Submitted Successfully!
To reward your contribution, here is a gift for you: A free trial for our video production service.
Thank you for your contribution! You can also upload a video entry or images related to this topic.
Version Summary Created by Modification Content Size Created at Operation
1 There is a growing need for a molecular vehicle that can successfully load and deliver CRISPR/Cas RNPs into target tissues. EVs are ideal candidates for a universal biological platform to produce CRISPR therapeutics + 1392 word(s) 1392 2020-10-12 10:17:55 |
2 format change Meta information modification 1392 2020-11-03 03:02:36 |

Video Upload Options

Do you have a full video?

Confirm

Are you sure to Delete?
Cite
If you have any further questions, please contact Encyclopedia Editorial Office.
Kostyushev, D.; Kostyusheva, A.; Brezgin, S.; Smirnov, V.; Volchkova, E.; Lukashev, A.; Chulanov, V. CRISPR/Cas-Based Gene Editing. Encyclopedia. Available online: https://encyclopedia.pub/entry/2856 (accessed on 28 March 2024).
Kostyushev D, Kostyusheva A, Brezgin S, Smirnov V, Volchkova E, Lukashev A, et al. CRISPR/Cas-Based Gene Editing. Encyclopedia. Available at: https://encyclopedia.pub/entry/2856. Accessed March 28, 2024.
Kostyushev, Dmitry, Anastasiya Kostyusheva, Sergey Brezgin, Valery Smirnov, Elena Volchkova, Alexander Lukashev, Vladimir Chulanov. "CRISPR/Cas-Based Gene Editing" Encyclopedia, https://encyclopedia.pub/entry/2856 (accessed March 28, 2024).
Kostyushev, D., Kostyusheva, A., Brezgin, S., Smirnov, V., Volchkova, E., Lukashev, A., & Chulanov, V. (2020, November 02). CRISPR/Cas-Based Gene Editing. In Encyclopedia. https://encyclopedia.pub/entry/2856
Kostyushev, Dmitry, et al. "CRISPR/Cas-Based Gene Editing." Encyclopedia. Web. 02 November, 2020.
CRISPR/Cas-Based Gene Editing
Edit

There is a growing need for a molecular vehicle that can successfully load and deliver CRISPR/Cas ribonucleoprotein complexes (and other gene editing systems) into target tissues. Synthetic delivery vehicles are being developed but so far have been only moderately successful. Extracellular vesicles are ideal candidates for a universal biological platform to produce ready-to-use, programmable, and highly biocompatible CRISPR therapeutics. Using extracellular vesicles in the CRISPR/Cas research and, ultimately, in the clinic, demands novel, advanced techniques for protein/RNA loading, surface engineering, and manufacturing. Safety of CRISPR/Cas systems and EVs also need to be tested extensively for every particular application.

gene editing biodistribution pharmacokinetics nanomedicines nanovesicles exosomes nanoparticles nanoblades stem cells mesenchymal stem cells

1. Introduction

CRISPR/Cas-based gene editing is a prominent, recently developed molecular technique that has already revolutionized biology and could dramatically transform clinical management of genetically defined conditions, including cancer, infectious, and genetic diseases[1]. CRISPR/Cas systems function by recruiting the Cas protein to a specific locus on a DNA or RNA molecule using a short RNA called single guide RNA (sgRNA)[2]. The Cas protein then introduces a break into the targeted nucleic acid[3][4]. Alternatively, nuclease-null Cas9 (or dead Cas9; dCas) proteins may serve as carriers to bring enzymes or functionally active factors to certain locations in the genome[5][6]. CRISPR/Cas systems provide the powerful means to directly modify genetic, epigenetic, and protein-based pathogenic mechanisms, projecting their application for treating numerous diseases[7][8].

2. Challenges 

So far, one of the major challenges is the lack of an optimized tissue-specific CRISPR/Cas delivery tool[9]. Many nanotechnological vehicles have been devised in recent years to deliver CRISPR/Cas systems into target cells (reviewed in[10][11][12][13]). Current strategies have numerous limitations, including: (1) high molecular mass and positive charge of Cas proteins that make them difficult to package using common drug delivery tools[14]; (2) the lack of robust tissue-specific delivery vehicles suitable for cell-specific gene editing applications[15]; (3) immunogenicity[16][17][18][19][20] and other safety issues (molecular, cellular and tissue toxicity)[21] to which the majority of novel synthetic delivery vehicles are prone; and, finally, (4) the lack of a universal CRISPR/Cas delivery platform that can be utilized for a wide array of CRISPR/Cas systems. Such a platform must allow use of CRISPR/Cas systems that are highly variable in size and molecular features[22]; systems isolated from various species (e.g., Neisseria meningitides[23], Streptococcus thermophiles[24], Streptococcus pyogenes[3], and others such as the recently described small CasX from Deltaproteobacteria[25]); and engineered CRISPR/Cas[26], such as CRISPRa/i tools[27][28][29], CRISPR base editors[30][31], and the PrimeEditing system[32]. The lack of robust and safe CRISPR/Cas delivery tools, especially with tissue-targeting modalities, delays translation of CRISPR/Cas-based therapeutics into the clinic. In particular, CRISPR/Cas systems have been shown to be highly potent antivirals eliminating or dramatically reducing viral loads in such infections as hepatitis B virus[33][34][35], hepatitis C virus[36], human immunodeficiency virus (HIV)[37][38][39], human papillomavirus[40], and even the recently emerged coronaviral SARS-CoV-2 infection[41]. Notably, CRISPR/Cas systems have been successfully leveraged to genetically modify the human genome for making primary CD4+ T cells resistant to HIV[42]; several ongoing clinical trials are underway using CRISPR/Cas for correcting mutations associated with genetic disorders and treating cancer.

