Cited by 210

The DOI matches its Crossref record; 39 of the 39 references it cites were found in a registry or an index.

In vivo delivery of CRISPR-Cas9 therapeutics: Progress and challenges

Behr, Matthew, Zhou, Jing, Xu, Bing, Zhang, Hongwei·2021·Acta Pharmaceutica Sinica B

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.apsb.2021.05.020

Cites

39 of 39 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

Flagged first, then by citations

Research works printed but not linked: 160

  1. Cox, 2015, Therapeutic genome editing: prospects and challenges, Nat Med, 21, 121, 10.1038/nm.3793 · DOI · Paper page
  2. Hardiman, 2020, The beginning of genomic therapies for ALS, N Engl J Med, 383, 180, 10.1056/NEJMe2012930 · DOI
  3. Verhaart, 2019, Therapeutic developments for Duchenne muscular dystrophy, Nat Rev Neurol, 15, 373, 10.1038/s41582-019-0203-3 · DOI
  4. Wang, 2020, CRISPR-based therapeutic genome editing: strategies and in vivo delivery by AAV vectors, Cell, 181, 136, 10.1016/j.cell.2020.03.023 · DOI
  5. Komor, 2017, CRISPR-based technologies for the manipulation of eukaryotic genomes, Cell, 169, 559, 10.1016/j.cell.2017.04.005 · DOI
  6. van der Oost, 2014, Unravelling the structural and mechanistic basis of CRISPR-Cas systems, Nat Rev Microbiol, 12, 479, 10.1038/nrmicro3279 · DOI
  7. Gasiunas, 2012, Cas9-crRNA ribonucleoprotein complex mediates specific DNA cleavage for adaptive immunity in bacteria, Proc Natl Acad Sci U S A, 109, E2579, 10.1073/pnas.1208507109 · DOI · Paper page
  8. Jinek, 2013, RNA-programmed genome editing in human cells, Elife, 2, 10.7554/eLife.00471 · DOI · Paper page
  9. Sternberg, 2014, DNA interrogation by the CRISPR RNA-guided endonuclease Cas9, Nature, 507, 62, 10.1038/nature13011 · DOI · Paper page
  10. Sternberg, 2015, Conformational control of DNA target cleavage by CRISPR-Cas9, Nature, 527, 110, 10.1038/nature15544 · DOI · Paper page
  11. McVey, 2008, MMEJ repair of double-strand breaks (directors cut): deleted sequences and alternative endings, Trends Genet, 24, 529, 10.1016/j.tig.2008.08.007 · DOI
  12. Sakuma, 2016, MMEJ-assisted gene knock-in using TALENs and CRISPR-Cas9 with the PITCh systems, Nat Protoc, 11, 118, 10.1038/nprot.2015.140 · DOI
  13. Yao, 2017, Homology-mediated end joining-based targeted integration using CRISPR/Cas9, Cell Res, 27, 801, 10.1038/cr.2017.76 · DOI
  14. Suzuki, 2016, In vivo genome editing via CRISPR/Cas9 mediated homology-independent targeted integration, Nature, 540, 144, 10.1038/nature20565 · DOI · Paper page
  15. Crudele, 2018, Cas9 immunity creates challenges for CRISPR gene editing therapies, Nat Commun, 9, 3497, 10.1038/s41467-018-05843-9 · DOI · Paper page
  16. Lattanzi, 2019, Optimization of CRISPR/Cas9 delivery to human hematopoietic stem and progenitor cells for therapeutic genomic rearrangements, Mol Ther, 27, 137, 10.1016/j.ymthe.2018.10.008 · DOI
  17. Senturk, 2017, Rapid and tunable method to temporally control gene editing based on conditional Cas9 stabilization, Nat Commun, 8, 14370, 10.1038/ncomms14370 · DOI
  18. Gaj, 2017, Targeted gene knock-in by homology-directed genome editing using Cas9 ribonucleoprotein and AAV donor delivery, Nucleic Acids Res, 45, e98, 10.1093/nar/gkx154 · DOI · Paper page
  19. Chen, 2019, CRISPR-READI: efficient generation of knockin mice by CRISPR RNP electroporation and AAV donor infection, Cell Rep, 27, 10.1016/j.celrep.2019.05.103 · DOI
