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CRISPR-mediated genome editing and human diseases

Cai, Liquan, Fisher, Alfred L., Huang, Haochu, Xie, Zijian·2016·Genes & Diseases
DOI 10.1016/j.gendis.2016.07.003PMC PMC6150104
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Fig. 1 CRISPR working mechanism. Guide RNA hybridizes with 20bp genomic DNA sequence and directs Cas9 endonuclease (colored in pink) to generate a double strand break which is usually located between 16 and 17bp region in the target sequence. Subsequently, DNA Mutagenesis is generated from DNA repair process, through either the non-homologous end-joining (NHEJ) or the homology-directed repair (HDR) mechanism. The final mutation could include insertion or deletion with several base pairs of DNA sequences (NHEJ pathway), or replacement with a particular DNA sequence used as a marker for further study (encoding for a fluorescence protein, tag protein, antibiotics, or the recognition sequence for a restriction enzyme digestion).
Fig. 1 CRISPR working mechanism. Guide RNA hybridizes with 20bp genomic DNA sequence and directs Cas9 endonuclease (colored in pink) to generate a double strand break which is usually located between 16 and 17bp region in the target sequence. Subsequently, DNA Mutagenesis is generated from DNA repair process, through either the non-homologous end-joining (NHEJ) or the homology-directed repair (HDR) mechanism. The final mutation could include insertion or deletion with several base pairs of DNA sequences (NHEJ pathway), or replacement with a particular DNA sequence used as a marker for further study (encoding for a fluorescence protein, tag protein, antibiotics, or the recognition sequence for a restriction enzyme digestion).CC BY-NC-ND · PubMed Central
Fig. 2 CRISPR engineering for the clinics. Advances in DNA sequencing technology make it easier to identify the disease-driving genetic mutations. Meanwhile, the patient-derived iPS cells have been established to model human diseases and drug discovery in vitro. The deteriorating mutations can be corrected via the use of CRISPR-mediated gene editing, and the modified cells can be then utilized as patient-specific medicine.
Fig. 2 CRISPR engineering for the clinics. Advances in DNA sequencing technology make it easier to identify the disease-driving genetic mutations. Meanwhile, the patient-derived iPS cells have been established to model human diseases and drug discovery in vitro. The deteriorating mutations can be corrected via the use of CRISPR-mediated gene editing, and the modified cells can be then utilized as patient-specific medicine.CC BY-NC-ND · PubMed Central

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Research works printed but not linked: 65

  1. Ishino, 1987, Nucleotide sequence of the iap gene, responsible for alkaline phosphatase isozyme conversion in Escherichia coli, and identification of the gene product, J Bacteriol, 169, 5429, 10.1128/jb.169.12.5429-5433.1987 · DOI · Paper page
  2. Lander, 2016, The heroes of CRISPR, Cell, 164, 18, 10.1016/j.cell.2015.12.041 · DOI
  3. Mojica, 2000, Biological significance of a family of regularly spaced repeats in the genomes of archaea, bacteria and mitochondria, Mol Microbiol, 36, 244, 10.1046/j.1365-2958.2000.01838.x · DOI
  4. Jansen, 2002, Identification of genes that are associated with DNA repeats in prokaryotes, Mol Microbiol, 43, 1565, 10.1046/j.1365-2958.2002.02839.x · DOI · Paper page