Three principal methods are available to deliver Cas and their guiding RNAs (gRNAs) into target cells: (1) coding DNA sequences; (2) coding RNA/mRNA; and (3) ribonucleoprotein complexes (RNPs), i.e., readily available Cas protein complexes with in vitro-transcribed or synthetically generated gRNAs. Delivery of coding DNA sequences can be performed by both viral (including adeno-associated virus and adenovirus) and non-viral methods; packaging and delivery of mRNA/RNA and RNPs are usually performed by non-viral methods[15]. Nanotechnological methods mostly rely on the use of liposomes and cationic lipids[43][44][45], amphiphilic peptides[46], DNA nanoclews[47][48], gold nanoparticles[49][50][51], and graphene-based nanosheets[52].

Delivering CRISPR/Cas as DNA coding sequences is fraught with poorly controllable intracellular synthesis of CRISPR/Cas components with an ensuing increase in off-target activity[53][54][55] and potential integration of DNA into the genome[56]. Although plenty of novel approaches have been proposed to hone the specificity of CRISPR/Cas systems (e.g., self-inactivating delivery systems[57][58], on/off-inducible systems[59][60]) and build additional levels of tunability (e.g., anti-CRISPR proteins[61][62]), these approaches add complexity and safety issues. Delivering large amounts of DNA is also associated with toxicity, may induce activation of the host factors involved in foreign DNA recognition, and may even cause cell death[63][64][65][66]. Additionally, the large molecular size of traditional CRISPR/Cas nucleases and, especially, dCas-based molecular tools exceeds the packaging capacity of commonly used AAV viral vectors and thus hampers their use. This is particularly true for hybrid CRISPR/Cas systems fused to additional functional moieties (epigenome modifiers[67], transposases[68][69], reverse transcriptases[32], etc.), that add molecular weight to Cas proteins.

Delivery of CRISPR/Cas as mRNA/RNAs is associated with instability and fragility of the long Cas mRNAs and may be substantially compromised by reduced efficacy of on-target editing[70][71][72]. The most straightforward approach is direct delivery of CRISPR/Cas RNPs into the cells[73]. Successful gene editing for treating a disease, whether a genetic disorder or an infectious illness, usually requires very transient expression of CRISPR/Cas, which may permanently correct the malfunctioning gene or rapidly destroy the viral genomes. Many recent studies demonstrated that the delivery of CRISPR/Cas RNPs is characterized by the highest efficacy and specificity of gene editing[74][75][76].

Proteins or RNPs cannot be delivered systemically as naked molecules. Human serum contains proteases that can rapidly destroy unprotected proteins. Protein and RNA components of CRISPR/Cas are therefore vulnerable to rapid degradation upon systemic injection and must be protected by nanoparticles for in vivo applications.

Moreover, pre-existing antibodies against Cas proteins[16] and immune response to Cas and sgRNAs[18] can limit efficacy of CRISPR/Cas approaches. Reducing and evading immune recognition can be achieved by rationally designing Cas proteins (e.g., epitope masking or limiting presentation of Cas epitopes to the immune system)[77] [78], using CRISPR/Cas systems from non-pathogenic organisms, inducing immune tolerance[78], or shielding Cas proteins in systemic circulation. Short-lived CRISPR/Cas complexes are sufficient for most clinical applications, especially in immune-privileged organs, and are less likely to induce a meaningful immune response. Nevertheless, in order to increase efficacy and preserve a second-use opportunity, it is desirable to shield Cas and RNPs from immune recognition.

Cas proteins are not naturally able to cross biological barriers without specially designed delivery vehicles and do not tend to accumulate in specific organs or tissues. High positive charge and molecular mass (>160 kDa for S. pyogenes Cas9) make CRISPR/Cas RNPs unsuitable for traditional methods of nanotechnological packaging and protein delivery. Thus, engineering advanced molecular vehicles encapsulating the CRISPR/Cas RNPs with penetrating and targeting ability is needed for tissue-specific delivery of gene editing complexes and clinical implementation of devised molecular techniques.

