10.3390/genes8020053 · DOI
  3. Smith, 2015, New and emerging technologies for the treatment of inherited retinal diseases: a horizon scanning review, Eye (Lond), 29, 1131, 10.1038/eye.2015.115 · DOI
  4. Hung, 2016, Genome engineering in ophthalmology: application of CRISPR/Cas to the treatment of eye disease, Prog Retin Eye Res, 53, 1, 10.1016/j.preteyeres.2016.05.001 · DOI
  5. Informed Health Online [Internet]. Cologne, Germany: Institute for Quality and Efficiency in Health Care (IQWiG); 2006-. How does the eye work? 2009 May 28 [Updated 2015 Jan 7]. Available from: https://www.ncbi.nlm.nih.gov/books/NBK279248/.
  6. Arshavsky, 2002, G proteins and phototransduction, Annu Rev Physiol, 64, 153, 10.1146/annurev.physiol.64.082701.102229 · DOI
  7. Gregg, 2012, Function and anatomy of the mammalian retina, vol. 1, 360
  8. Banin, 2015, Gene augmentation therapy restores retinal function and visual behavior in a sheep model of CNGA3 achromatopsia, Mol Ther, 23, 1423, 10.1038/mt.2015.114 · DOI · Paper page
  9. Chan, 2013, Advances in the genetics of eye diseases, Curr Opin Pediatr, 645, 10.1097/MOP.0000000000000026 · DOI
  10. Russell, 2017, Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65 — mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial, Lancet, 390, 849, 10.1016/S0140-6736(17)31868-8 · DOI · Paper page
  11. Maguire, 2008, Safety and efficacy of gene transfer for Leber’s congenital amaurosis, N Engl J Med, 358, 2240, 10.1056/NEJMoa0802315 · DOI · Paper page
  12. Ziviello, 2005, Molecular genetics of autosomal dominant retinitis pigmentosa (ADRP): a comprehensive study of 43 Italian families, J Med Genet, 42, 10.1136/jmg.2005.031682 · DOI
  13. Kermani, 1999, Refined genetic and physical positioning of the gene for Doyne honeycomb retinal dystrophy (DHRD), Hum Genet, 104, 77, 10.1007/s004390050913 · DOI
  14. Perrault, 1999, Leber congenital amaurosis, Mol Genet Metab, 68, 200, 10.1006/mgme.1999.2906 · DOI
  15. Parmeggiani, 2011, Retinitis pigmentosa: genes and disease mechanisms, Curr Genomics, 12, 238, 10.2174/138920211795860107 · DOI
  16. Wu, 2017, AAV-CRISPR/Cas9-mediated depletion of VEGFR2 blocks angiogenesis in vitro, Invest Ophthalmol Vis Sci, 58, 6082, 10.1167/iovs.17-21902 · DOI
  17. Mojica, 1993, Transcription at different salinities of Haloferax mediterranei sequences adjacent to partially modified PstI sites, Mol Microbiol, 9, 613, 10.1111/j.1365-2958.1993.tb01721.x · DOI · Paper page
  18. Lander, 2016, The heroes of CRISPR, Cell, 164, 18, 10.1016/j.cell.2015.12.041 · DOI
  19. Toro, 2014, Association of clustered regularly interspaced short palindromic repeat (CRISPR) elements with specific serotypes and virulence potential of Shiga toxin-producing Escherichia coli, Appl Environ Microbiol, 80, 1411, 10.1128/AEM.03018-13 · DOI
  20. Ostria-Hernández, 2015, Survey of clustered regularly interspaced short palindromic repeats and their associated Cas proteins (CRISPR/Cas) systems in multiple sequenced strains of Klebsiella pneumoniae, BMC Res Notes, 8, 332, 10.1186/s13104-015-1285-7 · DOI
