Cited by 540RNA Interference and Gene DeliveryCRISPR and Genetic EngineeringNanoparticle-Based Drug Delivery

The DOI matches its Crossref record; 44 of the 45 references it cites were found in a registry or an index.

Non‐Viral CRISPR/Cas Gene Editing In Vitro and In Vivo Enabled by Synthetic Nanoparticle Co‐Delivery of Cas9 mRNA and sgRNA

Open access

Miller, Jason B., Zhang, Shuyuan, Kos, Petra, Xiong, Hu, Zhou, Kejin, Perelman, Sofya S., Zhu, Hao, Siegwart, Daniel J.·2017·Angewandte Chemie International Edition

DOI 10.1002/anie.201610209PMC PMC5521011

Open this paper in CoChat — free account

Abstract

CRISPR/Cas is a revolutionary gene editing technology with wide‐ranging utility. The safe, non‐viral delivery of CRISPR/Cas components would greatly improve future therapeutic utility. We report the synthesis and development of zwitterionic amino lipids (ZALs) that are uniquely able to (co)deliver long RNAs including Cas9 mRNA and sgRNAs. ZAL nanoparticle (ZNP) delivery of low sgRNA doses (15 n m ) reduces protein expression by >90 % in cells. In contrast to transient therapies (such as RNAi), we show that ZNP delivery of sgRNA enables permanent DNA editing with an indefinitely sustained 95 % decrease in protein expression. ZNP delivery of mRNA results in high protein expression at low doses in vitro (<600 pM) and in vivo (1 mg kg −1 ). Intravenous co‐delivery of Cas9 mRNA and sgLoxP induced expression of floxed tdTomato in the liver, kidneys, and lungs of engineered mice. ZNPs provide a chemical guide for rational design of long RNA carriers, and represent a promising step towards improving the safety and utility of gene editing.

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.

Integrity signals

Registry

Crossref · 10.1002/anie.201610209

Cites

44 of 44 references verified0 citation errors · 0 not checkable

Statistics

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.

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.

A Programmable Dual-RNA–Guided DNA Endonuclease in Adaptive Bacterial Immunity
Jinek, M., Chylinski, K., Fonfara, I., Hauer, M., Doudna, J. A., & Charpentier, E. (2012) · Science · doi:10.1126/science.1225829 · Cited by 17,116
Multiplex Genome Engineering Using CRISPR/Cas Systems
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. (2013) · Science · doi:10.1126/science.1231143 · Cited by 15,339
Genome engineering using the CRISPR-Cas9 system
Ran, F. A., Hsu, P. D., Wright, J., Agarwala, V., Scott, D. A., & Zhang, F. (2013) · Nature Protocols · doi:10.1038/nprot.2013.143 · Cited by 11,133
RNA-Guided Human Genome Engineering via Cas9
Mali, P., Yang, L., Esvelt, K. M., Aach, J., Guell, M., DiCarlo, J. E., Norville, J. E., & Church, G. M. (2013) · Science · doi:10.1126/science.1232033 · Cited by 9,210
CRISPR-Cas systems for editing, regulating and targeting genomes
Sander, J. D., & Joung, J. K. (2014) · Nature Biotechnology · doi:10.1038/nbt.2842 · Cited by 3,153
CRISPR-Cas9 Knockin Mice for Genome Editing and Cancer Modeling
Platt, R. J., Chen, S., Zhou, Y., Yim, M. J., Swiech, L., Kempton, H. R., Dahlman, J. E., Parnas, O., Eisenhaure, T. M., Jovanovic, M., et al. (2014) · Cell · doi:10.1016/j.cell.2014.09.014 · Cited by 2,001
Rational design of cationic lipids for siRNA delivery
Semple, S. C., Akinc, A., Chen, J., Sandhu, A. P., Mui, B. L., Cho, C. K., Sah, D. W. Y., Stebbing, D., Crosley, E. J., Yaworski, E., et al. (2010) · Nature Biotechnology · doi:10.1038/nbt.1602 · Cited by 1,822
Delivery materials for siRNA therapeutics
Kanasty, R., Dorkin, J. R., Vegas, A., & Anderson, D. (2013) · Nature Materials · doi:10.1038/nmat3765 · Cited by 1,800
Image-based analysis of lipid nanoparticle–mediated siRNA delivery, intracellular trafficking and endosomal escape
Gilleron, J., Querbes, W., Zeigerer, A., Borodovsky, A., Marsico, G., Schubert, U., Manygoats, K., Seifert, S., Andree, C., Stöter, M., et al. (2013) · Nature Biotechnology · doi:10.1038/nbt.2612 · Cited by 1,681
Cationic lipid-mediated delivery of proteins enables efficient protein-based genome editing in vitro and in vivo
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. (2015) · Nature Biotechnology · doi:10.1038/nbt.3081 · Cited by 1,460

Flagged first, then by citations

Show all 45 references

1 of the 45 references couldn’t be matched to a known paper.