The ideal non-viral method for targeted in vivo drug delivery should fit the following criteria: (a) effectively package CRISPR/Cas RNPs of any type and species and with any modifications; (b) shield RNPs from an aggressive environment and the immune system; (c) effectively deliver RNPs into target organs; (d) escape endolysosomal pathways; and (e) be simple and scalable. To date, no such methods exist.

To date, the most successful approach for local targeted delivery of gene editing systems in the form of gold-linked nanoparticles combined with penetrating peptides demonstrated ~30% efficacy[49]. However, this method does not shield Cas proteins and suffers from other disadvantages, such as immunogenicity, toxicity, and rapid clearance upon systemic administration. On the other hand, EVs have emerged as a promising delivery system for proteins and RNAs, substantially outperforming synthetic nanocarriers in terms of safety and pharmacokinetics[79]. Evs are natural nanoparticles secreted by numerous cell types that exhibit very high biocompatibility and extraordinary ability to cross biological barriers[80]. Because Evs can transfer RNA, protein, and lipid cargo, display preferential tropism for certain tissues, and are amenable to engineering, they have been extensively utilized as potential drug delivery systems. Genetic engineering of EV-producing cells and modification of purified Evs enables direct loading of therapeutic macromolecules into the vesicles and targeted drug delivery.

The advantages of Evs have been increasingly utilized for CRISPR/Cas delivery, but translating EV-CRISPR/Cas therapies to the clinic requires the invention of new, more efficient techniques for EV cargo loading and surface engineering. Overall, there is great demand for developing effective, programmable, versatile, and safe delivery platforms that ideally can be used for any type of CRISPR/Cas system.

