Synthetic multi-layer nanoparticles for CRISPR-Cas9 genome editing
Tài liệu tham khảo
Cong, 2013, Multiplex genome engineering using CRISPR/Cas systems, Science, 339, 819, 10.1126/science.1231143
Jinek, 2013, RNA-programmed genome editing in human cells, Elife, 2, 10.7554/eLife.00471
Mali, 2013, RNA-guided human genome engineering via Cas9, Science, 339, 823, 10.1126/science.1232033
Doudna, 2014, The new frontier of genome engineering with CRISPR-Cas9, Science, 346, 1258096, 10.1126/science.1258096
Stoddard, 2011, Homing endonucleases: from microbial genetic invaders to reagents for targeted DNA modification, Structure, 19, 7, 10.1016/j.str.2010.12.003
Urnov, 2010, Genome editing with engineered zinc finger nucleases, Nat. Rev. Genet., 11, 636, 10.1038/nrg2842
Joung, 2013, TALENs: a widely applicable technology for targeted genome editing, Nat. Rev. Mol. Cell Biol., 14, 49, 10.1038/nrm3486
Wan, 2019, Material solutions for delivery of CRISPR/Cas-based genome editing tools: current status and future outlook, Mater. Today, 26, 40, 10.1016/j.mattod.2018.12.003
Travis, 2015, Genetic Engineering. Germline editing dominates DNA summit, Science, 350, 1299, 10.1126/science.350.6266.1299
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
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
Bolotin, 2005, Clustered regularly interspaced short palindrome repeats (CRISPRs) have spacers of extrachromosomal origin, Microbiology, 151, 2551, 10.1099/mic.0.28048-0
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
Pourcel, 2005, CRISPR elements in Yersinia pestis acquire new repeats by preferential uptake of bacteriophage DNA, and provide additional tools for evolutionary studies, Microbiology, 151, 653, 10.1099/mic.0.27437-0
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
Makarova, 2006, A putative RNA-interference-based immune system in prokaryotes: computational analysis of the predicted enzymatic machinery, functional analogies with eukaryotic RNAi, and hypothetical mechanisms of action, Biol. Direct, 1, 7, 10.1186/1745-6150-1-7
Barrangou, 2007, CRISPR provides acquired resistance against viruses in prokaryotes, Science, 315, 1709, 10.1126/science.1138140
Brouns, 2008, Small CRISPR RNAs guide antiviral defense in prokaryotes, Science, 321, 960, 10.1126/science.1159689
Marraffini, 2008, CRISPR interference limits horizontal gene transfer in staphylococci by targeting DNA, Science, 322, 1843, 10.1126/science.1165771
Deltcheva, 2011, CRISPR RNA maturation by trans-encoded small RNA and host factor RNase III, Nature, 471, 602, 10.1038/nature09886
Jinek, 2012, A programmable dual-RNA-guided DNA endonuclease in adaptive bacterial immunity, Science, 337, 816, 10.1126/science.1225829
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
Lin, 2014, Enhanced homology-directed human genome engineering by controlled timing of CRISPR/Cas9 delivery, Elife, 3, 10.7554/eLife.04766
Kim, 2014, Highly efficient RNA-guided genome editing in human cells via delivery of purified Cas9 ribonucleoproteins, Genome Res., 24, 1012, 10.1101/gr.171322.113
Hsu, 2013, DNA targeting specificity of RNA-guided Cas9 nucleases, Nat. Biotechnol., 31, 827, 10.1038/nbt.2647
Davis, 2015, Small molecule-triggered Cas9 protein with improved genome-editing specificity, Nat. Chem. Biol., 11, 316, 10.1038/nchembio.1793