Sorted by citations

Items 101–200 of 540

Triple‐Targeting Delivery of CRISPR/Cas9 To Reduce the Risk of Cardiovascular Diseases
Zhang, L., Wang, L., Xie, Y., Wang, P., Deng, S., Qin, A., Zhang, J., Yu, X., Zheng, W., & Jiang, X. (2019) · Angewandte Chemie International Edition · doi:10.1002/anie.201903618 · Cited by 144
Virus-like nanoparticle as a co-delivery system to enhance efficacy of CRISPR/Cas9-based cancer immunotherapy
Liu, Q., Wang, C., Zheng, Y., Zhao, Y., Wang, Y., Hao, J., Zhao, X., Yi, K., Shi, L., Kang, C., et al. (2020) · Biomaterials · doi:10.1016/j.biomaterials.2020.120275 · Cited by 144
Hypoxia‐Responsive Gene Editing to Reduce Tumor Thermal Tolerance for Mild‐Photothermal Therapy
Li, X., Pan, Y., Chen, C., Gao, Y., Liu, X., Yang, K., Luan, X., Zhou, D., Zeng, F., Han, X., et al. (2021) · Angewandte Chemie International Edition · doi:10.1002/anie.202107036 · Cited by 148
Phytonanotechnology applications in modern agriculture
Jiang, M., Song, Y., Kanwar, M. K., Ahammed, G. J., Shao, S., & Zhou, J. (2021) · Journal of Nanobiotechnology · doi:10.1186/s12951-021-01176-w · Cited by 143
mRNA vaccination with charge-altering releasable transporters elicits human T cell responses and cures established tumors in mice
Haabeth, O. A. W., Blake, T. R., McKinlay, C. J., Waymouth, R. M., Wender, P. A., & Levy, R. (2018) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1810002115 · Cited by 144
Immunogenicity of CRISPR therapeutics—Critical considerations for clinical translation
Ewaisha, R., & Anderson, K. S. (2023) · Frontiers in Bioengineering and Biotechnology · doi:10.3389/fbioe.2023.1138596 · Cited by 153
Nanomedicine-Based Approaches for mRNA Delivery
Uchida, S., Perche, F., Pichon, C., & Cabral, H. (2020) · Molecular Pharmaceutics · doi:10.1021/acs.molpharmaceut.0c00618 · Cited by 139
A Systematic Study of Unsaturation in Lipid Nanoparticles Leads to Improved mRNA Transfection In Vivo
Lee, S. M., Cheng, Q., Yu, X., Liu, S., Johnson, L. T., & Siegwart, D. J. (2020) · Angewandte Chemie International Edition · doi:10.1002/anie.202013927 · Cited by 142
Self-citation
High-throughput barcoding of nanoparticles identifies cationic, degradable lipid-like materials for mRNA delivery to the lungs in female preclinical models
Xue, L., Hamilton, A. G., Zhao, G., Xiao, Z., El-Mayta, R. D., Han, X., Gong, N., Xiong, X., Xu, J., Figueroa‐Espada, C. G., et al. (2024) · Nature Communications · doi:10.1038/s41467-024-45422-9 · Cited by 158
Nanoparticles for nucleic acid delivery: Applications in cancer immunotherapy
Mukalel, A. J., Riley, R., Zhang, R., & Mitchell, M. J. (2019) · Cancer Letters · doi:10.1016/j.canlet.2019.04.040 · Cited by 143
Aptamer/Peptide‐Functionalized Genome‐Editing System for Effective Immune Restoration through Reversal of PD‐L1‐Mediated Cancer Immunosuppression
He, X., Ren, X., Peng, Y., Zhang, J., Ai, S., Liu, B., Xu, C., & Cheng, S. (2020) · Advanced Materials · doi:10.1002/adma.202000208 · Cited by 138
Delivery of Tissue-Targeted Scalpels: Opportunities and Challenges for In Vivo CRISPR/Cas-Based Genome Editing
Wei, T., Cheng, Q., Farbiak, L., Anderson, D. G., Langer, R., & Siegwart, D. J. (2020) · ACS Nano · doi:10.1021/acsnano.0c04707 · Cited by 143
Self-citation
Activating PTEN Tumor Suppressor Expression with the CRISPR/dCas9 System
Moses, C., Nugent, F., Waryah, C. B., García‐Bloj, B., Harvey, A. R., & Blancafort, P. (2018) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2018.12.003 · Cited by 133
Peptide-encoding mRNA barcodes for the high-throughput in vivo screening of libraries of lipid nanoparticles for mRNA delivery
Rhym, L. H., Manan, R. S., Koller, A. A., Stephanie, G., & Anderson, D. G. (2023) · Nature Biomedical Engineering · doi:10.1038/s41551-023-01030-4 · Cited by 137
Engineered Extracellular Vesicle-Delivered CRISPR/Cas9 for Radiotherapy Sensitization of Glioblastoma
Liu, X., Cao, Z., Wang, W., Zou, C., Wang, Y., Pan, L., Jia, B., Zhang, K., Zhang, W., Li, W., et al. (2023) · ACS Nano · doi:10.1021/acsnano.2c12857 · Cited by 132
Current Status and Future Perspectives on MRNA Drug Manufacturing
Webb, C., Ip, S. Y., Bathula, N. V., Popova, P. G., Soriano, S. K. V., Ly, B.-H., Eryilmaz, B., Huu, V. A. N., Broadhead, R., Rabel, M., et al. (2022) · Molecular Pharmaceutics · doi:10.1021/acs.molpharmaceut.2c00010 · Cited by 134