References

  1. Patrick D. Hsu; Eric S. Lander; Feng Zhang; Development and Applications of CRISPR-Cas9 for Genome Engineering. Cell 2014, 157, 1262-1278, 10.1016/j.cell.2014.05.010.
  2. Martin Jinek; Krzysztof Chylinski; Ines Fonfara; Michael Hauer; Jennifer A. Doudna; Emmanuelle Charpentier; A Programmable Dual-RNA-Guided DNA Endonuclease in Adaptive Bacterial Immunity. Science 2012, 337, 816-821, 10.1126/science.1225829.
  3. Cong, L.; Ran, F.A.; Cox, D.; Lin, S.; Barretto, R.; Habib, N.; Hsu, P.D.; Wu, X.; Jiang, W.; Marraffini, L.A.; et al. Multiplex Genome Engineering Using CRISPR/Cas Systems. Science 2013, 339, 819–823.
  4. Ran, F.A.; Hsu, P.D.; Wright, J.; Agarwala, V.; Scott, D.A.; Zhang, F. Genome engineering using the CRISPR-Cas9 system. Nat. Protoc. 2013, 8, 2281–2308.
  5. Qi, L.S.; Larson, M.H.; Gilbert, L.A.; Doudna, J.A.; Weissman, J.S.; Arkin, A.P.; Lim, W.A. Repurposing CRISPR as an RNA-Guided Platform for Sequence-Specific Control of Gene Expression. Cell 2013, 152, 1173–1183.
  6. Gilbert, L.A.; Larson, M.H.; Morsut, L.; Liu, Z.; Brar, G.A.; Torres, S.E.; Stern-Ginossar, N.; Brandman, O.; Whitehead, E.H.; Doudna, J.A.; et al. CRISPR-Mediated Modular RNA-Guided Regulation of Transcription in Eukaryotes. Cell 2013, 154, 442–451.
  7. Wang, H.; La Russa, M.; Qi, L.S. CRISPR/Cas9 in Genome Editing and Beyond. Annu. Rev. Biochem. 2016, 85, 227–264.
  8. Savić, N.; Schwank, G. Advances in therapeutic CRISPR/Cas9 genome editing. Transl. Res. 2016, 168, 15–21.
  9. Ling Li; Zhiyao He; Xiawei Wei; Guang-Ping Gao; Yuquan Wei; Challenges in CRISPR/CAS9 Delivery: Potential Roles of Nonviral Vectors. Human Gene Therapy 2015, 26, 452-462, 10.1089/hum.2015.069.
  10. Liu, C.; Zhang, L.; Liu, H.; Cheng, K. Delivery strategies of the CRISPR-Cas9 gene-editing system for therapeutic applications. J. Control. Release 2017, 266, 17–26.
  11. Wang, H.-X.; Li, M.; Lee, C.M.; Chakraborty, S.; Kim, H.-W.; Bao, G.; Leong, K.W. CRISPR/Cas9-Based Genome Editing for Disease Modeling and Therapy: Challenges and Opportunities for Nonviral Delivery. Chem. Rev. 2017, 117, 9874–9906.
  12. Li, L.; Hu, S.; Chen, X. Non-viral delivery systems for CRISPR/Cas9-based genome editing: Challenges and opportunities. Biomaterials 2018, 171, 207–218.
  13. Chen, F.; Alphonse, M.; Liu, Q. Strategies for nonviral nanoparticle-based delivery of CRISPR/Cas9 therapeutics. Wiley Interdiscip. Rev. Nanomed. Nanobiotechnol. 2020, 12, 1609.
  14. Rubul Mout; Moumita Ray; Yi-Wei Lee; Federica Scaletti; Vincent M. Rotello; In Vivo Delivery of CRISPR/Cas9 for Therapeutic Gene Editing: Progress and Challenges. Bioconjugate Chemistry 2017, 28, 880-884, 10.1021/acs.bioconjchem.7b00057.
  15. Christopher A. Lino; Jason C. Harper; James P. Carney; Jerilyn A. Timlin; Delivering CRISPR: a review of the challenges and approaches. Drug Delivery 2018, 25, 1234-1257, 10.1080/10717544.2018.1474964.
  16. Charlesworth, C.T.; Deshpande, P.S.; Dever, D.P.; Camarena, J.; Lemgart, V.T.; Cromer, M.K.; Vakulskas, C.A.; Collingwood, M.A.; Zhang, L.; Bode, N.M.; et al. Identification of preexisting adaptive immunity to Cas9 proteins in humans. Nat. Med. 2019, 25, 249–254.
  17. Kim, S.; Koo, T.; Jee, H.-G.; Cho, H.-Y.; Lee, G.; Lim, D.-G.; Shin, H.S.; Kim, J.H. CRISPR RNAs trigger innate immune responses in human cells. Genome Res. 2018, 28, 367–373.
  18. Wagner, D.L.; Amini, L.; Wendering, D.J.; Burkhardt, L.-M.; Akyüz, L.; Reinke, P.; Volk, H.-D.; Schmueck-Henneresse, M. High prevalence of Streptococcus pyogenes Cas9-reactive T cells within the adult human population. Nat. Med. 2018, 25, 242–248.
  19. Chew, W.L.; Tabebordbar, M.; Cheng, J.K.; Mali, P.; Wu, E.Y.; Ng, A.H.; Zhu, K.; Wagers, A.J.; Church, G.M. A multifunctional AAV–CRISPR–Cas9 and its host response. Nat. Methods 2016, 13, 868–874.
  20. Simhadri, V.L.; McGill, J.; McMahon, S.; Wang, J.; Jiang, H.; Sauna, Z.E. Prevalence of Pre-existing Antibodies to CRISPR-Associated Nuclease Cas9 in the USA Population. Mol. Ther. Methods Clin. Dev. 2018, 10, 105–112.