Hemphill, 2015, Optical control of CRISPR/Cas9 gene editing, J. Am. Chem. Soc., 137, 5642, 10.1021/ja512664v
Slaymaker, 2016, Rationally engineered Cas9 nucleases with improved specificity, Science, 351, 84, 10.1126/science.aad5227
Kleinstiver, 2016, High-fidelity CRISPR-Cas9 nucleases with no detectable genome-wide off-target effects, Nature, 529, 490, 10.1038/nature16526
Fu, 2014, Improving CRISPR-Cas nuclease specificity using truncated guide RNAs, Nat. Biotechnol., 32, 279, 10.1038/nbt.2808
Yin, 2017, Delivery technologies for genome editing, Nat. Rev. Drug Discov., 16, 387, 10.1038/nrd.2016.280
Cornu, 2017, Refining strategies to translate genome editing to the clinic, Nat. Med., 23, 415, 10.1038/nm.4313
Tong, 2019, Engineered materials for in vivo delivery of genome-editing machinery, Nat. Rev. Mater., 4, 726, 10.1038/s41578-019-0145-9
Wang, 2017, CRISPR/Cas9-based genome editing for disease modeling and therapy: challenges and opportunities for nonviral delivery, Chem. Rev., 117, 9874, 10.1021/acs.chemrev.6b00799
Yu, 2017, Nrl knockdown by AAV-delivered CRISPR/Cas9 prevents retinal degeneration in mice, Nat. Commun., 8, 14716, 10.1038/ncomms14716
Niu, 2014, Generation of gene-modified cynomolgus monkey via Cas9/RNA-mediated gene targeting in one-cell embryos, Cell, 156, 836, 10.1016/j.cell.2014.01.027
Xue, 2014, CRISPR-mediated direct mutation of cancer genes in the mouse liver, Nature, 514, 380, 10.1038/nature13589
Li, 2018, Non-viral delivery systems for CRISPR/Cas9-based genome editing: challenges and opportunities, Biomaterials, 171, 207, 10.1016/j.biomaterials.2018.04.031
Lehrman, 1999, Virus treatment questioned after gene therapy death, Nature, 401, 517, 10.1038/43977
Sun, 2003, Immune responses to adeno-associated virus and its recombinant vectors, Gene Ther., 10, 964, 10.1038/sj.gt.3302039
Pack, 2005, Design and development of polymers for gene delivery, Nat. Rev. Drug Discov., 4, 581, 10.1038/nrd1775
Li, 2015, Challenges in CRISPR/Cas9 delivery: potential roles of nonviral vectors, Hum. Gene Ther., 26, 452, 10.1089/hum.2015.069
Zhang, 2017, Lipid nanoparticle-mediated efficient delivery of CRISPR/Cas9 for tumor therapy, NPG Asia Mater., 9, 10.1038/am.2017.185
Ran, 2015, In vivo genome editing using Staphylococcus aureus Cas9, Nature, 520, 186, 10.1038/nature14299
Hughes, 2009, Immunogenicity of intrathecal plasmid gene delivery: cytokine release and effects on transgene expression, J. Gene Med., 11, 782, 10.1002/jgm.1364
Shen, 2014, Efficient genome modification by CRISPR-Cas9 nickase with minimal off-target effects, Nat. Methods, 11, 399, 10.1038/nmeth.2857
Chang, 2013, Genome editing with RNA-guided Cas9 nuclease in zebrafish embryos, Cell Res., 23, 465, 10.1038/cr.2013.45
Woo, 2015, DNA-free genome editing in plants with preassembled CRISPR-Cas9 ribonucleoproteins, Nat. Biotechnol., 33, 1162, 10.1038/nbt.3389
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
Zhang, 2019, Triple-targeting delivery of CRISPR/Cas9 to reduce the risk of cardiovascular diseases, Angew. Chem. Int. Ed., 58, 12404, 10.1002/anie.201903618
Wang, 2017, Genome editing for cancer therapy: delivery of Cas9 protein/sgRNA plasmid via a gold nanocluster/lipid core-shell nanocarrier, Adv. Sci., 4, 1700175, 10.1002/advs.201700175