  21. Makarova, 2011, Evolution and classification of the CRISPR–Cas systems, Nat Rev Microbiol, 9, 467, 10.1038/nrmicro2577 · DOI · Paper page
  22. Zetsche, 2015, Cpf1 is a single RNA-guided endonuclease of a class 2 CRISPR-Cas system, Cell, 163, 759, 10.1016/j.cell.2015.09.038 · DOI · Paper page
  23. Mojica, 2005, Intervening sequences of regularly spaced prokaryotic repeats derive from foreign genetic elements, J Mol Evol, 60, 174, 10.1007/s00239-004-0046-3 · DOI · Paper page
  24. Bolotin, 2005, Clustered regularly interspaced short palindrome repeats (CRISPRs) have spacers of extrachromosomal origin, Microbiology, 151, 2551, 10.1099/mic.0.28048-0 · DOI · Paper page
  25. Jinek, 2012, A programmable Dual-RNA — guided, Science (80-), 337, 816, 10.1126/science.1225829 · DOI · Paper page
  26. Ran, 2015, In vivo genome editing using Staphylococcus aureus Cas9, Nature, 520, 186, 10.1038/nature14299 · DOI · Paper page
  27. Qi, 2013, Repurposing CRISPR as an RNA-guided platform for sequence-specific control of gene expression, Cell, 152, 1173, 10.1016/j.cell.2013.02.022 · DOI · Paper page
  28. Gilbert, 2013, CRISPR-mediated modular RNA-guided regualtion of transcription in eukaryotes, Cell, 154, 442, 10.1016/j.cell.2013.06.044 · DOI · Paper page
  29. Liu, 2016, Single-cell analysis of long non-coding RNAs in the developing human neocortex, Genome Biol, 1, 7
  30. Cheng, 2016, Casilio: a versatile CRISPR-Cas9-Pumilio hybrid for gene regulation and genomic labeling, Cell Res, 26, 254, 10.1038/cr.2016.3 · DOI
  31. Guilinger, 2014, Fusion of catalytically inactive Cas9 to FokI nuclease improves the specificity of genome modification, Nat Biotechnol, 32, 577, 10.1038/nbt.2909 · DOI · Paper page
  32. Ran, 2013, Genome engineering using the CRISPR-Cas9 system, Nat Protoc, 8, 2281, 10.1038/nprot.2013.143 · DOI · Paper page
  33. Cong, 2013, Multiplex genome engineering using CRISPR/Cas systems, Science, 339, 819, 10.1126/science.1231143 · DOI · Paper page
  34. Gasiunas, 2012, Cas9-crRNA ribonucleoprotein complex mediates specific DNA cleavage for adaptive immunity in bacteria, Proc Natl Acad Sci, 109, E2579, 10.1073/pnas.1208507109 · DOI · Paper page
  35. Ran, 2013, Double nicking by RNA-guided CRISPR cas9 for enhanced genome editing specificity, Cell, 154, 1380, 10.1016/j.cell.2013.08.021 · DOI · Paper page
  36. Koch, 2017, Genetic rescue models refute nonautonomous rod cell death in retinitis pigmentosa, Proc Natl Acad Sci, 114, 5259, 10.1073/pnas.1615394114 · DOI
  37. Mao, 2008, Comparison of nonhomologous end joining and homologous recombination in human cells, DNA Repair (Amst), 7, 1765, 10.1016/j.dnarep.2008.06.018 · DOI
  38. Miyaoka, 2016, Systematic quantification of HDR and NHEJ reveals effects of locus, nuclease, and cell type on genome-editing, Sci Rep, 6, 10.1038/srep23549 · DOI
  39. Hsu, 2013, DNA targeting specificity of RNA-guided Cas9 nucleases, Nat Biotechnol, 31, 827, 10.1038/nbt.2647 · DOI · Paper page
  40. Fu, 2013, High-frequency off-target mutagenesis induced by CRISPR-Cas nucleases in human cells, Nat Biotechnol, 31, 822, 10.1038/nbt.2623 · DOI · Paper page