  5. Jinek, 2012, A programmable dual-RNA-guided DNA endonuclease in adaptive bacterial immunity, Science, 337, 816, 10.1126/science.1225829 · DOI · Paper page
  6. Barrangou, 2007, CRISPR provides acquired resistance against viruses in prokaryotes, Science, 315, 1709, 10.1126/science.1138140 · DOI · Paper page
  7. Cong, 2013, Multiplex genome engineering using CRISPR/Cas systems, Science, 339, 819, 10.1126/science.1231143 · DOI · Paper page
  8. Mali, 2013, RNA-guided human genome engineering via Cas9, Science, 339, 823, 10.1126/science.1232033 · DOI · Paper page
  9. Dickinson, 2013, Engineering the Caenorhabditis elegans genome using Cas9-triggered homologous recombination, Nat Methods, 10, 1028, 10.1038/nmeth.2641 · DOI
  10. Lin, 2014, Enhanced homology-directed human genome engineering by controlled timing of CRISPR/Cas9 delivery, Elife, 3, e04766, 10.7554/eLife.04766 · DOI · Paper page
  11. Irion, 2014, Precise and efficient genome editing in zebrafish using the CRISPR/Cas9 system, Development, 141, 4827, 10.1242/dev.115584 · DOI
  12. Baena-Lopez, 2013, Accelerated homologous recombination and subsequent genome modification in Drosophila, Development, 140, 4818, 10.1242/dev.100933 · DOI
  13. Cox, 2015, Therapeutic genome editing: prospects and challenges, Nat Med, 21, 121, 10.1038/nm.3793 · DOI · Paper page
  14. Maeder, 2016, Genome-editing technologies for gene and cell therapy, Mol Ther, 24, 430, 10.1038/mt.2016.10 · DOI
  15. Hsu, 2014, Development and applications of CRISPR-Cas9 for genome engineering, Cell, 157, 1262, 10.1016/j.cell.2014.05.010 · DOI · Paper page
  16. 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
  17. Larson, 2013, CRISPR interference (CRISPRi) for sequence-specific control of gene expression, Nat Protoc, 8, 2180, 10.1038/nprot.2013.132 · DOI
  18. 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
  19. Gao, 2016, DNA-guided genome editing using the Natronobacterium gregoryi Argonaute, Nat Biotechnol, 34, 768, 10.1038/nbt.3547 · DOI
  20. Merkle, 2013, Modeling human disease with pluripotent stem cells: from genome association to function, Cell Stem Cell, 12, 656, 10.1016/j.stem.2013.05.016 · DOI
  21. Takahashi, 2006, Induction of pluripotent stem cells from mouse embryonic and adult fibroblast cultures by defined factors, Cell, 126, 663, 10.1016/j.cell.2006.07.024 · DOI · Paper page
  22. Anokye-Danso, 2011, Highly efficient miRNA-mediated reprogramming of mouse and human somatic cells to pluripotency, Cell Stem Cell, 8, 376, 10.1016/j.stem.2011.03.001 · DOI
  23. Zhu, 2010, Reprogramming of human primary somatic cells by OCT4 and chemical compounds, Cell Stem Cell, 7, 651, 10.1016/j.stem.2010.11.015 · DOI · Paper page
  24. Shi, 2008, Induction of pluripotent stem cells from mouse embryonic fibroblasts by Oct4 and Klf4 with small-molecule compounds, Cell Stem Cell, 3, 568, 10.1016/j.stem.2008.10.004 · DOI
  25. Hou, 2013, Pluripotent stem cells induced from mouse somatic cells by small-molecule compounds, Science, 341, 651, 10.1126/science.1239278 · DOI · Paper page
  26. Soldner, 2009, Parkinson's disease patient-derived induced pluripotent stem cells free of viral reprogramming factors, Cell, 136, 964, 10.1016/j.cell.2009.02.013 · DOI
  27. Dimos, 2008, Induced pluripotent stem cells generated from patients with ALS can be differentiated into motor neurons, Science, 321, 1218, 10.1126/science.1158799 · DOI