Hwang, 2013, Efficient genome editing in zebrafish using a CRISPR-Cas system, Nat. Biotechnol., 31, 227, 10.1038/nbt.2501
Yan, 2014, Generation of multi-gene knockout rabbits using the Cas9/gRNA system, Cell Regener., 3, 12, 10.1186/2045-9769-3-12
June, 2018, CAR T cell immunotherapy for human cancer, Science, 359, 1361, 10.1126/science.aar6711
Cyranoski, 2016, CRISPR gene-editing tested in a person for the first time, Nature, 539, 479, 10.1038/nature.2016.20988
Lu, 2018, A phase I trial of PD-1 deficient engineered T cells with CRISPR/Cas9 in patients with advanced non-small cell lung cancer, J. Clin. Oncol., 36, 3050, 10.1200/JCO.2018.36.15_suppl.3050
Maeder, 2019, Development of a gene-editing approach to restore vision loss in Leber congenital amaurosis type 10, Nat. Med., 25, 229, 10.1038/s41591-018-0327-9
Liu, 2013, PEGylation and zwitterionization: pros and cons in the renal clearance and tumor targeting of near-IR-emitting gold nanoparticles, Angew. Chem. Int. Ed., 52, 12572, 10.1002/anie.201304465
Du, 2017, Glomerular barrier behaves as an atomically precise bandpass filter in a sub-nanometre regime, Nat. Nanotechnol., 12, 1096, 10.1038/nnano.2017.170
Lei, 2017, Gold nanoclusters-assisted delivery of NGF siRNA for effective treatment of pancreatic cancer, Nat. Commun., 8, 15130, 10.1038/ncomms15130
Vankayala, 2015, Nucleus-targeting gold nanoclusters for simultaneous in vivo fluorescence imaging, gene delivery, and NIR-Light activated photodynamic therapy, Adv. Funct. Mater., 25, 5934, 10.1002/adfm.201502650
Xia, 2016, Effect of surface properties on liposomal siRNA delivery, Biomaterials, 79, 56, 10.1016/j.biomaterials.2015.11.056
Tseng, 2009, Lipid-based systemic delivery of siRNA, Adv. Drug Deliv. Rev., 61, 721, 10.1016/j.addr.2009.03.003
Betteridge, 2013, Cardiovascular endocrinology in 2012: PCSK9-an exciting target for reducing LDL-cholesterol levels, Nat. Rev. Endocrinol., 9, 76, 10.1038/nrendo.2012.254
Raal, 2015, Inhibition of PCSK9 with evolocumab in homozygous familial hypercholesterolaemia (TESLA Part B): a randomised, double-blind, placebo-controlled trial, Lancet, 385, 341, 10.1016/S0140-6736(14)61374-X
Robinson, 2015, Efficacy and safety of alirocumab in reducing lipids and cardiovascular events, N. Engl. J. Med., 372, 1489, 10.1056/NEJMoa1501031
Hu, 2013, A highly efficient synthetic vector: nonhydrodynamic delivery of DNA to hepatocyte nuclei in vivo, ACS Nano, 7, 5376, 10.1021/nn4012384
Elahi, 2018, Recent biomedical applications of gold nanoparticles: a review, Talanta, 184, 537, 10.1016/j.talanta.2018.02.088
Mout, 2017, Direct cytosolic delivery of CRISPR/Cas9-ribonucleoprotein for efficient gene editing, ACS Nano, 11, 2452, 10.1021/acsnano.6b07600
Mout, 2017, Cytosolic and nuclear delivery of CRISPR/Cas9-ribonucleoprotein for gene editing using arginine functionalized gold nanoparticles, Bio-Protoc., 7, 10.21769/BioProtoc.2586
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
Lee, 2018, Nanoparticle delivery of CRISPR into the brain rescues a mouse model of fragile X syndrome from exaggerated repetitive behaviours, Nat. Biomed. Eng., 2, 497, 10.1038/s41551-018-0252-8
Shahbazi, 2019, Targeted homology-directed repair in blood stem and progenitor cells with CRISPR nanoformulations, Nat. Mater., 18, 1124, 10.1038/s41563-019-0385-5