  41. Bibikova, 2001, Stimulation of homologous recombination through targeted cleavage by chimeric nucleases, Mol Cell Biol, 21, 289, 10.1128/MCB.21.1.289-297.2001 · DOI
  42. Kim, 1994, Chimeric restriction endonuclease, Proc Natl Acad Sci U S A, 91, 883, 10.1073/pnas.91.3.883 · DOI
  43. Kim, 1996, Hybrid restriction enzymes: zinc finger fusions to Fok I cleavage domain, Proc Natl Acad Sci, 93, 1156, 10.1073/pnas.93.3.1156 · DOI · Paper page
  44. Kim, 1998, Chimeric restriction enzyme: Gal4 fusion to Fok cleavage domain, Biol Chem Hoppe Seyler, 379, 489
  45. Smith, 2000, Requirements for double-strand cleavage by chimeric restriction enzymes with zinc finger DNA-recognition domains, Nucleic Acids Res, 28, 3361, 10.1093/nar/28.17.3361 · DOI
  46. Bitinaite, 1998, FokI dimerization is required for DNA cleavage, Biochemistry, 95, 10570
  47. Mandell, 2006, Zinc finger tools: custom DNA-binding domains for transcription factors and nucleases, Nucleic Acids Res, 34, 10.1093/nar/gkl209 · DOI
  48. Gupta, 2014, Expanding the genetic editing tool kit: ZFNs, TALENs, and CRISPR-Cas9, J Clin Invest, 124, 4154, 10.1172/JCI72992 · DOI
  49. Christian, 2010, Targeting DNA double-strand breaks with TAL effector nucleases, Genetics, 186, 756, 10.1534/genetics.110.120717 · DOI · Paper page
  50. Nemudryi, 2014, TALEN and CRISPR/Cas genome editing systems: tools of discovery, Acta Naturae, 6, 19, 10.32607/20758251-2014-6-3-19-40 · DOI
  51. Gaj, 2013, ZFN, TALEN and CRISPR/Cas based methods for genome engineering, Trends Biotechnol, 31, 397, 10.1016/j.tibtech.2013.04.004 · DOI · Paper page
  52. Tsubura, 2010, Animal models for retinitis pigmentosa induced by MNU; disease progression, mechanisms and therapeutic trials, Histol Histopathol, 25, 933
  53. Lyraki, 2016, Disease mechanisms of X-linked retinitis pigmentosa due to RP2 and RPGR mutations, Biochem Soc Trans, 44, 1235, 10.1042/BST20160148 · DOI
  54. Arno, 2016, Mutations in REEP6 cause autosomal-recessive retinitis pigmentosa, Am J Hum Genet, 99, 1305, 10.1016/j.ajhg.2016.10.008 · DOI
  55. Wu, 2016, CRISPR repair reveals causative mutation in a preclinical model of retinitis pigmentosa, Mol Ther, 24, 1388, 10.1038/mt.2016.107 · DOI
  56. Lv, 2017, Targeted RP9 ablation and mutagenesis in mouse photoreceptor cells by CRISPR-Cas9, Sci Rep, 7, 43062, 10.1038/srep43062 · DOI
  57. Coppieters, 2010, Genetic screening of LCA in Belgium: predominance of CEP290 and identification of potential modifier alleles in AHI1 of CEP290-related phenotypes, Hum Mutat, 3, 1
  58. Zhong, 2015, CRISPR-engineered mosaicism rapidly reveals that loss of Kcnj13 function in mice mimics human disease phenotypes, Sci Rep, 5, 8366, 10.1038/srep08366 · DOI
  59. Wang, 2010, Transplantation of reprogrammed embryonic stem cells improves visual function in a mouse model for retinitis pigmentosa, Transplantation, 89, 911, 10.1097/TP.0b013e3181d45a61 · DOI
  60. Yu-Wai-Man, 2016, Genetic manipulation for inherited neurodegenerative diseases: myth or reality?, Br J Ophthalmol, 100, 1322, 10.1136/bjophthalmol-2015-308329 · DOI
  61. Bakondi, 2016, In vivo CRISPR/Cas9 gene editing corrects retinal dystrophy in the S334ter-3 rat model of autosomal dominant retinitis pigmentosa, Mol Ther, 24, 556, 10.1038/mt.2015.220 · DOI