  21. Joy Wolfram; Motao Zhu; Yong Yang; Jianliang Shen; Emanuela Gentile; Donatella Paolino; Massimo Fresta; Guangjun Nie; Chunying Chen; Haifa Shen; et al.Mauro FerrariYuliang Zhao Safety of Nanoparticles in Medicine. Current Drug Targets 2015, 16, 1671-1681, 10.2174/1389450115666140804124808.
  22. Kira S. Makarova; Yuri I. Wolf; Jaime Iranzo; Sergey A. Shmakov; Omer S. Alkhnbashi; Stan J. J. Brouns; Emmanuelle Charpentier; David Cheng; Daniel H. Haft; Philippe Horvath; et al.Sylvain MoineauFrancisco J. M. MojicaDavid ScottShiraz A. ShahVirginijus SiksnysMichael P. TernsČeslovas VenclovasMalcolm F. WhiteAlexander F. YakuninWinston YanFeng ZhangRoger A. GarrettRolf BackofenJohn Van Der OostRodolphe BarrangouEugene V. Koonin Evolutionary classification of CRISPR–Cas systems: a burst of class 2 and derived variants. Nature Reviews Microbiology 2019, 18, 67-83, 10.1038/s41579-019-0299-x.
  23. Ciaran M. Lee; Thomas J. Cradick; Gang Bao; The Neisseria meningitidis CRISPR-Cas9 System Enables Specific Genome Editing in Mammalian Cells. Molecular Therapy 2016, 24, 645-654, 10.1038/mt.2016.8.
  24. Rimantas Sapranauskas; Giedrius Gasiunas; Christophe Fremaux; Rodolphe Barrangou; Philippe Horvath; Virginijus Siksnys; The Streptococcus thermophilus CRISPR/Cas system provides immunity in Escherichia coli. Nucleic Acids Research 2011, 39, 9275-9282, 10.1093/nar/gkr606.
  25. David Burstein; Lucas B. Harrington; Steven C. Strutt; Alexander J. Probst; Karthik Anantharaman; Brian C. Thomas; Jennifer A. Doudna; Jillian F. Banfield; New CRISPR–Cas systems from uncultivated microbes. Nature 2016, 542, 237-241, 10.1038/nature21059.
  26. Sergey Brezgin; Anastasiya Kostyusheva; Dmitry Kostyushev; Vladimir Chulanov; Dead Cas Systems: Types, Principles, and Applications. International Journal of Molecular Sciences 2019, 20, 6041, 10.3390/ijms20236041.
  27. Maeder, M.L.; Linder, S.J.; Cascio, V.M.; Fu, Y.; Ho, Q.H.; Joung, J.K. CRISPR RNA–guided activation of endogenous human genes. Nat. Chem. Biol. 2013, 10, 977–979.
  28. Gilbert, L.A.; Horlbeck, M.A.; Adamson, B.; Villalta, J.E.; Chen, Y.; Whitehead, E.H.; Guimaraes, C.; Panning, B.; Ploegh, H.L.; Bassik, M.C.; et al. Genome-Scale CRISPR-Mediated Control of Gene Repression and Activation. Cell 2014, 159, 647–661.
  29. Hilton, I.B.; D’Ippolito, A.M.; Vockley, C.M.; Thakore, P.I.; Crawford, G.E.; Reddy, T.E.; Gersbach, C.A. Epigenome editing by a CRISPR-Cas9-based acetyltransferase activates genes from promoters and enhancers. Nat. Biotechnol. 2015, 33, 510–517
  30. Komor, A.C.; Kim, Y.B.; Packer, M.S.; Zuris, J.A.; Liu, D.R. Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage. Nat. Cell Biol. 2016, 533, 420–424.
  31. Gaudelli, N.M.; Komor, A.C.; Rees, H.A.; Packer, M.S.; Badran, A.H.; Bryson, D.I.; Liu, D.R. Programmable base editing of A•T to G•C in genomic DNA without DNA cleavage. Nat. Cell Biol. 2017, 551, 464–471.
  32. Andrew V. Anzalone; Peyton B. Randolph; Jessie R. Davis; Alexander A. Sousa; Luke W. Koblan; Jonathan M. Levy; Peter J. Chen; Christopher Wilson; Gregory A. Newby; Aditya Raguram; et al.David R. Liu Search-and-replace genome editing without double-strand breaks or donor DNA. Nature 2019, 576, 149-157, 10.1038/s41586-019-1711-4.
  33. Lin, S.-R.; Yang, H.-C.; Kuo, Y.-T.; Liu, C.-J.; Yang, T.-Y.; Sung, K.-C.; Lin, Y.-Y.; Wang, H.-Y.; Wang, C.-C.; Shen, Y.-C.; et al. The CRISPR/Cas9 System Facilitates Clearance of the Intrahepatic HBV Templates In Vivo. Mol. Ther. Nucleic Acids 2014, 3, e186.
  34. Liu, X.; Hao, R.; Chen, S.; Guo, D.; Chen, Y. Inhibition of hepatitis B virus by the CRISPR/Cas9 system via targeting the conserved regions of the viral genome. J. Gen. Virol. 2015, 96, 2252–2261.