Wirth, 2014, Plasmonically enhanced electron escape from gold nanoparticles and their polarization-dependent excitation transfer along DNA nanowires, Nano Lett., 14, 3809, 10.1021/nl5009184
Zhang, 2012, Mesoporous silica-coated gold nanorods as a light-mediated multifunctional theranostic platform for cancer treatment, Adv. Mater., 24, 1418, 10.1002/adma.201104714
Wang, 2018, Thermo-triggered release of CRISPR-Cas9 system by lipid-encapsulated gold nanoparticles for tumor therapy, Angew. Chem. Int. Ed., 57, 1491, 10.1002/anie.201708689
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
Fitzgerald, 2014, Effect of an RNA interference drug on the synthesis of proprotein convertase subtilisin/kexin type 9 (PCSK9) and the concentration of serum LDL cholesterol in healthy volunteers: a randomised, single-blind, placebo-controlled, phase 1 trial, Lancet, 383, 60, 10.1016/S0140-6736(13)61914-5
Coelho, 2013, Safety and efficacy of RNAi therapy for transthyretin amyloidosis, N. Engl. J. Med., 369, 819, 10.1056/NEJMoa1208760
Sun, 2015, Tunable rigidity of (polymeric core)–(lipid shell) nanoparticles for regulated cellular uptake, Adv. Mater., 27, 1402, 10.1002/adma.201404788
Zhang, 2015, Microfluidic synthesis of hybrid nanoparticles with controlled lipid layers: understanding flexibility-regulated cell-nanoparticle interaction, ACS Nano, 9, 9912, 10.1021/acsnano.5b05792
Feng, 2017, One-Step microfluidic synthesis of nanocomplex with tunable rigidity and acid-switchable surface charge for overcoming drug resistance, Small, 13, 1603109, 10.1002/smll.201603109
Alabi, 2013, Multiparametric approach for the evaluation of lipid nanoparticles for siRNA delivery, Proc. Natl. Acad. Sci. U. S. A., 110, 12881, 10.1073/pnas.1306529110
Dong, 2014, Lipopeptide nanoparticles for potent and selective siRNA delivery in rodents and nonhuman primates, Proc. Natl. Acad. Sci. U. S. A., 111, 3955, 10.1073/pnas.1322937111
Whitehead, 2014, Degradable lipid nanoparticles with predictable in vivo siRNA delivery activity, Nat. Commun., 5, 4277, 10.1038/ncomms5277
Chen, 2014, Upconversion nanoparticles: design, nanochemistry, and applications in theranostics, Chem. Rev., 114, 5161, 10.1021/cr400425h
Pan, 2019, Near-infrared upconversion–activated CRISPR-Cas9 system: a remote-controlled gene editing platform, Sci. Adv., 5, 10.1126/sciadv.aav7199
Fenton, 2018, Advances in biomaterials for drug delivery, Adv. Mater., 30, 1705328, 10.1002/adma.201705328
Tang, 2018, Tumor specific and renal excretable star-like triblock polymer-doxorubicin conjugates for safe and efficient anticancer therapy, Biomacromolecules, 19, 2849, 10.1021/acs.biomac.8b00425
Sun, 2017, Rational design of cancer nanomedicine: nanoproperty integration and synchronization, Adv. Mater., 29, 1606628, 10.1002/adma.201606628
Wang, 2018, Enhancing the in vitro and in vivo stabilities of polymeric nucleic acid delivery nanosystems, Bioconjug. Chem., 30, 325, 10.1021/acs.bioconjchem.8b00749
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
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
Lu, 2011, Ionic polypeptides with unusual helical stability, Nat. Commun., 2, 206, 10.1038/ncomms1209
Gabrielson, 2012, Reactive and bioactive cationic alpha-helical polypeptide template for nonviral gene delivery, Angew. Chem. Int. Ed., 51, 1143, 10.1002/anie.201104262