  20. Bak, 2017, CRISPR-mediated integration of large gene cassettes using AAV donor vectors, Cell Rep, 20, 750, 10.1016/j.celrep.2017.06.064 · DOI · Paper page
  21. Nishiyama, 2017, Virus-mediated genome editing via homology-directed repair in mitotic and postmitotic cells in mammalian brain, Neuron, 96, 10.1016/j.neuron.2017.10.004 · DOI · Paper page
  22. Wan, 2021, Delivery of genome-editing biomacromolecules for treatment of lung genetic disorders, Adv Drug Deliv Rev, 168, 196, 10.1016/j.addr.2020.05.002 · DOI
  23. Kay, 2001, Viral vectors for gene therapy: the art of turning infectious agents into vehicles of therapeutics, Nat Med, 7, 33, 10.1038/83324 · DOI
  24. Anguela, 2019, Entering the modern era of gene therapy, Annu Rev Med, 70, 273, 10.1146/annurev-med-012017-043332 · DOI
  25. High, 2020, Turning genes into medicines—what have we learned from gene therapy drug development in the past decade?, Nat Commun, 11, 5821, 10.1038/s41467-020-19507-0 · DOI
  26. Wang, 2019, Adeno-associated virus vector as a platform for gene therapy delivery, Nat Rev Drug Discov, 18, 358, 10.1038/s41573-019-0012-9 · DOI · Paper page
  27. Hastie, 2015, Adeno-associated virus at 50: a golden anniversary of discovery, research, and gene therapy success—a personal perspective, Hum Gene Ther, 26, 257, 10.1089/hum.2015.025 · DOI
  28. Flotte, 2013, Birth of a new therapeutic platform: 47 years of adeno-associated virus biology from virus discovery to licensed gene therapy, Mol Ther, 21, 1976, 10.1038/mt.2013.226 · DOI
  29. Weitzman, 2011, Adeno-associated virus biology, Methods Mol Biol, 807, 1
  30. Friedland, 2015, Characterization of Staphylococcus aureus Cas9: a smaller Cas9 for all-in-one adeno-associated virus delivery and paired nickase applications, Genome Biol, 16, 257, 10.1186/s13059-015-0817-8 · DOI
  31. Hung, 2016, AAV-mediated CRISPR/Cas gene editing of retinal cells in vivo, Invest Ophthalmol Vis Sci, 57, 3470, 10.1167/iovs.16-19316 · DOI · Paper page
  32. Vakulskas, 2019, Evaluation and reduction of CRISPR off-target cleavage events, Nucleic Acid Therapeut, 29, 167, 10.1089/nat.2019.0790 · DOI
  33. Zhang, 2009, Adenovirus-adeno-associated virus hybrid for large-scale recombinant adeno-associated virus production, Hum Gene Ther, 20, 922, 10.1089/hum.2009.125 · DOI
  34. Grieger, 2012, Adeno-associated virus vectorology, manufacturing, and clinical applications, Methods Enzymol, 507, 229, 10.1016/B978-0-12-386509-0.00012-0 · DOI · Paper page
  35. Qu, 2015, Scalable downstream strategies for purification of recombinant adeno- associated virus vectors in light of the properties, Curr Pharmaceut Biotechnol, 16, 684, 10.2174/1389201016666150505122228 · DOI
  36. Zhang, 2019, Curing hemophilia A by NHEJ-mediated ectopic F8 insertion in the mouse, Genome Biol, 20, 276, 10.1186/s13059-019-1907-9 · DOI
  37. Chen, 2019, Hemophilia A ameliorated in mice by CRISPR-based in vivo genome editing of human Factor VIII, Sci Rep, 9, 16838, 10.1038/s41598-019-53198-y · DOI
  38. Wang, 2020, CRISPR-Cas9-mediated in vivo gene integration at the albumin locus recovers hemostasis in neonatal and adult hemophilia B mice, Mol Ther Methods Clin Dev, 18, 520, 10.1016/j.omtm.2020.06.025 · DOI
  39. Richards, 2020, AAV-mediated CRISPR/Cas9 gene editing in murine phenylketonuria, Mol Ther Methods Clin Dev, 17, 234, 10.1016/j.omtm.2019.12.004 · DOI