  62. McGill, 2012, Optomotor and immunohistochemical changes in the juvenile S334ter rat, Exp Eye Res, 104, 65, 10.1016/j.exer.2012.09.006 · DOI
  63. Latella, 2016, In vivo editing of the human mutant rhodopsin gene by electroporation of plasmid-based CRISPR/Cas9 in the mouse retina, Mol Ther Nucleic Acids, 5, 10.1038/mtna.2016.92 · DOI
  64. Olsson, 1992, Transgenic mice with a rhodopsin mutation (Pro23His): a mouse model of autosomal dominant retinitis pigmentosa, Neuron, 9, 815, 10.1016/0896-6273(92)90236-7 · DOI · Paper page
  65. Giannelli, 2017, Cas9/sgRNA selective targeting of the P23H Rhodopsin mutant allele for treating retinitis pigmentosa by intravitreal AAV9. PHP.B-based delivery, Hum Mol Genet, 27, 761, 10.1093/hmg/ddx438 · DOI
  66. Li, 2017, Allele-specific editing of rhodopsin P23H knock-in mice broadens therapeutic potential of CRISPR/Cas for dominant genetic diseases, bioRxiv
  67. Bassuk, 2016, Precision medicine: genetic repair of retinitis pigmentosa in patient-derived stem cells, Sci Rep, 6, 19969, 10.1038/srep19969 · DOI
  68. Suzuki, 2016, In vivo genome editing via CRISPR/Cas9 mediated homology-independent targeted integration, Nature, 540, 144, 10.1038/nature20565 · DOI · Paper page
  69. Kim, 2017, Genome surgery using Cas9 ribonucleoproteins for the treatment of age-related macular degeneration, Genome Res, 27, 419, 10.1101/gr.219089.116 · DOI
  70. Huang, 2017, Editing VEGFR2 blocks VEGF-induced activation of akt and tube formation, Investig Ophthalmol Vis Sci, 58, 1228, 10.1167/iovs.16-20537 · DOI
  71. Yiu, 2016, Genomic disruption of VEGF-A expression in human retinal pigment epithelial cells using CRISPR-Cas9 endonuclease, Investig Opthalmol Vis Sci, 57, 5490, 10.1167/iovs.16-20296 · DOI
  72. Ruan, 2017, CRISPR/Cas9-mediated genome editing as a therapeutic approach for leber congenital amaurosis 10, Mol Ther, 25, 331, 10.1016/j.ymthe.2016.12.006 · DOI · Paper page

Cited by

Sorted by citations

Metadata

Authors
Xu, C. L., Park, K. S., & Tsang, S. H.
Venue
Drug Discovery Today: Technologies
Publisher
Elsevier BV
Published
2018-08
Volume
28
Pages
23-32
ISSN
1740-6749
DOI
10.1016/j.ddtec.2018.05.001
PMC
PMC6136254
Cited by
14
References
6
Language
en
Licence
Funders
  • National Eye Institute, NIH (P30EY019007, R01EY018213, R01EY024698, R01EY026682, R21AG050437)
  • National Cancer Institute Core (5P30CA013696)
  • Tistou and Charlotte Kerstan Foundation
  • Schneeweiss Stem Cell Fund, New York State (C029572)
  • Foundation Fighting Blindness New York Regional Research Center Grant (C-NY05-0705-0312)
  • Crowley Family Fund
  • Gebroe Family Foundation

About these checks

Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.

Statistics and arithmetic are recomputed, not judged.

Confidence bands are shown only for fields calibrated on our annotated test set. None is calibrated yet, so no band is shown.

What counts as a claim ›How we measure confidence ›How we check this ›

Are you an author, or do you see a problem with this page? Request a review