Nanoplatforms for mRNA Therapeutics
Meng, C., Chen, Z., Li, G., Welte, T., & Shen, H. (2021) · Advanced Therapeutics · doi:10.1002/adtp.202000099 · Cited by 130
Nanoparticle-Based Delivery of CRISPR/Cas9 Genome-Editing Therapeutics
Givens, B. E., Naguib, Y. W., Geary, S. M., Devor, E. J., & Salem, A. K. (2018) · The AAPS Journal · doi:10.1208/s12248-018-0267-9 · Cited by 133
Enhancing RNA-lipid nanoparticle delivery: Organ- and cell-specificity and barcoding strategies
Wei, P.-S., Thota, N., John, G., Chang, E., Lee, S., Wang, Y., Ma, Z., Tsai, Y.-H., & Mei, K. (2024) · Journal of Controlled Release · doi:10.1016/j.jconrel.2024.08.030 · Cited by 138
Customizable Lipid Nanoparticle Materials for the Delivery of siRNAs and mRNAs
Fenton, O. S., Kauffman, K. J., McClellan, R. L., Kaczmarek, J. C., Zeng, M. D., Andresen, J. L., Rhym, L. H., Heartlein, M. W., DeRosa, F., & Anderson, D. G. (2018) · Angewandte Chemie International Edition · doi:10.1002/anie.201809056 · Cited by 129
Nanoparticle Delivery Systems with Cell-Specific Targeting for Pulmonary Diseases
Deng, Z., Kalin, G. T., Shi, D., & Kalinichenko, V. V. (2020) · American Journal of Respiratory Cell and Molecular Biology · doi:10.1165/rcmb.2020-0306tr · Cited by 134
Biomedical applications of mRNA nanomedicine
Xiong, Q., Lee, G. Y., Ding, J., Li, W., & Shi, J. (2018) · Nano Research · doi:10.1007/s12274-018-2146-1 · Cited by 125
Applications of CRISPR-Cas Enzymes in Cancer Therapeutics and Detection
Huang, C., Lee, K.-C., & Doudna, J. A. (2018) · Trends in cancer · doi:10.1016/j.trecan.2018.05.006 · Cited by 119
mRNA therapeutics deliver a hopeful message
Zhong, Z., Cafferty, S. M., Combes, F., Huysmans, H., Temmerman, J. D., Gitsels, A., Vanrompay, D. C. G., Catani, J. P., & Sanders, N. N. (2018) · Nano Today · doi:10.1016/j.nantod.2018.10.005 · Cited by 124
Material solutions for delivery of CRISPR/Cas-based genome editing tools: Current status and future outlook
Wan, T. Y., Niu, D., Wu, C., Xu, F., Church, G. M., & Yuan, P. (2019) · Materials Today · doi:10.1016/j.mattod.2018.12.003 · Cited by 119
Tumor-Activated Water-Soluble Photosensitizers for Near-Infrared Photodynamic Cancer Therapy
Xiong, H., Zhou, K., Yan, Y., Miller, J. B., & Siegwart, D. J. (2018) · ACS Applied Materials & Interfaces · doi:10.1021/acsami.8b04710 · Cited by 116
Self-citation
Non-Viral Delivery To Enable Genome Editing
Rui, Y., Wilson, D. R., & Green, J. J. (2018) · Trends in biotechnology · doi:10.1016/j.tibtech.2018.08.010 · Cited by 115
Systemic mRNA delivery to the lungs by functional polyester-based carriers
Yan, Y., Xiong, H., Zhang, X., Cheng, Q., & Siegwart, D. J. (2017) · Biomacromolecules · doi:10.1021/acs.biomac.7b01356 · Cited by 111
Self-citation
Engineering of monosized lipid-coated mesoporous silica nanoparticles for CRISPR delivery
Noureddine, A., Maestas-Olguin, A., Saada, E. A., LaBauve, A. E., Agola, J. O., Baty, K. E., Howard, T. A., Sabo, J. K., Espinoza, C. R. S., Doudna, J. A., et al. (2020) · Acta Biomaterialia · doi:10.1016/j.actbio.2020.07.027 · Cited by 113
Cationic Polymer‐Mediated CRISPR/Cas9 Plasmid Delivery for Genome Editing
Zhang, Z., Wan, T. Y., Chen, Y., Chen, Y., Sun, H., Cao, T., Songyang, Z., Tang, G., Wu, C., Yuan, P., et al. (2018) · Macromolecular Rapid Communications · doi:10.1002/marc.201800068 · Cited by 109
Improved Nucleic Acid Therapy with Advanced Nanoscale Biotechnology
Weng, Y., Huang, Q., Li, C., Yang, Y., Wang, X., Yu, J., Huang, Y., & Liang, X. (2019) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2019.12.004 · Cited by 110
Biodegradable Amino-Ester Nanomaterials for Cas9 mRNA Delivery in Vitro and in Vivo
Zhang, X., Li, B., Luo, X., Zhao, W., Jiang, J., Zhang, C., Gao, M., Chen, X., & Dong, Y. (2017) · ACS Applied Materials & Interfaces · doi:10.1021/acsami.7b08163 · Cited by 107
Different kinetics for the hepatic uptake of lipid nanoparticles between the apolipoprotein E/low density lipoprotein receptor and the N-acetyl-d-galactosamine/asialoglycoprotein receptor pathway
Sato, Y., Kinami, Y., Hashiba, K., & Harashima, H. (2020) · Journal of Controlled Release · doi:10.1016/j.jconrel.2020.03.006 · Cited by 112
Recent advances in using liposomes for delivery of nucleic acid-based therapeutics
Nsairat, H., Alshaer, W., Odeh, F., Esawi, E., Khater, D., Bawab, A. A., El‐Tanani, M., Awidi, A., & Mubarak, M. S. (2023) · OpenNano · doi:10.1016/j.onano.2023.100132 · Cited by 119