  40. Villiger, 2018, Treatment of a metabolic liver disease by in vivo genome base editing in adult mice, Nat Med, 24, 1519, 10.1038/s41591-018-0209-1 · DOI
  41. Wang, 2020, A mutation-independent CRISPR-Cas9-mediated gene targeting approach to treat a murine model of ornithine transcarbamylase deficiency, Sci Adv, 6
  42. Zhang, 2020, Enhanced CRISPR-Cas9 correction of Duchenne muscular dystrophy in mice by a self-complementary AAV delivery system, Sci Adv, 6
  43. Xu, 2019, Life-Long AAV-mediated CRISPR genome editing in dystrophic heart improves cardiomyopathy without causing serious lesions in MDX mice, Mol Ther, 27, 1407, 10.1016/j.ymthe.2019.05.001 · DOI
  44. Koo, 2018, Functional rescue of dystrophin deficiency in mice caused by frameshift mutations using Campylobacter jejuni Cas9, Mol Ther, 26, 1529, 10.1016/j.ymthe.2018.03.018 · DOI
  45. Hu, 2020, In vivo CRISPR/Cas9-Mediated genome editing mitigates photoreceptor degeneration in a mouse model of X-linked retinitis pigmentosa, Invest Ophthalmol Vis Sci, 61, 31, 10.1167/iovs.61.4.31 · DOI
  46. Moreno, 2018, In situ gene therapy via AAV-CRISPR-Cas9-mediated targeted gene regulation, Mol Ther, 26, 1818, 10.1016/j.ymthe.2018.04.017 · DOI · Paper page
  47. Weinberg, 1991, Productive human immunodeficiency virus type 1 (HIV-1) infection of nonproliferating human monocytes, J Exp Med, 174, 1477, 10.1084/jem.174.6.1477 · DOI
  48. Naldini, 1998, Lentiviruses as gene transfer agents for delivery to non-dividing cells, Curr Opin Biotechnol, 9, 457, 10.1016/S0958-1669(98)80029-3 · DOI
  49. Miyoshi, 1999, Transduction of human CD34+ cells that mediate long-term engraftment of NOD/SCID mice by HIV vectors, Science, 283, 682, 10.1126/science.283.5402.682 · DOI
  50. Dull, 1998, A third-generation lentivirus vector with a conditional packaging system, J Virol, 72, 8463, 10.1128/JVI.72.11.8463-8471.1998 · DOI · Paper page
  51. Naldini, 2016, Lentiviral vectors, two decades later, Science, 353, 1101, 10.1126/science.aah6192 · DOI
  52. Consiglio, 2001, In vivo gene therapy of metachromatic leukodystrophy by lentiviral vectors: correction of neuropathology and protection against learning impairments in affected mice, Nat Med, 7, 310, 10.1038/85454 · DOI
  53. Gentner, 2009, Stable knockdown of microRNA in vivo by lentiviral vectors, Nat Methods, 6, 63, 10.1038/nmeth.1277 · DOI
  54. Kay, 2011, State-of-the-art gene-based therapies: the road ahead, Nat Rev Genet, 12, 316, 10.1038/nrg2971 · DOI
  55. Naldini, 2011, Ex vivo gene transfer and correction for cell-based therapies, Nat Rev Genet, 12, 301, 10.1038/nrg2985 · DOI
  56. Ling, 2021, Lentiviral delivery of co-packaged Cas9 mRNA and a VEGFa-targeting guide RNA prevents wet age-related macular degeneration in mice, Nat Biomed Eng, 5, 144, 10.1038/s41551-020-00656-y · DOI
  57. Kim, 2018, Targeting mutant KRAS with CRISPR-Cas9 controls tumor growth, Genome Res, 28, 374, 10.1101/gr.223891.117 · DOI · Paper page
  58. Chen, 2020, ABL genomic editing sufficiently abolishes oncogenesis of human chronic myeloid leukemia cells in vitro and in vivo, Cancers (Basel), 12
  59. Wei, 2020, Delivery of tissue-targeted scalpels: opportunities and challenges for in vivo CRISPR/Cas-based genome editing, ACS Nano, 14, 9243, 10.1021/acsnano.0c04707 · DOI
  60. Allen, 2004, Drug delivery systems: entering the mainstream, Science, 303, 1818, 10.1126/science.1095833 · DOI · Paper page