  35. Kostyushev, D.S.; Brezgin, S.; Kostyusheva, A.; Zarifyan, D.; Goptar, I.; Chulanov, V. Orthologous CRISPR/Cas9 systems for specific and efficient degradation of covalently closed circular DNA of hepatitis B virus. Cell. Mol. Life Sci. 2019, 76, 1779–1794.
  36. Aryn A. Price; Timothy R. Sampson; Hannah K. Ratner; Arash Grakoui; David S. Weiss; Cas9-mediated targeting of viral RNA in eukaryotic cells. Proceedings of the National Academy of Sciences 2015, 112, 6164-6169, 10.1073/pnas.1422340112.
  37. Ebina, H.; Misawa, N.; Kanemura, Y.; Koyanagi, Y. Harnessing the CRISPR/Cas9 system to disrupt latent HIV-1 provirus. Sci. Rep. 2013, 3, srep02510.
  38. Liao, H.-K.; Gu, Y.; Diaz, A.; Marlett, J.; Takahashi, Y.; Li, M.; Suzuki, K.; Xu, R.; Hishida, T.; Chang, C.-J.; et al. Use of the CRISPR/Cas9 system as an intracellular defense against HIV-1 infection in human cells. Nat. Commun. 2015, 6, 6413.
  39. Kaminski, R.; Chen, Y.; Fischer, T.; Tedaldi, E.; Napoli, A.; Zhang, Y.; Karn, J.; Hu, W.; Khalili, K. Elimination of HIV-1 genomes from human T-lymphoid cells by CRISPR/Cas9 gene editing. Sci. Rep. 2016, 6, 1–15.
  40. Zheng Hu; Lan Yu; Da Zhu; Wencheng Ding; Xiaoli Wang; Changlin Zhang; Liming Wang; Xiaohui Jiang; Hui Shen; Dan He; et al.Kezhen LiLing XiDing MaHui Wang Disruption of HPV16-E7 by CRISPR/Cas System Induces Apoptosis and Growth Inhibition in HPV16 Positive Human Cervical Cancer Cells. BioMed Research International 2014, 2014, 1-9, 10.1155/2014/612823.
  41. Timothy R. Abbott; Girija Dhamdhere; Yanxia Liu; Xueqiu Lin; Laine Goudy; Leiping Zeng; Augustine Chemparathy; Stephen Chmura; Nicholas S. Heaton; Robert Debs; et al.Tara PandeDrew EndyMarie F. La RussaDavid B. LewisLei S. Qi Development of CRISPR as an Antiviral Strategy to Combat SARS-CoV-2 and Influenza. Cell 2020, 181, 865-876.e12, 10.1016/j.cell.2020.04.020.
  42. Lei Xu; Jun Wang; Yulin Liu; Liangfu Xie; Bin Su; Danlei Mou; Longteng Wang; Tingting Liu; Xiaobao Wang; Bin Zhang; et al.Long ZhaoLiangding HuHongmei NingYufeng ZhangKai DengLifeng LiuXiaofan LuTong ZhangJun XuCheng LiHao WuHongkui DengHu Chen CRISPR-Edited Stem Cells in a Patient with HIV and Acute Lymphocytic Leukemia. New England Journal of Medicine 2019, 381, 1240-1247, 10.1056/nejmoa1817426.
  43. Zuris, J.A.; Thompson, D.B.; Shu, Y.; Guilinger, J.P.; Bessen, J.L.; Hu, J.H.; Maeder, M.L.; Joung, J.K.; Chen, Z.-Y.; Liu, D.R. Cationic lipid-mediated delivery of proteins enables efficient protein-based genome editing in vitro and in vivo. Nat. Biotechnol. 2014, 33, 73–80.
  44. Kang, Y.K.; Kwon, K.; Ryu, J.S.; Lee, H.N.; Park, C.; Chung, H.J. Nonviral Genome Editing Based on a Polymer-Derivatized CRISPR Nanocomplex for Targeting Bacterial Pathogens and Antibiotic Resistance. Bioconjug. Chem. 2017, 28, 957–967.
  45. Kooijmans, S.; Fliervoet, L.; Van Der Meel, R.; Fens, M.; Heijnen, H.; Henegouwen, P.V.B.E.; Vader, P.; Schiffelers, R. PEGylated and targeted extracellular vesicles display enhanced cell specificity and circulation time. J. Control. Release 2016, 224, 77–85.
  46. Sateesh Krishnamurthy; Christine Wohlford-Lenane; Suhas Kandimalla; Gilles Sartre; David K. Meyerholz; Vanessa Théberge; Stéphanie Hallée; Anne-Marie Duperré; Thomas Del’Guidice; Jean-Pascal Lepetit-Stoffaes; et al.Xavier BarbeauDavid GuayPaul B. McCray Jr. Engineered amphiphilic peptides enable delivery of proteins and CRISPR-associated nucleases to airway epithelia. Nature Communications 2019, 10, 1-12, 10.1038/s41467-019-12922-y.
  47. Sun, W.; Wang, J.; Hu, Q.; Zhou, X.; Khademhosseini, A.; Gu, Z. CRISPR-Cas12a delivery by DNA-mediated bioresponsive editing for cholesterol regulation. Sci. Adv. 2020, 6, eaba2983.
  48. Sun, W.; Ji, W.; Hall, J.M.; Hu, Q.; Wang, C.; Beisel, C.L.; Gu, Z. Self-Assembled DNA Nanoclews for the Efficient Delivery of CRISPR-Cas9 for Genome Editing. Angew. Chem. Int. Ed. 2015, 54, 12029–12033.
  49. Mout, R.; Ray, M.; Tonga, G.Y.; Lee, Y.-W.; Tay, T.; Sasaki, K.; Rotello, V.M. Direct Cytosolic Delivery of CRISPR/Cas9-Ribonucleoprotein for Efficient Gene Editing. ACS Nano 2017, 11, 2452–2458.