The dichotomy of nanotechnology as the cutting edge of agriculture: Nano-farming as an asset versus nanotoxicity
Behl, T., Kaur, I., Sehgal, A., Singh, S., Sharma, N., Bhatia, S., Al‐Harrasi, A., & BUNGAU, S. G. (2021) · Chemosphere · doi:10.1016/j.chemosphere.2021.132533 · Cited by 106
Recent advances in nanoparticulate RNA delivery systems
Witten, J. J., Hu, Y., Langer, R., & Anderson, D. G. (2024) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.2307798120 · Cited by 113
Rapid, Single-Cell Analysis and Discovery of Vectored mRNA Transfection In Vivo with a loxP-Flanked tdTomato Reporter Mouse
Kauffman, K. J., Oberli, M. A., Dorkin, J. R., Hurtado, J. E., Kaczmarek, J. C., Bhadani, S., Wyckoff, J., Langer, R., Jaklenec, A., & Anderson, D. G. (2017) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2017.11.005 · Cited by 104
Lipid nanoparticle-based strategies for extrahepatic delivery of nucleic acid therapies – challenges and opportunities
Simonsen, J. B. (2024) · Journal of Controlled Release · doi:10.1016/j.jconrel.2024.04.022 · Cited by 115
Ultrasound-Controlled CRISPR/Cas9 System Augments Sonodynamic Therapy of Hepatocellular Carcinoma
Yin, H., Sun, L., Pu, Y., Yu, J., Feng, W., Dong, C., Zhou, B., Du, D., Zhang, Y., Chen, Y., et al. (2021) · ACS Central Science · doi:10.1021/acscentsci.1c01143 · Cited by 102
Gold Nanocluster-Mediated Efficient Delivery of Cas9 Protein through pH-Induced Assembly-Disassembly for Inactivation of Virus Oncogenes
Ju, E., Li, T., da Silva, S. R., & Gao, S. (2019) · ACS Applied Materials & Interfaces · doi:10.1021/acsami.9b12335 · Cited by 103
Overcoming the delivery problem for therapeutic genome editing: Current status and perspective of non-viral methods
Mashel, T. V., Tarakanchikova, Y. V., Muslimov, A. R., Zyuzin, M. V., Timin, A. S., Lepik, K. V., & Fehse, B. (2020) · Biomaterials · doi:10.1016/j.biomaterials.2020.120282 · Cited by 102
Polymeric Carriers for Nucleic Acid Delivery: Current Designs and Future Directions
Peng, L., & Wagner, E. (2019) · Biomacromolecules · doi:10.1021/acs.biomac.9b00999 · Cited by 102
All‐In‐One Dendrimer‐Based Lipid Nanoparticles Enable Precise HDR‐Mediated Gene Editing In Vivo
Farbiak, L., Cheng, Q., Wei, T., Álvarez‐Benedicto, E., Johnson, L. T., Lee, S. M., & Siegwart, D. J. (2021) · Advanced Materials · doi:10.1002/adma.202006619 · Cited by 103
Self-citation
Targeted delivery of CRISPR interference system against Fabp4 to white adipocytes ameliorates obesity, inflammation, hepatic steatosis, and insulin resistancePreprint
Chung, J. Y., Ain, Q. U., Song, Y., Yong, S., & Kim, Y. (2019) · Genome Research · doi:10.1101/gr.246900.118 · Cited by 102
Messenger RNA Delivery for Tissue Engineering and Regenerative Medicine Applications
Patel, S., Athirasala, A., Menezes, P. D. P., Ashwanikumar, N., Zou, T., Sahay, G., & Bertassoni, L. E. (2018) · Tissue Engineering Part A · doi:10.1089/ten.tea.2017.0444 · Cited by 101
Co-encapsulation of Cas9 mRNA and guide RNA in polyplex micelles enables genome editing in mouse brain
Abbasi, S., Uchida, S., Toh, K., Tockary, T. A., Dirisala, A., Hayashi, K., Fukushima, S., & Kataoka, K. (2021) · Journal of Controlled Release · doi:10.1016/j.jconrel.2021.02.026 · Cited by 100
Cell-Selective Messenger RNA Delivery and CRISPR/Cas9 Genome Editing by Modulating the Interface of Phenylboronic Acid-Derived Lipid Nanoparticles and Cellular Surface Sialic Acid
Tang, Q., Liu, J., Jiang, Y. Y., Zhang, M., Mao, L., & Wang, M. (2019) · ACS Applied Materials & Interfaces · doi:10.1021/acsami.9b17749 · Cited by 99
Lipid nanoparticle chemistry determines how nucleoside base modifications alter mRNA delivery
Melamed, J. R., Hajj, K. A., Chaudhary, N., Strelkova, D., Arral, M. L., Pardi, N., Alameh, M., Miller, J. B., Farbiak, L., Siegwart, D. J., et al. (2021) · Journal of Controlled Release · doi:10.1016/j.jconrel.2021.11.022 · Cited by 101
Self-citation
In situ repurposing of dendritic cells with CRISPR/Cas9-based nanomedicine to induce transplant tolerance
Zhang, Y., Shen, S., Zhao, G., Xu, C., Zhang, H., Luo, Y., Cao, Z., Shi, J. Y., Zhao, Z., Lian, Z., et al. (2019) · Biomaterials · doi:10.1016/j.biomaterials.2019.119302 · Cited by 96
Recent advances in chemical modifications of guide RNA, mRNA and donor template for CRISPR-mediated genome editing