  61. Lasic, 1988, The mechanism of vesicle formation, Biochem J, 256, 1, 10.1042/bj2560001 · DOI
  62. Witzigmann, 2020, Lipid nanoparticle technology for therapeutic gene regulation in the liver, Adv Drug Deliv Rev, 159, 344, 10.1016/j.addr.2020.06.026 · DOI · Paper page
  63. Zuris, 2015, Cationic lipid-mediated delivery of proteins enables efficient protein-based genome editing in vitro and in vivo, Nat Biotechnol, 33, 73, 10.1038/nbt.3081 · DOI · Paper page
  64. Leone, 1999, Global CNS gene transfer for a childhood neurogenetic enzyme deficiency: canavan disease, Curr Opin Mol Ther, 1, 487
  65. Leone, 2000, Aspartoacylase gene transfer to the mammalian central nervous system with therapeutic implications for Canavan disease, Ann Neurol, 48, 27, 10.1002/1531-8249(200007)48:1<27::AID-ANA6>3.0.CO;2-6 · DOI
  66. Qiu, 2021, Lipid nanoparticle-mediated codelivery of Cas9 mRNA and single-guide RNA achieves liver-specific in vivo genome editing of Angptl3, Proc Natl Acad Sci U S A, 118, 10.1073/pnas.2020401118 · DOI
  67. Wei, 2020, Systemic nanoparticle delivery of CRISPR-Cas9 ribonucleoproteins for effective tissue specific genome editing, Nat Commun, 11, 3232, 10.1038/s41467-020-17029-3 · DOI
  68. Wang, 2016, Efficient delivery of genome-editing proteins using bioreducible lipid nanoparticles, Proc Natl Acad Sci U S A, 113, 2868, 10.1073/pnas.1520244113 · DOI · Paper page
  69. Bottger, 2020, Lipid-based nanoparticle technologies for liver targeting, Adv Drug Deliv Rev, 154–155, 79, 10.1016/j.addr.2020.06.017 · DOI
  70. Anselmo, 2019, Nanoparticles in the clinic: an update, Bioeng Transl Med, 4, 10.1002/btm2.10143 · DOI · Paper page
  71. Chen, 2019, A biodegradable nanocapsule delivers a Cas9 ribonucleoprotein complex for in vivo genome editing, Nat Nanotechnol, 14, 974, 10.1038/s41565-019-0539-2 · DOI
  72. Wan, 2020, Genome editing of mutant KRAS through supramolecular polymer-mediated delivery of Cas9 ribonucleoprotein for colorectal cancer therapy, J Control Release, 322, 236, 10.1016/j.jconrel.2020.03.015 · DOI
  73. Guo, 2019, Therapeutic genome editing of triple-negative breast tumors using a noncationic and deformable nanolipogel, Proc Natl Acad Sci U S A, 116, 18295, 10.1073/pnas.1904697116 · DOI
  74. Deng, 2020, Cdk 5 knocking out mediated by CRISPR-Cas9 genome editing for PD-L1 attenuation and enhanced antitumor immunity, Acta Pharm Sin B, 10, 358, 10.1016/j.apsb.2019.07.004 · DOI
  75. Xu, 2018, Targeting of NLRP3 inflammasome with gene editing for the amelioration of inflammatory diseases, Nat Commun, 9, 4092, 10.1038/s41467-018-06522-5 · DOI
  76. Zhang, 2019, In situ repurposing of dendritic cells with CRISPR/Cas9-based nanomedicine to induce transplant tolerance, Biomaterials, 217, 119302, 10.1016/j.biomaterials.2019.119302 · DOI
  77. Rui, 2019, Carboxylated branched poly(beta-amino ester) nanoparticles enable robust cytosolic protein delivery and CRISPR-Cas9 gene editing, Sci Adv, 5, 10.1126/sciadv.aay3255 · DOI
  78. Liu, 2018, Optimization of lipid-assisted nanoparticle for disturbing neutrophils-related inflammation, Biomaterials, 172, 92, 10.1016/j.biomaterials.2018.04.052 · DOI
  79. Luo, 2018, Macrophage-specific in vivo gene editing using cationic lipid-assisted polymeric nanoparticles, ACS Nano, 12, 994, 10.1021/acsnano.7b07874 · DOI