  28. Sampaziotis, 2015, Cholangiocytes derived from human induced pluripotent stem cells for disease modeling and drug validation, Nat Biotechnol, 33, 845, 10.1038/nbt.3275 · DOI
  29. Juopperi, 2011, Modeling neurological diseases using patient-derived induced pluripotent stem cells, Future Neurol, 6, 363, 10.2217/fnl.11.14 · DOI
  30. Freude, 2014, Induced pluripotent stem cells derived from Alzheimer's disease patients: the promise, the hope and the path ahead, J Clin Med, 3, 1402, 10.3390/jcm3041402 · DOI
  31. Saric, 2015, Barth syndrome: from mitochondrial dysfunctions associated with aberrant production of reactive oxygen species to pluripotent stem cell studies, Front Genet, 6, 359
  32. Wang, 2014, Modeling the mitochondrial cardiomyopathy of Barth syndrome with induced pluripotent stem cell and heart-on-chip technologies, Nat Med, 20, 616, 10.1038/nm.3545 · DOI · Paper page
  33. Park, 2015, Functional correction of large factor VIII gene chromosomal inversions in hemophilia a patient-derived iPSCs using CRISPR-Cas9, Cell Stem Cell, 17, 213, 10.1016/j.stem.2015.07.001 · DOI
  34. Flynn, 2015, CRISPR-mediated genotypic and phenotypic correction of a chronic granulomatous disease mutation in human iPS cells, Exp Hematol, 43, 10.1016/j.exphem.2015.06.002 · DOI
  35. Xu, 2015, Both TALENs and CRISPR/Cas9 directly target the HBB IVS2-654 (C > T) mutation in beta-thalassemia-derived iPSCs, Sci Rep, 5, 12065, 10.1038/srep12065 · DOI
  36. Xie, 2014, Seamless gene correction of beta-thalassemia mutations in patient-specific iPSCs using CRISPR/Cas9 and piggyBac, Genome Res, 24, 1526, 10.1101/gr.173427.114 · DOI · Paper page
  37. Li, 2015, Precise correction of the dystrophin gene in duchenne muscular dystrophy patient induced pluripotent stem cells by TALEN and CRISPR-Cas9, Stem Cell Rep, 4, 143, 10.1016/j.stemcr.2014.10.013 · DOI · Paper page
  38. Ramos, 2016, CAR-T cell therapy for lymphoma, Annu Rev Med, 67, 165, 10.1146/annurev-med-051914-021702 · DOI
  39. Morgan, 2013, Cancer regression and neurological toxicity following anti-MAGE-A3 TCR gene therapy, J Immunother, 36, 133, 10.1097/CJI.0b013e3182829903 · DOI
  40. Topalian, 2012, Safety, activity, and immune correlates of anti-PD-1 antibody in cancer, N Engl J Med, 366, 2443, 10.1056/NEJMoa1200690 · DOI · Paper page
  41. Brahmer, 2010, Phase I study of single-agent anti-programmed death-1 (MDX-1106) in refractory solid tumors: safety, clinical activity, pharmacodynamics, and immunologic correlates, J Clin Oncol, 28, 3167, 10.1200/JCO.2009.26.7609 · DOI
  42. Beane, 2015, Clinical scale zinc finger nuclease-mediated gene editing of PD-1 in tumor infiltrating lymphocytes for the treatment of metastatic melanoma, Mol Ther, 23, 1380, 10.1038/mt.2015.71 · DOI
  43. Schumann, 2015, Generation of knock-in primary human T cells using Cas9 ribonucleoproteins, Proc Natl Acad Sci U S A, 112, 10437, 10.1073/pnas.1512503112 · DOI · Paper page
  44. Tebas, 2014, Gene editing of CCR5 in autologous CD4 T cells of persons infected with HIV, N Engl J Med, 370, 901, 10.1056/NEJMoa1300662 · DOI · Paper page
  45. Gu, 2015, Genome editing-based HIV therapies, Trends Biotechnol, 33, 172, 10.1016/j.tibtech.2014.12.006 · DOI
  46. Yin, 2014, Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype, Nat Biotechnol, 32, 551, 10.1038/nbt.2884 · DOI · Paper page