Yin, 2013, Supramolecular self-assembled nanoparticles mediate oral delivery of therapeutic TNF-alpha siRNA against systemic inflammation, Angew. Chem. Int. Ed., 52, 5757, 10.1002/anie.201209991
Wang, 2018, Nonviral gene editing via CRISPR/Cas9 delivery by membrane-disruptive and endosomolytic helical polypeptide, Proc. Natl. Acad. Sci. U. S. A., 115, 4903, 10.1073/pnas.1712963115
Liu, 2019, Multistage delivery nanoparticle facilitates efficient CRISPR/dCas9 activation and tumor growth suppression in vivo, Adv. Sci., 6, 1801423, 10.1002/advs.201801423
Liu, 2019, NanoRNP overcomes tumor heterogeneity in cancer treatment, Nano Lett., 19, 7662, 10.1021/acs.nanolett.9b02501
Zhang, 2019, Dual-locking nanoparticles disrupt the PD-1/PD-L1 pathway for efficient cancer immunotherapy, Adv. Mater., 31, 1905751, 10.1002/adma.201905751
He, 2019, Multifunctional vector for delivery of genome editing plasmid targeting β-catenin to remodulate cancer cell properties, ACS Appl. Mater. Interfaces, 11, 226, 10.1021/acsami.8b17481
Liu, 2018, Reversal of tumor malignization and modulation of cell behaviors through genome editing mediated by a multi-functional nanovector, Nanoscale, 10, 21209, 10.1039/C8NR07321J
Wang, 2017, Hyaluronic acid modification of RNase A and its intracellular delivery using lipid-like nanoparticles, J. Control. Release, 263, 39, 10.1016/j.jconrel.2017.01.037
Li, 2017, Artificial virusdelivers CRISPR-Cas9 system for genome editing of cells in mice, ACS Nano, 11, 95, 10.1021/acsnano.6b04261
Chang, 2018, Integrating combinatorial lipid nanoparticle and chemically modified protein for intracellular delivery and genome editing, Acc. Chem. Res., 52, 665, 10.1021/acs.accounts.8b00493
Wang, 2014, Combinatorially designed lipid-like nanoparticles for intracellular delivery of cytotoxic protein for cancer therapy, Angew. Chem. Int. Ed., 53, 2893, 10.1002/anie.201311245
Altınoglu, 2015, Combinatorial library strategies for synthesis of cationic lipid-like nanoparticles and their potential medical applications, Nanomedicine, 10, 643, 10.2217/nnm.14.192
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
Akinc, 2008, A combinatorial library of lipid-like materials for delivery of RNAi therapeutics, Nat. Biotechnol., 26, 561, 10.1038/nbt1402
Li, 2018, Combinatorial library of chalcogen-containing lipidoids for intracellular delivery of genome-editing proteins, Biomaterials, 178, 652, 10.1016/j.biomaterials.2018.03.011
Liu, 2019, Fast and efficient CRISPR/Cas9 genome editing in vivo enabled by bioreducible lipid and messenger RNA nanoparticles, Adv. Mater., 31, 1902575, 10.1002/adma.201902575
Abifadel, 2003, Mutations in PCSK9 cause autosomal dominant hypercholesterolemia, Nat. Genet., 34, 154, 10.1038/ng1161
Li, 2018, Intracellular delivery of His-tagged genome-editing proteins enabled by nitrilotriacetic acid–containing lipidoid nanoparticles, Adv. Healthcare Mater., 8, 1800996, 10.1002/adhm.201800996
Makadia, 2011, Poly lactic-co-glycolic acid (PLGA) as biodegradable controlled drug delivery carrier, Polymers (Basel), 3, 1377, 10.3390/polym3031377
Fredenberg, 2011, The mechanisms of drug release in poly(lactic-co-glycolic acid)-based drug delivery systems-a review, Int. J. Pharm., 415, 34, 10.1016/j.ijpharm.2011.05.049