  80. Spicer, 2018, Peptide and protein nanoparticle conjugates: versatile platforms for biomedical applications, Chem Soc Rev, 47, 3574, 10.1039/C7CS00877E · DOI
  81. Fan, 2017, Peptide self-assembled nanostructures for drug delivery applications, J Nanomater, 2017, 4562474, 10.1155/2017/4562474 · DOI
  82. Nakase, 2010, Endosome-disruptive peptides for improving cytosolic delivery of bioactive macromolecules, Pept Sci, 94, 763, 10.1002/bip.21487 · DOI
  83. Krishnamurthy, 2019, Engineered amphiphilic peptides enable delivery of proteins and CRISPR-associated nucleases to airway epithelia, Nat Commun, 10, 4906, 10.1038/s41467-019-12922-y · DOI
  84. Shen, 2018, CRISPR-delivery particles targeting nuclear receptor-interacting protein 1 (NRIP1) in adipose cells to enhance energy expenditure, J Biol Chem, 293, 17291, 10.1074/jbc.RA118.004554 · DOI
  85. Park, 2019, In vivo neuronal gene editing via CRISPR-Cas9 amphiphilic nanocomplexes alleviates deficits in mouse models of Alzheimer's disease, Nat Neurosci, 22, 524, 10.1038/s41593-019-0352-0 · DOI
  86. Lostalé-Seijo, 2017, Peptide/Cas9 nanostructures for ribonucleoprotein cell membrane transport and gene edition, Chem Sci, 8, 7923, 10.1039/C7SC03918B · DOI
  87. He, 2020, Enzymatic noncovalent synthesis for mitochondrial genetic engineering of cancer cells, Cell Reports Physical Science, 1, 10.1016/j.xcrp.2020.100270 · DOI
  88. Ding, 2014, Gold nanoparticles for nucleic acid delivery, Mol Ther, 22, 1075, 10.1038/mt.2014.30 · DOI · Paper page
  89. Giljohann, 2010, Gold nanoparticles for biology and medicine, Angew Chem Int Ed Engl, 49, 3280, 10.1002/anie.200904359 · DOI
  90. Dykman, 2017, Immunological properties of gold nanoparticles, Chem Sci, 8, 1719, 10.1039/C6SC03631G · DOI
  91. Ahmad, 2017, Targeting dendritic cells through gold nanoparticles: a review on the cellular uptake and subsequent immunological properties, Mol Immunol, 91, 123, 10.1016/j.molimm.2017.09.001 · DOI
  92. Lee, 2017, Nanoparticle delivery of Cas9 ribonucleoprotein and donor DNA in vivo induces homology-directed DNA repair, Nat Biomed Eng, 1, 889, 10.1038/s41551-017-0137-2 · DOI · Paper page
  93. Zhou, 2018, Mesoporous silica nanoparticles for drug and gene delivery, Acta Pharm Sin B, 8, 165, 10.1016/j.apsb.2018.01.007 · DOI
  94. Gong, 2020, A versatile nonviral delivery system for multiplex gene-editing in the liver, Adv Mater, 32, 2003537, 10.1002/adma.202003537 · DOI
  95. Zhang, 2020, Co-delivery of sorafenib and CRISPR/Cas9 based on targeted core-shell hollow mesoporous organosilica nanoparticles for synergistic HCC therapy, ACS Appl Mater Interfac, 12, 57362, 10.1021/acsami.0c17660 · DOI
  96. Pan, 2019, Near-infrared upconversion-activated CRISPR-Cas9 system: a remote-controlled gene editing platform, Sci Adv, 5, 10.1126/sciadv.aav7199 · DOI
  97. Liu, 2020, Virus-like nanoparticle as a co-delivery system to enhance efficacy of CRISPR/Cas9-based cancer immunotherapy, Biomaterials, 258, 120275, 10.1016/j.biomaterials.2020.120275 · DOI
  98. Wang, 2020, A pH-responsive silica-metal-organic framework hybrid nanoparticle for the delivery of hydrophilic drugs, nucleic acids, and CRISPR-Cas9 genome-editing machineries, J Control Release, 324, 194, 10.1016/j.jconrel.2020.04.052 · DOI
  99. Noureddine, 2020, Engineering of monosized lipid-coated mesoporous silica nanoparticles for CRISPR delivery, Acta Biomater, 114, 358, 10.1016/j.actbio.2020.07.027 · DOI