  50. Lostalé-Seijo, I.; Louzao, I.; Juanes, M.; Montenegro, J. Peptide/Cas9 nanostructures for ribonucleoprotein cell membrane transport and gene edition. Chem. Sci. 2017, 8, 7923–7931.
  51. Wang, P.; Zhang, L.; Zheng, W.; Cong, L.; Guo, Z.; Xie, Y.; Wang, L.; Tang, R.; Feng, Q.; Hamada, Y.; et al. Thermo-triggered Release of CRISPR-Cas9 System by Lipid-Encapsulated Gold Nanoparticles for Tumor Therapy. Angew. Chem. Int. Ed. 2018, 57, 1491–1496.
  52. Huahua Yue; XiaoMing Zhou; Meng Cheng; Da Xing; Graphene oxide-mediated Cas9/sgRNA delivery for efficient genome editing. Nanoscale 2018, 10, 1063-1071, 10.1039/c7nr07999k.
  53. Kosicki, M.; Tomberg, K.; Bradley, A. Repair of double-strand breaks induced by CRISPR–Cas9 leads to large deletions and complex rearrangements. Nat. Biotechnol. 2018, 36, 765–771.
  54. Kim, S.; Kim, D.; Cho, S.W.; Kim, J. Highly efficient RNA-guided genome editing in human cells via delivery of purified Cas9 ribonucleoproteins. Genome Res. 2014, 24, 1012–1019.
  55. Cho, S.W.; Kim, S.; Kim, Y.; Kweon, J.; Kim, H.S.; Bae, S.; Kim, J.-S. Analysis of off-target effects of CRISPR/Cas-derived RNA-guided endonucleases and nickases. Genome Res. 2013, 24, 132–141.
  56. Z Wang; P J Troilo; X Wang; T G Griffiths; S J Pacchione; A B Barnum; L B Harper; C J Pauley; Z Niu; L Denisova; et al.T T FollmerG RizzutoG CilibertoE FattoriN L MonicaS ManamB J Ledwith Detection of integration of plasmid DNA into host genomic DNA following intramuscular injection and electroporation. Gene Therapy 2004, 11, 711-721, 10.1038/sj.gt.3302213.
  57. Merienne, N.; Vachey, G.; De Longprez, L.; Meunier, C.; Zimmer, V.; Perriard, G.; Canales, M.; Mathias, A.; Herrgott, L.; Beltraminelli, T.; et al. The Self-Inactivating KamiCas9 System for the Editing of CNS Disease Genes. Cell Rep. 2017, 20, 2980–2991.
  58. Li, A.; Lee, C.M.; Hurley, A.E.; Jarrett, K.E.; De Giorgi, M.; Lu, W.; Balderrama, K.S.; Doerfler, A.M.; Deshmukh, H.; Ray, A.; et al. A Self-Deleting AAV-CRISPR System for In Vivo Genome Editing. Mol. Ther. Methods Clin. Dev. 2019, 12, 111–122.
  59. Kelkar, A.; Zhu, Y.; Groth, T.; Stolfa, G.; Stablewski, A.B.; Singhi, N.; Nemeth, M.; Neelamegham, S. Doxycycline-Dependent Self-Inactivation of CRISPR-Cas9 to Temporally Regulate On- and Off-Target Editing. Mol. Ther. 2020, 28, 29–41.
  60. E Dow, L.; Fisher, J.; O’Rourke, K.P.; Muley, A.; Kastenhuber, E.R.; Livshits, G.; Tschaharganeh, D.F.; Socci, N.D.; Lowe, S.W. Inducible in vivo genome editing with CRISPR-Cas9. Nat. Biotechnol. 2015, 33, 390–394.
  61. Bondy-Denomy, J.; Pawluk, A.; Maxwell, K.L.; Davidson, A.R. Bacteriophage genes that inactivate the CRISPR/Cas bacterial immune system. Nat. Cell Biol. 2012, 493, 429–432.
  62. Hynes, A.P.; Rousseau, G.M.; Agudelo, D.; Goulet, A.; Amigues, B.; Loehr, J.; Romero, D.A.; Fremaux, C.; Horvath, P.; Doyon, Y.; et al. Widespread anti-CRISPR proteins in virulent bacteriophages inhibit a range of Cas9 proteins. Nat. Commun. 2018, 9, 2919.
  63. Paludan, S.R.; Bowie, A.G. Immune Sensing of DNA. Immunity 2013, 38, 870–880.
  64. Zhang, X.; Brann, T.W.; Zhou, M.; Yang, J.; Oguariri, R.M.; Lidie, K.B.; Imamichi, H.; Huang, D.-W.; Lempicki, R.A.; Baseler, M.W.; et al. Cutting Edge: Ku70 Is a Novel Cytosolic DNA Sensor That Induces Type III Rather Than Type I IFN. J. Immunol. 2011, 186, 4541–4545.
  65. Semenova, N.; Bosnjak, M.; Markelc, B.; Znidar, K.; Cemazar, M.; Heller, L. Multiple cytosolic DNA sensors bind plasmid DNA after transfection. Nucleic Acids Res. 2019, 47, 10235–10246.
  66. Znidar, K.; Bosnjak, M.; Semenova, N.; Pakhomova, O.; Heller, L.C.; Cemazar, M. Tumor cell death after electrotransfer of plasmid DNA is associated with cytosolic DNA sensor upregulation. Oncotarget 2018, 9, 18665–18681.
  67. Sanne E. Klompe; Phuc L. H. Vo; Tyler S. Halpin-Healy; Samuel H. Sternberg; Transposon-encoded CRISPR–Cas systems direct RNA-guided DNA integration. Nature 2019, 571, 219-225, 10.1038/s41586-019-1323-z.