Li, 2018, Microfluidics for producing poly (lactic-co-glycolic acid)-based pharmaceutical nanoparticles, Adv. Drug Deliv. Rev., 128, 101, 10.1016/j.addr.2017.12.015
Pustulka, 2013, Flash nanoprecipitation: particle structure and stability, Mol. Pharm., 10, 4367, 10.1021/mp400337f
Fan, 2018, Cationic lipid-assisted nanoparticles for delivery of mRNA cancer vaccine, Biomater. Sci., 6, 3009, 10.1039/C8BM00908B
Xu, 2015, Targeting glucose uptake with siRNA-based nanomedicine for cancer therapy, Biomaterials, 51, 1, 10.1016/j.biomaterials.2015.01.068
Liu, 2014, Triple negative breast cancer therapy with CDK1 siRNA delivered by cationic lipid assisted PEG-PLA nanoparticles, J. Control. Release, 192, 114, 10.1016/j.jconrel.2014.07.001
Yang, 2011, Systemic delivery of siRNA with cationic lipid assisted PEG-PLA nanoparticles for cancer therapy, J. Control. Release, 156, 203, 10.1016/j.jconrel.2011.07.035
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
Broz, 2016, Inflammasomes: mechanism of assembly, regulation and signalling, Nat. Rev. Immunol., 16, 407, 10.1038/nri.2016.58
Vandanmagsar, 2011, The NLRP3 inflammasome instigates obesity-induced inflammation and insulin resistance, Nat. Med., 17, 179, 10.1038/nm.2279
Liu, 2018, Optimization of lipid-assisted nanoparticle for disturbing neutrophils-related inflammation, Biomaterials, 172, 92, 10.1016/j.biomaterials.2018.04.052
Wang, 2016, Surface charge critically affects tumor penetration and therapeutic efficacy of cancer nanomedicines, Nano Today, 11, 133, 10.1016/j.nantod.2016.04.008
Yan, 2015, Dopamine controls systemic inflammation through inhibition of NLRP3 inflammasome, Cell, 160, 62, 10.1016/j.cell.2014.11.047
Wen, 2011, Fatty acid-induced NLRP3-ASC inflammasome activation interferes with insulin signaling, Nat. Immunol., 12, 408, 10.1038/ni.2022
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
Elgueta, 2009, Molecular mechanism and function of CD40/CD40L engagement in the immune system, Immunol. Rev., 229, 152, 10.1111/j.1600-065X.2009.00782.x
Kinnear, 2013, Costimulation blockade: current perspectives and implications for therapy, Transplantation, 95, 527, 10.1097/TP.0b013e31826d4672
Shen, 2014, Conditional knockouts generated by engineered CRISPR-Cas9 endonuclease reveal the roles of coronin in C. elegans neural development, Dev. Cell, 30, 625, 10.1016/j.devcel.2014.07.017
Wang, 2015, Egg cell-specific promoter-controlled CRISPR/Cas9 efficiently generates homozygous mutants for multiple target genes in Arabidopsis in a single generation, Genome Biol., 16, 144, 10.1186/s13059-015-0715-0
Luo, 2018, Macrophage-specific in vivo gene editing using cationic lipid-assisted polymeric nanoparticles, ACS Nano, 12, 994, 10.1021/acsnano.7b07874
Cai, 2016, Quantitative study of effects of free cationic chains on gene transfection in different intracellular stages, J. Control. Release, 238, 71, 10.1016/j.jconrel.2016.07.031
Yue, 2011, Revisit complexation between DNA and polyethylenimine-effect of length of free polycationic chains on gene transfection, J. Control. Release, 152, 143, 10.1016/j.jconrel.2011.03.020
Nelson, 2016, Engineering delivery vehicles for genome editing, Annu. Rev. Chem. Biomol. Eng., 7, 637, 10.1146/annurev-chembioeng-080615-034711