  100. Maxwell, 2004, Adenoviral-mediated expression of Pcsk9 in mice results in a low-density lipoprotein receptor knockout phenotype, Proc Natl Acad Sci U S A, 101, 7100, 10.1073/pnas.0402133101 · DOI · Paper page
  101. Abifadel, 2003, Mutations in PCSK9 cause autosomal dominant hypercholesterolemia, Nat Genet, 34, 154, 10.1038/ng1161 · DOI · Paper page
  102. Blau, 2010, Phenylketonuria, Lancet, 376, 1417, 10.1016/S0140-6736(10)60961-0 · DOI
  103. Tuchman, 1992, The clinical, biochemical, and molecular spectrum of ornithine transcarbamylase deficiency, J Lab Clin Med, 120, 836
  104. Tuchman, 1998, The biochemical and molecular spectrum of ornithine transcarbamylase deficiency, J Inherit Metab Dis, 21, 40, 10.1023/A:1005353407220 · DOI
  105. Deng, 2020, Codelivery of CRISPR-Cas9 and chlorin e6 for spatially controlled tumor-specific gene editing with synergistic drug effects, Sci Adv, 6, 10.1126/sciadv.abb4005 · DOI
  106. Dorand, 2016, Cdk 5 disruption attenuates tumor PD-L1 expression and promotes antitumor immunity, Science, 353, 399, 10.1126/science.aae0477 · DOI
  107. Finck, 2020, Cancer immunotherapy comes of age and looks for maturity, Nat Commun, 11, 3325, 10.1038/s41467-020-17140-5 · DOI
  108. Riley, 2019, Delivery technologies for cancer immunotherapy, Nat Rev Drug Discov, 18, 175, 10.1038/s41573-018-0006-z · DOI
  109. Gao, 2015, The dystrophin complex: structure, function, and implications for therapy, Comp Physiol, 5, 1223, 10.1002/j.2040-4603.2015.tb00638.x · DOI
  110. Gee, 2020, Extracellular nanovesicles for packaging of CRISPR-Cas9 protein and sgRNA to induce therapeutic exon skipping, Nat Commun, 11, 1334, 10.1038/s41467-020-14957-y · DOI
  111. Wehrens, 2005, Intracellular calcium release and cardiac disease, Annu Rev Physiol, 67, 69, 10.1146/annurev.physiol.67.040403.114521 · DOI
  112. Pan, 2018, In vivo Ryr2 editing corrects catecholaminergic polymorphic ventricular tachycardia, Circ Res, 123, 953, 10.1161/CIRCRESAHA.118.313369 · DOI
  113. Wang, 2017, A systemic view of Alzheimer disease—insights from amyloid-beta metabolism beyond the brain, Nat Rev Neurol, 13, 612, 10.1038/nrneurol.2017.111 · DOI · Paper page
  114. Yan, 2014, Targeting the beta secretase BACE1 for Alzheimer's disease therapy, Lancet Neurol, 13, 319, 10.1016/S1474-4422(13)70276-X · DOI
  115. Vassar, 2004, BACE1: the beta-secretase enzyme in Alzheimer's disease, J Mol Neurosci, 23, 105, 10.1385/JMN:23:1-2:105 · DOI
  116. Bates, 2015, Huntington disease, Nat Rev Dis Prim, 1, 15005, 10.1038/nrdp.2015.5 · DOI · Paper page
  117. Ekman, 2019, CRISPR-Cas9-mediated genome editing increases lifespan and improves motor deficits in a Huntington's disease mouse model, Mol Ther Nucleic Acids, 17, 829, 10.1016/j.omtn.2019.07.009 · DOI
  118. Hardiman, 2017, Amyotrophic lateral sclerosis, Nat Rev Dis Prim, 3, 17085, 10.1038/nrdp.2017.85 · DOI
  119. Brown, 2017, Amyotrophic lateral sclerosis, N Engl J Med, 377, 1602, 10.1056/NEJMra1603471 · DOI
  120. Lim, 2020, Treatment of a mouse model of ALS by in vivo base editing, Mol Ther, 28, 1177, 10.1016/j.ymthe.2020.01.005 · DOI
  121. Molday, 2012, X-linked juvenile retinoschisis: clinical diagnosis, genetic analysis, and molecular mechanisms, Prog Retin Eye Res, 31, 195, 10.1016/j.preteyeres.2011.12.002 · DOI