Chen, Q., Zhang, Y., & Yin, H. (2020) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2020.10.014 · Cited by 99
Non-viral Delivery of Zinc Finger Nuclease mRNA Enables Highly Efficient In Vivo Genome Editing of Multiple Therapeutic Gene Targets
Conway, A., Mendel, M., Kim, K., McGovern, K., Boyko, A., Zhang, L., Miller, J. C., DeKelver, R. C., Paschon, D. E., Mui, B. L., et al. (2019) · Molecular Therapy · doi:10.1016/j.ymthe.2019.03.003 · Cited by 95
Self-citation
Theranostic dendrimer-based lipid nanoparticles containing PEGylated BODIPY dyes for tumor imaging and systemic mRNA delivery in vivo
Xiong, H., Liu, S., Wei, T., Cheng, Q., & Siegwart, D. J. (2020) · Journal of Controlled Release · doi:10.1016/j.jconrel.2020.06.030 · Cited by 92
Self-citation
Far-red light-mediated programmable anti-cancer gene delivery in cooperation with photodynamic therapy
Wang, J., He, H., Xu, X., Wang, X., Chen, Y., & Yin, L. (2018) · Biomaterials · doi:10.1016/j.biomaterials.2018.04.020 · Cited by 91
Fine-Tuning of Hydrophobicity in Amphiphilic Polyaspartamide Derivatives for Rapid and Transient Expression of Messenger RNA Directed Toward Genome Engineering in Brain
Kim, H. J., Ogura, S., Otabe, T., Kamegawa, R., Sato, M., Kataoka, K., & Miyata, K. (2019) · ACS Central Science · doi:10.1021/acscentsci.9b00843 · Cited by 90
Biomaterials as vectors for the delivery of CRISPR–Cas9
Eoh, J. H., & Gu, L. (2019) · Biomaterials Science · doi:10.1039/c8bm01310a · Cited by 92
Optimized lipid nanoparticles (LNPs) for organ-selective nucleic acids delivery in vivo
Zhang, T., Yin, H., Li, Y., Yang, H., Ge, K., Zhang, J., Yuan, Q., Dai, X., Naeem, A., Weng, Y., et al. (2024) · iScience · doi:10.1016/j.isci.2024.109804 · Cited by 99
Frameworks for transformational breakthroughs in RNA-based medicines
Androsavich, J. R. (2024) · Nature Reviews Drug Discovery · doi:10.1038/s41573-024-00943-2 · Cited by 100
CRISPR-Cas9 library screening approach for anti-cancer drug discovery: overview and perspectives
Chan, Y.-T., Lu, Y., Wu, J., Zhang, C., Tan, H., Bian, Z., Wang, N., & Feng, Y. (2022) · Theranostics · doi:10.7150/thno.71144 · Cited by 93
Non-viral gene therapy using multifunctional nanoparticles: Status, challenges, and opportunities
Lin, G., Li, L., Panwar, N., Wang, J., Tjin, S. C., Wang, X., & Yong, K. (2018) · Coordination Chemistry Reviews · doi:10.1016/j.ccr.2018.07.001 · Cited by 88
mRNA nanodelivery systems: targeting strategies and administration routes
Yuan, M., Han, Z., Liang, Y., Sun, Y., He, B., Chen, W., & Fan, L. (2023) · Biomaterials Research · doi:10.1186/s40824-023-00425-3 · Cited by 98
High‐Contrast Fluorescence Detection of Metastatic Breast Cancer Including Bone and Liver Micrometastases via Size‐Controlled pH‐Activatable Water‐Soluble Probes
Xiong, H., Zuo, H., Yan, Y., Occhialini, G., Zhou, K., Wan, Y., & Siegwart, D. J. (2017) · Advanced Materials · doi:10.1002/adma.201700131 · Cited by 86
Self-citation
Lipid Polymer Hybrid Nanomaterials for mRNA Delivery
Zhao, W., Zhang, C., Li, B., Zhang, X., Luo, X. L., Zeng, C., Li, W., Gao, M., & Dong, Y. (2018) · Cellular and Molecular Bioengineering · doi:10.1007/s12195-018-0536-9 · Cited by 86
A comprehensive review on regulatory invention of nano pesticides in Agricultural nano formulation and food system
Raj, S. N., Anooj, E. S., Rajendran, K., & Vallinayagam, S. (2021) · Journal of Molecular Structure · doi:10.1016/j.molstruc.2021.130517 · Cited by 84
The mixing method used to formulate lipid nanoparticles affects mRNA delivery efficacy and organ tropism
Petersen, D. M. S., Chaudhary, N., Arral, M. L., Weiss, R. M., & Whitehead, K. A. (2023) · European Journal of Pharmaceutics and Biopharmaceutics · doi:10.1016/j.ejpb.2023.10.006 · Cited by 86
Exosome/Liposome-like Nanoparticles: New Carriers for CRISPR Genome Editing in Plants
Alghuthaymi, M. A., Ahmad, A., Khan, Z., Khan, S. H., Ahmed, F. K., Faiz, S., Nepovimova, E., Kuča, K., & Abd-Elsalam, K. A. (2021) · International Journal of Molecular Sciences · doi:10.3390/ijms22147456 · Cited by 83
Testing thousands of nanoparticles in vivo using DNA barcodes
Lokugamage, M. P., Sago, C. D., & Dahlman, J. E. (2018) · Current Opinion in Biomedical Engineering · doi:10.1016/j.cobme.2018.08.001 · Cited by 84