Behzadi, 2017, Cellular uptake of nanoparticles: journey inside the cell, Chem. Soc. Rev., 46, 4218, 10.1039/C6CS00636A
Ganas, 2014, Biodegradable capsules as non-viral vectors for in vitro delivery of PEI/siRNA polyplexes for efficient gene silencing, J. Control. Release, 196, 132, 10.1016/j.jconrel.2014.10.006
Givens, 2018, Nanoparticle-based delivery of CRISPR/Cas9 genome-editing therapeutics, AAPS J., 20, 108, 10.1208/s12248-018-0267-9
Neu, 2005, Recent advances in rational gene transfer vector design based on poly(ethylene imine) and its derivatives, J. Gene Med., 7, 992, 10.1002/jgm.773
Kang, 2017, Nonviral genome editing based on a polymer-derivatized CRISPR nanocomplex for targeting bacterial pathogens and antibiotic resistance, Bioconjug. Chem., 28, 957, 10.1021/acs.bioconjchem.6b00676
Zhang, 2018, Cationic polymer-mediated CRISPR/Cas9 plasmid delivery for genome editing, Macromol. Rapid Commun., 40, 1800068, 10.1002/marc.201800068
Ping, 2013, FGFR-targeted gene delivery mediated by supramolecular assembly between beta-cyclodextrin-crosslinked PEI and redox-sensitive PEG, Biomaterials, 34, 6482, 10.1016/j.biomaterials.2013.03.071
Hu, 2015, Engineering nanoparticle-coated bacteria as oral DNA vaccines for cancer immunotherapy, Nano Lett., 15, 2732, 10.1021/acs.nanolett.5b00570
Alvarez, 2014, A phase II trial of intraperitoneal EGEN-001, an IL-12 plasmid formulated with PEG–PEI–cholesterol lipopolymer in the treatment of persistent or recurrent epithelial ovarian, fallopian tube or primary peritoneal cancer: a gynecologic oncology group study, Gynecol. Oncol., 133, 433, 10.1016/j.ygyno.2014.03.571
Fewell, 2005, Synthesis and application of a non-viral gene delivery system for immunogene therapy of cancer, J. Control. Release, 109, 288, 10.1016/j.jconrel.2005.09.024
Liang, 2017, Tumor cell-targeted delivery of CRISPR/Cas9 by aptamer-functionalized lipopolymer for therapeutic genome editing of VEGFA in osteosarcoma, Biomaterials, 147, 68, 10.1016/j.biomaterials.2017.09.015
Yang, 2015, Surface-engineered dendrimers in gene delivery, Chem. Rev., 115, 5274, 10.1021/cr500542t
Kretzmann, 2017, Synthetically controlling dendrimer flexibility improves delivery of large plasmid DNA, Chem. Sci., 8, 2923, 10.1039/C7SC00097A
Chang, 2017, Rational design of a polymer with robust efficacy for intracellular protein and peptide delivery, Nano Lett., 17, 1678, 10.1021/acs.nanolett.6b04955
Liu, 2019, A boronic acid–rich dendrimer with robust and unprecedented efficiency for cytosolic protein delivery and CRISPR-Cas9 gene editing, Sci. Adv., 5, 10.1126/sciadv.aaw8922
Khutoryanskiy, 2018, Beyond PEGylation: alternative surface-modification of nanoparticles with mucus-inert biomaterials, Adv. Drug Deliv. Rev., 124, 140, 10.1016/j.addr.2017.07.015
Mi, 2014, Integrated antimicrobial and nonfouling zwitterionic polymers, Angew. Chem. Int. Ed., 53, 1746, 10.1002/anie.201304060
Schlenoff, 2014, Zwitteration: coating surfaces with zwitterionic functionality to reduce nonspecific adsorption, Langmuir, 30, 9625, 10.1021/la500057j
Miller, 2017, Non-viral CRISPR/Cas gene editing in vitro and in vivo enabled by synthetic nanoparticle co-delivery of Cas9 mRNA and sgRNA, Angew. Chem. Int. Ed., 56, 1059, 10.1002/anie.201610209