  122. Strupaite, 2018, X-linked juvenile retinoschisis: phenotypic and genetic characterization, Int J Ophthalmol, 11, 1875
  123. Nishiguchi, 2020, Single AAV-mediated mutation replacement genome editing in limited number of photoreceptors restores vision in mice, Nat Commun, 11, 482, 10.1038/s41467-019-14181-3 · DOI
  124. Kent, 2015, Mechanism of microhomology-mediated end-joining promoted by human DNA polymerase theta, Nat Struct Mol Biol, 22, 230, 10.1038/nsmb.2961 · DOI
  125. Truong, 2013, Microhomology-mediated end joining and homologous recombination share the initial end resection step to repair DNA double-strand breaks in mammalian cells, Proc Natl Acad Sci U S A, 110, 7720, 10.1073/pnas.1213431110 · DOI
  126. Lamb, 2013, Evolution of phototransduction, vertebrate photoreceptors and retina, Prog Retin Eye Res, 36, 52, 10.1016/j.preteyeres.2013.06.001 · DOI
  127. Gao, 2018, Treatment of autosomal dominant hearing loss by in vivo delivery of genome editing agents, Nature, 553, 217, 10.1038/nature25164 · DOI · Paper page
  128. György, 2019, Allele-specific gene editing prevents deafness in a model of dominant progressive hearing loss, Nat Med, 25, 1123, 10.1038/s41591-019-0500-9 · DOI
  129. Dash, 2019, Sequential LASER ART and CRISPR treatments eliminate HIV-1 in a subset of infected humanized mice, Nat Commun, 10, 2753, 10.1038/s41467-019-10366-y · DOI · Paper page
  130. Mancuso, 2020, CRISPR based editing of SIV proviral DNA in ART treated non-human primates, Nat Commun, 11, 6065, 10.1038/s41467-020-19821-7 · DOI
  131. Komor, 2017, CRISPR-based technologies for the manipulation of eukaryotic genomes, Cell, 168, 20, 10.1016/j.cell.2016.10.044 · DOI
  132. Caplan, 2015, No time to waste—the ethical challenges created by CRISPR: CRISPR/Cas, being an efficient, simple, and cheap technology to edit the genome of any organism, raises many ethical and regulatory issues beyond the use to manipulate human germ line cells, EMBO Rep, 16, 1421, 10.15252/embr.201541337 · DOI
  133. He, 2020, Progress in systemic co-delivery of microRNAs and chemotherapeutics for cancer treatment by using lipid-based nanoparticles, Ther Deliv, 11, 591, 10.4155/tde-2020-0052 · DOI
  134. Klinman, 1997, Contribution of CpG motifs to the immunogenicity of DNA vaccines, J Immunol, 158, 3635, 10.4049/jimmunol.158.8.3635 · DOI · Paper page
  135. de Wolf, 2008, Plasmid CpG depletion improves degree and duration of tumor gene expression after intravenous administration of polyplexes, Pharm Res, 25, 1654, 10.1007/s11095-008-9558-7 · DOI
  136. Shmakov, 2015, Discovery and functional characterization of diverse class 2 CRISPR-Cas systems, Mol Cell, 60, 385, 10.1016/j.molcel.2015.10.008 · DOI · Paper page
  137. Harris, 2003, Effect of pegylation on pharmaceuticals, Nat Rev Drug Discov, 2, 214, 10.1038/nrd1033 · DOI · Paper page
  138. Hoffman, 2020, Three significant highlights of controlled drug delivery over the past 55 years: PEGylation, ADCs, and EPR, Adv Drug Deliv Rev, 158, 2, 10.1016/j.addr.2020.05.013 · DOI
  139. Suk, 2016, PEGylation as a strategy for improving nanoparticle-based drug and gene delivery, Adv Drug Deliv Rev, 99, 28, 10.1016/j.addr.2015.09.012 · DOI · Paper page
  140. Ishida, 2005, Accelerated blood clearance of PEGylated liposomes following preceding liposome injection: effects of lipid dose and PEG surface-density and chain length of the first-dose liposomes, J Control Release, 105, 305, 10.1016/j.jconrel.2005.04.003 · DOI