Spatiotemporal Delivery of CRISPR/Cas9 Genome Editing Machinery Using Stimuli‐Responsive Vehicles
Cai, W., Luo, T., Mao, L., & Wang, M. (2020) · Angewandte Chemie International Edition · doi:10.1002/anie.202005644 · Cited by 83
RNA delivery biomaterials for the treatment of genetic and rare diseases
Zhao, W., Hou, X., Vick, O. G., & Dong, Y. (2019) · Biomaterials · doi:10.1016/j.biomaterials.2019.119291 · Cited by 82
In Vivo Introduction of mRNA Encapsulated in Lipid Nanoparticles to Brain Neuronal Cells and Astrocytes via Intracerebroventricular Administration
Tanaka, H., Nakatani, T., Furihata, T., Tange, K., Nakai, Y., Yoshioka, H., Harashima, H., & Akita, H. (2018) · Molecular Pharmaceutics · doi:10.1021/acs.molpharmaceut.7b01084 · Cited by 82
Optimizing synthetic nucleic acid and protein nanocarriers: The chemical evolution approach
Freitag, F., & Wagner, E. (2020) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2020.03.005 · Cited by 83
Harnessing nanoparticles for the efficient delivery of the CRISPR/Cas9 system
Rahimi, H., Salehiabar, M., Charmi, J., Barsbay, M., Ghaffarlou, M., Razlighi, M. R., Davaran, S., Khalilov, R. I., Sugiyama, M., Nosrati, H., et al. (2020) · Nano Today · doi:10.1016/j.nantod.2020.100895 · Cited by 83
Overcoming the Blood–Brain Barrier for Gene Therapy via Systemic Administration of GSH‐Responsive Silica Nanocapsules
Wang, Y., Wang, X., Xie, R., Burger, J. C., Tong, Y., & Gong, S. (2022) · Advanced Materials · doi:10.1002/adma.202208018 · Cited by 84
Novel zwitterionic vectors: Multi-functional delivery systems for therapeutic genes and drugs
Zhou, L., Zhu, Y., Wang, X., Shen, C., Wei, X., Xu, T., & He, Z. (2020) · Computational and Structural Biotechnology Journal · doi:10.1016/j.csbj.2020.07.015 · Cited by 81
Lysosomal-mediated drug release and activation for cancer therapy and immunotherapy
Sun, Y., Sha, Y., Cui, G., Meng, F., & Zhong, Z. (2022) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2022.114624 · Cited by 78
CRISPR/Cas9 Genome Editing for Tissue‐Specific In Vivo Targeting: Nanomaterials and Translational Perspective
Sahel, D. K., Vora, L. K., Saraswat, A. L., Sharma, S., Monpara, J. D., D’souza, A. A., Mishra, D., Tryphena, K. P., Kawakita, S., Khan, S. U., et al. (2023) · Advanced Science · doi:10.1002/advs.202207512 · Cited by 79
Synthetic multi-layer nanoparticles for CRISPR-Cas9 genome editing
Tang, H., Zhao, X., & Jiang, X. (2020) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2020.03.001 · Cited by 80
Lipid Nanoparticles for Organ-Specific mRNA Therapeutic Delivery
Żak, M. M., & Zangi, L. (2021) · Pharmaceutics · doi:10.3390/pharmaceutics13101675 · Cited by 75
Targeted lipid nanoparticles for RNA therapeutics and immunomodulation in leukocytes
Veiga, N., Diesendruck, Y., & Peer, D. (2020) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2020.04.002 · Cited by 78
Advances in Nanoparticles as Non-Viral Vectors for Efficient Delivery of CRISPR/Cas9
Kim, M., Hwang, Y., Lim, S., Jang, H., & Kim, H. (2024) · Pharmaceutics · doi:10.3390/pharmaceutics16091197 · Cited by 77
Block and Lock HIV Cure Strategies to Control the Latent Reservoir
Ahlenstiel, C., Symonds, G. P., Kent, S. J., & Kelleher, A. D. (2020) · Frontiers in Cellular and Infection Microbiology · doi:10.3389/fcimb.2020.00424 · Cited by 73
mRNA – A game changer in regenerative medicine, cell-based therapy and reprogramming strategies
Chabanovska, O., Galow, A.-M., David, R., & Lemcke, H. (2021) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2021.114002 · Cited by 81
Augmented lipid-nanoparticle-mediated in vivo genome editing in the lungs and spleen by disrupting Cas9 activity in the liver
Sago, C. D., Lokugamage, M. P., Loughrey, D., Lindsay, K. E., Hincapie, R., Krupczak, B. R., Kalathoor, S., Sato, M., Echeverri, E. S., Fitzgerald, J. P., et al. (2022) · Nature Biomedical Engineering · doi:10.1038/s41551-022-00847-9 · Cited by 75
Use of Microfluidics to Prepare Lipid-Based Nanocarriers
Vogelaar, A., Marcotte, S., Cheng, J., Oluoch, B., & Zaro, J. L. (2023) · Pharmaceutics · doi:10.3390/pharmaceutics15041053 · Cited by 76
Poly(Beta-Amino Ester) Nanoparticles Enable Nonviral Delivery of CRISPR-Cas9 Plasmids for Gene Knockout and Gene Deletion
Rui, Y., Varanasi, M., Mendes, S., Yamagata, H. M., Wilson, D. R., & Green, J. J. (2020) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2020.04.005 · Cited by 71