Tabebordbar, 2016, In vivo gene editing in dystrophic mouse muscle and muscle stem cells, Science, 351, 407, 10.1126/science.aad5177
Yue, 2018, Graphene oxide-mediated Cas9/sgRNA delivery for efficient genome editing, Nanoscale, 10, 1063, 10.1039/C7NR07999K
Zhou, 2018, Enhanced cytosolic delivery and release of CRISPR/Cas9 by black phosphorus nanosheets for genome editing, Angew. Chem. Int. Ed., 57, 10268, 10.1002/anie.201806941
Schedin, 2007, Detection of individual gas molecules adsorbed on graphene, Nat. Mater., 6, 652, 10.1038/nmat1967
Liu, 2008, PEGylated nanographene oxide for delivery of water-insoluble cancer drugs, J. Am. Chem. Soc., 130, 10876, 10.1021/ja803688x
Zhu, 2018, Intracellular mechanistic understanding of 2D MoS2 nanosheets for anti-exocytosis-enhanced synergistic cancer therapy, ACS Nano, 12, 2922, 10.1021/acsnano.8b00516
Liu, 2018, Theranostic 2D ultrathin MnO2 nanosheets with fast responsibility to endogenous tumor microenvironment and exogenous NIR irradiation, Biomaterials, 155, 54, 10.1016/j.biomaterials.2017.11.015
Kirchon, 2018, From fundamentals to applications: a toolbox for robust and multifunctional MOF materials, Chem. Soc. Rev., 47, 8611, 10.1039/C8CS00688A
Liu, 2018, The geometry of periodic knots, polycatenanes and weaving from a chemical perspective: a library for reticular chemistry, Chem. Soc. Rev., 47, 4642, 10.1039/C7CS00695K
Wang, 2017, Organelle-specific triggered release of immunostimulatory oligonucleotides from intrinsically coordinated DNA-metal-organic frameworks with soluble exoskeleton, J. Am. Chem. Soc., 139, 15784, 10.1021/jacs.7b07895
Yang, 2019, Nanoscale ATP-responsive zeolitic imidazole framework-90 as a general platform for cytosolic protein delivery and genome editing, J. Am. Chem. Soc., 141, 3782, 10.1021/jacs.8b11996
Di, 2019, An acidic-microenvironment-driven DNA nanomachine enables specific ATP imaging in the extracellular milieu of tumor, Adv. Mater., 31, 1901885, 10.1002/adma.201901885
Alsaiari, 2018, Endosomal escape and delivery of CRISPR/Cas9 genome editing machinery enabled by nanoscale zeolitic imidazolate framework, J. Am. Chem. Soc., 140, 143, 10.1021/jacs.7b11754
Alyami, 2020, Cell-type-specific CRISPR/Cas9 delivery by biomimetic metal organic frameworks, J. Am. Chem. Soc., 142, 1715, 10.1021/jacs.9b11638
Rui, 2018, Non-viral delivery to enable genome editing, Trends Biotechnol., 37, 281, 10.1016/j.tibtech.2018.08.010
Wang, 2019, Clinical translation of gene medicine, J. Gene Med., 21, 10.1002/jgm.3108
Ginn, 2018, Gene therapy clinical trials worldwide to 2017: an update, J. Gene Med., 20, 10.1002/jgm.3015
Wang, 2018, Delivery of CRISPR/Cas9 by novel strategies for gene therapy, ChemBioChem, 20, 634, 10.1002/cbic.201800629
Yin, 2014, Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype, Nat. Biotechnol., 32, 551, 10.1038/nbt.2884
Jiang, 2017, A non-viral CRISPR/Cas9 delivery system for therapeutically targeting HBV DNA and pcsk9 in vivo, Cell Res., 27, 440, 10.1038/cr.2017.16
Hadjidemetriou, 2019, The human in vivo biomolecule corona onto PEGylated liposomes: a proof-of-concept clinical study, Adv. Mater., 31, 1803335, 10.1002/adma.201803335
Zhang, 2017, Biodegradable amino-ester nanomaterials for Cas9 mRNA delivery in vitro and in vivo, ACS Appl. Mater. Interfaces, 9, 25481, 10.1021/acsami.7b08163