  141. Wang, 2005, Influence of the physicochemical properties of liposomes on the accelerated blood clearance phenomenon in rats, J Control Release, 104, 91, 10.1016/j.jconrel.2005.01.008 · DOI
  142. Zhang, 2017, Cytosolic co-delivery of miRNA-34a and docetaxel with core-shell nanocarriers via caveolae-mediated pathway for the treatment of metastatic breast cancer, Sci Rep, 7, 46186, 10.1038/srep46186 · DOI
  143. Zhao, 2020, Targeting strategies for tissue-specific drug delivery, Cell, 181, 151, 10.1016/j.cell.2020.02.001 · DOI
  144. Mitchell, 2021, Engineering precision nanoparticles for drug delivery, Nat Rev Drug Discov, 20, 101, 10.1038/s41573-020-0090-8 · DOI · Paper page
  145. Xu, 1996, Mechanism of DNA release from cationic liposome/DNA complexes used in cell transfection, Biochemistry, 35, 5616, 10.1021/bi9602019 · DOI
  146. Wang, 2013, A window onto siRNA delivery, Nat Biotechnol, 31, 611, 10.1038/nbt.2634 · DOI
  147. Zhang, 2010, Multifunctional peptide-PEG intercalating conjugates: programmatic of gene delivery to the blood–brain barrier, Pharm Res, 27, 2528, 10.1007/s11095-010-0256-x · DOI
  148. Li, 2004, GALA: a designed synthetic pH-responsive amphipathic peptide with applications in drug and gene delivery, Adv Drug Deliv Rev, 56, 967, 10.1016/j.addr.2003.10.041 · DOI · Paper page
  149. Zhang, 2010, Short biodegradable polyamines for gene delivery and transfection of brain capillary endothelial cells, J Control Release, 143, 359, 10.1016/j.jconrel.2010.01.020 · DOI
  150. Slone, 2020, The special considerations of gene therapy for mitochondrial diseases, NPJ Genom Med, 5, 7, 10.1038/s41525-020-0116-5 · DOI
  151. Wienert, 2020, CRISPR off-target detection with DISCOVER-seq, Nat Protoc, 15, 1775, 10.1038/s41596-020-0309-5 · DOI
  152. Lazzarotto, 2018, Defining CRISPR-Cas9 genome-wide nuclease activities with CIRCLE-seq, Nat Protoc, 13, 2615, 10.1038/s41596-018-0055-0 · DOI
  153. Kim, 2021, Profiling Genome-wide specificity of CRISPR-Cas9 using digenome-seq, Methods Mol Biol, 2162, 233, 10.1007/978-1-0716-0687-2_13 · DOI
  154. Akcakaya, 2018, In vivo CRISPR editing with no detectable genome-wide off-target mutations, Nature, 561, 416, 10.1038/s41586-018-0500-9 · DOI
  155. 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
  156. Tsai, 2014, Dimeric CRISPR RNA-guided FokI nucleases for highly specific genome editing, Nat Biotechnol, 32, 569, 10.1038/nbt.2908 · DOI · Paper page
  157. Aschenbrenner, 2020, Coupling Cas9 to artificial inhibitory domains enhances CRISPR-Cas9 target specificity, Sci Adv, 6, 10.1126/sciadv.aay0187 · DOI
  158. 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
  159. Cai, 2021, Spatiotemporal delivery of CRISPR/Cas9 genome editing machinery using stimuli-responsive vehicles, Angew Chem Int Ed Engl, 60, 8596, 10.1002/anie.202005644 · DOI
  160. Chen, 2020, Near-infrared optogenetic engineering of photothermal nanoCRISPR for programmable genome editing, Proc Natl Acad Sci U S A, 117, 2395, 10.1073/pnas.1912220117 · DOI

Cited by

Sorted by citations

See all 210

Metadata

Authors
Behr, M., Zhou, J., Xu, B., & Zhang, H.
Venue
Acta Pharmaceutica Sinica B
Publisher
Elsevier BV
Published
2021-08
Volume
11
Issue
8
Pages
2150-2171
ISSN
2211-3835
Cited by
210
References
39
Language
en

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