Nanoparticles-mediated CRISPR/Cas9 delivery: Recent advances in cancer treatment
Aghamiri, S., Talaei, S., Ghavidel, A. A., Zandsalimi, F., Masoumi, S., Hafshejani, N. H., & Jajarmi, V. (2020) · Journal of Drug Delivery Science and Technology · doi:10.1016/j.jddst.2020.101533 · Cited by 70
Harnessing lipid nanoparticles for efficient CRISPR delivery
Yan, J., Kang, D. D., & Dong, Y. (2021) · Biomaterials Science · doi:10.1039/d1bm00537e · Cited by 69
TRIAMF: A New Method for Delivery of Cas9 Ribonucleoprotein Complex to Human Hematopoietic Stem Cells
Yen, J. S., Fiorino, M., Liu, Y., Paula, S., Clarkson, S., Quinn, L., Tschantz, W. R., Klock, H. E., Guo, N., Russ, C., et al. (2018) · Scientific Reports · doi:10.1038/s41598-018-34601-6 · Cited by 65
Current updates of CRISPR/Cas9‐mediated genome editing and targeting within tumor cells: an innovative strategy of cancer management
Allemailem, K. S., Alsahli, M. A., Almatroudi, A., Alrumaihi, F., Alkhaleefah, F. K., Rahmani, A. H., & Khan, A. A. (2022) · 癌症：英文版 · doi:10.1002/cac2.12366 · Cited by 69
Non-viral delivery of chemically modified mRNA to the retina: Subretinal versus intravitreal administration
Devoldere, J., Peynshaert, K., Dewitte, H., Vanhove, C., Groef, L. D., Moons, L., Özcan, S. Y., Dalkara, D., Smedt, S. C. D., & Remaut, K. (2019) · Journal of Controlled Release · doi:10.1016/j.jconrel.2019.06.042 · Cited by 70
The use of design of experiments with multiple responses to determine optimal formulations for in vivo hepatic mRNA delivery
Hashiba, A., Toyooka, M., Sato, Y., Maeki, M., Tokeshi, M., & Harashima, H. (2020) · Journal of Controlled Release · doi:10.1016/j.jconrel.2020.08.031 · Cited by 66
NanoRNP Overcomes Tumor Heterogeneity in Cancer Treatment
Liu, Q., Cai, J., Zheng, Y., Tan, Y., Wang, Y., Zhang, Z., Zheng, C., Zhao, Y., Liu, C., An, Y., et al. (2019) · Nano Letters · doi:10.1021/acs.nanolett.9b02501 · Cited by 67
Degradable redox-responsive disulfide-based nanogel drug carriers via dithiol oxidation polymerization
Elkassih, S. A., Kós, P., Xiong, H., & Siegwart, D. J. (2018) · Biomaterials Science · doi:10.1039/c8bm01120f · Cited by 65
Self-citation
Engineered Nanomaterials to Potentiate CRISPR/Cas9 Gene Editing for Cancer Therapy
Yi, K., Kong, H., Lao, Y., Li, D., Mintz, R. L., Fang, T., Chen, G., Tao, Y., Li, M., & Ding, J. (2023) · Advanced Materials · doi:10.1002/adma.202300665 · Cited by 66
Creating cell and animal models of human disease by genome editing using CRISPR/Cas9
Zarei, A., Razban, V., Hosseini, S. E., & Tabei, K. (2019) · The Journal of Gene Medicine · doi:10.1002/jgm.3082 · Cited by 68
Co-delivery of p38α MAPK and p65 siRNA by novel liposomal glomerulus-targeting nano carriers for effective immunoglobulin a nephropathy treatment
Wang, Y., Wu, Q., Wang, J., Li, L., Sun, X., Zhang, Z., & Zhang, L. (2020) · Journal of Controlled Release · doi:10.1016/j.jconrel.2020.01.024 · Cited by 65
Non-viral strategies for delivering genome editing enzymes
Li, J., Røise, J. J., He, M., Das, R., & Murthy, N. (2020) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2020.09.004 · Cited by 63
Spatial and Temporal Control of CRISPR-Cas9-Mediated Gene Editing Delivered via a Light-Triggered Liposome System
Aksoy, Y. A., Yang, B., Chen, W., Hung, T., Kuchel, R. P., Zammit, N. W., Grey, S. T., Goldys, E. M., & Deng, W. (2020) · ACS Applied Materials & Interfaces · doi:10.1021/acsami.0c16380 · Cited by 63
Influence of ionizable lipid tail length on lipid nanoparticle delivery of mRNA of varying length
Mrksich, K., Padilla, M. S., Joseph, R. A., Han, E. L., Kim, D., Palanki, R., Xu, J., & Mitchell, M. J. (2024) · Journal of Biomedical Materials Research Part A · doi:10.1002/jbm.a.37705 · Cited by 67
Aptamer-Based Erythrocyte-Derived Mimic Vesicles Loaded with siRNA and Doxorubicin for the Targeted Treatment of Multidrug-Resistant Tumors
Wang, T., Luo, Y., Lv, H., Wang, J., Zhang, Y., & Pei, R. (2019) · ACS Applied Materials & Interfaces · doi:10.1021/acsami.9b16637 · Cited by 65
Chemotherapy drugs derived nanoparticles encapsulating mRNA encoding tumor suppressor proteins to treat triple-negative breast cancer
Zhang, C., Zhang, X., Zhao, W., Zeng, C., Li, W., Li, B., Luo, X., Li, J., Jiang, J., Deng, B., et al. (2019) · Nano Research · doi:10.1007/s12274-019-2308-9 · Cited by 61