  47. Chen, 2015, Genome-wide CRISPR screen in a mouse model of tumor growth and metastasis, Cell, 160, 1246, 10.1016/j.cell.2015.02.038 · DOI · Paper page
  48. Nakamura, 2014, Generation of muscular dystrophy model rats with a CRISPR/Cas system, Sci Rep, 4, 5635, 10.1038/srep05635 · DOI
  49. Chen, 2015, Functional disruption of the dystrophin gene in rhesus monkey using CRISPR/Cas9, Hum Mol Genet, 24, 3764, 10.1093/hmg/ddv120 · DOI · Paper page
  50. Nelson, 2016, In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy, Science, 351, 403, 10.1126/science.aad5143 · DOI · Paper page
  51. Long, 2016, Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophy, Science, 351, 400, 10.1126/science.aad5725 · DOI
  52. Tabebordbar, 2016, In vivo gene editing in dystrophic mouse muscle and muscle stem cells, Science, 351, 407, 10.1126/science.aad5177 · DOI · Paper page
  53. Kaminski, 2016, Excision of HIV-1 DNA by gene editing: a proof-of-concept in vivo study, Gene Ther, 23, 690, 10.1038/gt.2016.41 · DOI
  54. Kaminski, 2016, Elimination of HIV-1 genomes from human T-lymphoid cells by CRISPR/Cas9 gene editing, Sci Rep, 6, 22555, 10.1038/srep22555 · DOI
  55. Reardon, 2015, New life for pig-to-human transplants, Nature, 527, 152, 10.1038/527152a · DOI
  56. Yang, 2015, Genome-wide inactivation of porcine endogenous retroviruses (PERVs), Science, 350, 1101, 10.1126/science.aad1191 · DOI
  57. Elliott, 2007, Live encapsulated porcine islets from a type 1 diabetic patient 9.5 yr after xenotransplantation, Xenotransplantation, 14, 157, 10.1111/j.1399-3089.2007.00384.x · DOI
  58. Peng, 2015, Production of human albumin in pigs through CRISPR/Cas9-mediated knockin of human cDNA into swine albumin locus in the zygotes, Sci Rep, 5, 16705, 10.1038/srep16705 · DOI
  59. Han, 2015, CRISPR-Cas9 delivery to hard-to-transfect cells via membrane deformation, Sci Adv, 1, e1500454, 10.1126/sciadv.1500454 · DOI
  60. Qin, 2015, Efficient CRISPR/Cas9-mediated genome editing in mice by zygote electroporation of nuclease, Genetics, 200, 423, 10.1534/genetics.115.176594 · DOI
  61. Cheng, 2014, Efficient gene editing in adult mouse livers via adenoviral delivery of CRISPR/Cas9, FEBS Lett, 588, 3954, 10.1016/j.febslet.2014.09.008 · DOI
  62. Yin, 2015, A geminivirus-based guide RNA delivery system for CRISPR/Cas9 mediated plant genome editing, Sci Rep, 5, 14926, 10.1038/srep14926 · DOI
  63. Zinn, 2015, In silico reconstruction of the viral evolutionary lineage yields a potent gene therapy vector, Cell Rep, 12, 1056, 10.1016/j.celrep.2015.07.019 · DOI · Paper page
  64. Liang, 2015, CRISPR/Cas9-mediated gene editing in human tripronuclear zygotes, Protein Cell, 6, 363, 10.1007/s13238-015-0153-5 · DOI · Paper page
  65. Kang, 2016, Introducing precise genetic modifications into human 3PN embryos by CRISPR/Cas-mediated genome editing, J Assist Reprod Genet, 33, 581, 10.1007/s10815-016-0710-8 · DOI

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Metadata

Authors
Cai, L., Fisher, A. L., Huang, H., & Xie, Z.
ORCID
Venue
Genes & Diseases
Publisher
Elsevier BV
Published
2016-12
Volume
3
Issue
4
Pages
244-251
ISSN
2352-3042
DOI
10.1016/j.gendis.2016.07.003
PMC
PMC6150104
Cited by
83
References
15
Language
en
Licence
Funders
  • National Institutes of Health
  • National Institute of Allergy and Infectious Diseases (AG013319, AG044271, AG044768, ES017761)

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