  68. Strecker, J.; Ladha, A.; Gardner, Z.; Schmid-Burgk, J.L.; Makarova, K.S.; Koonin, E.V.; Zhang, F. RNA-guided DNA insertion with CRISPR-associated transposases. Science 2019, 365, 48–53.
  69. Wadhwa, A.; Aljabbari, A.; Lokras, A.; Foged, C.; Thakur, A. Opportunities and Challenges in the Delivery of mRNA-Based Vaccines. Pharmaceutics 2020, 12, 102.
  70. Schlake, T.; Thess, A.; Thran, M.; Jordan, I. mRNA as novel technology for passive immunotherapy. Cell. Mol. Life Sci. 2018, 76, 301–328.
  71. Luther, D.C.; Lee, Y.; Nagaraj, H.; Scaletti, F.; Rotello, V. Delivery approaches for CRISPR/Cas9 therapeutics in vivo: Advances and challenges. Expert Opin. Drug Deliv. 2018, 15, 905–913.
  72. Gasiunas, G.; Barrangou, R.; Horvath, P.; Siksnys, V. Cas9-crRNA ribonucleoprotein complex mediates specific DNA cleavage for adaptive immunity in bacteria. Proc. Natl. Acad. Sci. USA 2012, 109, E2579–E2586.
  73. Mark A. DeWitt; Jacob E. Corn; Dana Carroll; Genome editing via delivery of Cas9 ribonucleoprotein. Methods 2017, 121, 9-15, 10.1016/j.ymeth.2017.04.003.
  74. Vakulskas, C.A.; Dever, D.P.; Rettig, G.R.; Turk, R.; Jacobi, A.M.; Collingwood, M.A.; Bode, N.M.; McNeill, M.S.; Yan, S.; Camarena, J.; et al. A high-fidelity Cas9 mutant delivered as a ribonucleoprotein complex enables efficient gene editing in human hematopoietic stem and progenitor cells. Nat. Med. 2018, 24, 1216–1224.
  75. Chen, S.; Lee, B.; Lee, A.Y.-F.; Modzelewski, A.J.; He, L. Highly Efficient Mouse Genome Editing by CRISPR Ribonucleoprotein Electroporation of Zygotes. J. Boil. Chem. 2016, 291, 14457–14467.
  76. Ferdosi, S.R.; Ewaisha, R.; Moghadam, F.; Krishna, S.; Park, J.G.; Ebrahimkhani, M.R.; Kiani, S.; Anderson, K.S. Multifunctional CRISPR-Cas9 with engineered immunosilenced human T cell epitopes. Nat. Commun. 2019, 10, 1842.
  77. Thirushan Wignakumar; Paul J. Fairchild; Evasion of Pre-Existing Immunity to Cas9: a Prerequisite for Successful Genome Editing In Vivo?. Current Transplantation Reports 2019, 6, 127-133, 10.1007/s40472-019-00237-2.
  78. Pieter Vader; Emma A. Mol; Gerard Pasterkamp; Raymond M. Schiffelers; Extracellular vesicles for drug delivery. Advanced Drug Delivery Reviews 2016, 106, 148-156, 10.1016/j.addr.2016.02.006.
  79. María Yáñez-Mó; Pia R.-M. Siljander; Zoraida Andreu; Apolonija Bedina Zavec; Francesc E. Borràs; Edit I. Buzas; Krisztina Buzas; Enriqueta Casal; Francesco Cappello; Joana Carvalho; et al.Eva ColásAnabela Cordeiro-Da SilvaStefano FaisJuan M. Falcon-PerezIrene M. GhobrialBernd GiebelMario GimonaMichael GranerIhsan GurselMayda GurselNiels H. H. HeegaardAn HendrixPeter KierulfKatsutoshi KokubunMaja KosanovicVeronika Kralj-IglicEva-Maria Krämer-AlbersSaara LaitinenCecilia LässerThomas LenerErzsébet LigetiAija LinēGeorg LippsAlicia LlorenteJan LötvallMateja Manček-KeberAntonio MarcillaMaria MittelbrunnIrina NazarenkoEsther N.M. Nolte-‘T HoenTuula A. NymanLorraine O'driscollMireia OlivanCarla OliveiraÉva PállingerHernando A. Del PortilloJaume ReventósMarina RigauEva RohdeMarei SammarFrancisco Sánchez-MadridN. SantarémKatharina SchallmoserMarie Stampe OstenfeldWillem StoorvogelRoman StukeljSusanne G. Van Der GreinM. Helena VasconcelosMarca H. M. WaubenOlivier De Wever Biological properties of extracellular vesicles and their physiological functions. Journal of Extracellular Vesicles 2015, 4, 27066, 10.3402/jev.v4.27066.
  80. Guillaume Van Niel; Gisela D'angelo; Graça Raposo; Shedding light on the cell biology of extracellular vesicles. Nature Reviews Molecular Cell Biology 2018, 19, 213-228, 10.1038/nrm.2017.125.
More
Information
Contributors MDPI registered users' name will be linked to their SciProfiles pages. To register with us, please refer to https://encyclopedia.pub/register : , , , , , ,
View Times: 482
Revisions: 2 times (View History)
Update Date: 03 Nov 2020
1000/1000