Authors: Miller, J. B., Zhang, S., Kos, P., Xiong, H., Zhou, K., Perelman, S. S., Zhu, H., & Siegwart, D. J.
Affiliations: Harold C. Simmons Comprehensive Cancer Center (US)
The University of Texas Southwestern Medical Center (US)
University of Texas Southwestern Medical Center Simmons Comprehensive Cancer Center Department of Biochemistry Dallas TX 75390 USA
Corresponding author: Siegwart, Daniel J.
ORCID: Miller, Jason B.
Zhang, Shuyuan
Xiong, Hu
Perelman, Sofya S.
Zhu, Hao
Siegwart, Daniel J.
Venue: Angewandte Chemie International Edition
Publisher: Wiley
Published: 2016-12-16
Volume: 56
Issue: 4
Pages: 1059-1063
ISSN: 1433-7851 (ISSN-L)
1521-3773
DOI: 10.1002/anie.201610209
PMC: PMC5521011
OpenAlex: W2560987571
Cited by: 540
References: 45
Citations by year: 2026: 35 · 2025: 39 · 2024: 47 · 2023: 48 · 2022: 66 · 2021: 86 · 2020: 79 · 2019: 59 · 2018: 55 · 2017: 24 · 2016: 1
FWCI: 24.09
Type: journal article
Language: en
Open access: green
www.ncbi.nlm.nih.gov
Licence: onlinelibrary.wiley.com/termsAndConditions#vor (vor)
Locations: Angewandte Chemie International Edition (publishedVersion)
PubMed (publishedVersion)
PubMed Central (acceptedVersion)
Funders: National Cancer Institute (R01CA190525)
Topics: Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › CRISPR and Genetic Engineering
Physical Sciences › Materials Science › Biomaterials › Nanoparticle-Based Drug Delivery
Keywords: CRISPR gene editing, lipid nanoparticles, Cas9 mRNA, single guide RNA, co-delivery, in vivo gene editing, loxP
MeSH terms: Gene Editing, RNA, Guide, CRISPR-Cas Systems, Lipids, RNA, Messenger, Molecular Structure, RNA, Guide, Kinetoplastida, Gene Transfer Techniques, Nanoparticles, CRISPR-Cas Systems

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

Non‐Viral CRISPR/Cas Gene Editing In Vitro and In Vivo Enabled by Synthetic Nanoparticle Co‐Delivery of Cas9 mRNA and sgRNA

Abstract

Overview

Integrity signals

Verification

Claims the paper makes

Claims other papers make about this one

Self-check

Citations

Cites

Cited by

Related

Metadata

Record

About these checks