Delivery technologies for genome editing

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

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

The new frontier of genome engineering with CRISPR-Cas9

Doudna, J. A., & Charpentier, E. (2014) · Science · doi:10.1126/science.1258096 · Cited by 7,062

DNA targeting specificity of RNA-guided Cas9 nucleases

Hsu, P. D., Scott, D. A., Weinstein, J. A., Ran, F. A., Konermann, S., Agarwala, V., Li, Y., Fine, E. J., Wu, X., Shalem, O., et al. (2013) · Nature Biotechnology · doi:10.1038/nbt.2647 · Cited by 4,741

Cystic Fibrosis

Webb, A. K. (2006) · Cited by 4,369

LMO2 -Associated Clonal T Cell Proliferation in Two Patients after Gene Therapy for SCID-X1

Hacein-Bey-Abina, S., Von Kalle, C., Schmidt, M., McCormack, M. P., Wulffraat, N., Leboulch, P., Lim, A., Osborne, C. S., Pawliuk, R., Morillon, E., et al. (2003) · Science · doi:10.1126/science.1088547 · Cited by 3,587

One-step generation of mice carrying mutations in multiple genes by CRISPR/Cas-mediated genome engineering

Wang, H., Yang, H., Shivalila, C. S., Dawlaty, M. M., Cheng, A. W., Zhang, F., & Jaenisch, R. (2013) · Cell · doi:10.1016/j.cell.2013.04.025 · Cited by 3,495

Double nicking by RNA-guided CRISPR Cas9 for enhanced genome editing specificity

Ran, F. A., Hsu, P. D., Lin, C.-Y., Gootenberg, J. S., Konermann, S., Trevino, A. E., Scott, D. A., Inoue, A., Matoba, S., Zhang, Y., et al. (2013) · Cell · doi:10.1016/j.cell.2013.08.021 · Cited by 3,299

Non-viral vectors for gene-based therapy

Yin, H., Kanasty, R. L., Eltoukhy, A. A., Vegas, A. J., Dorkin, J. R., & Anderson, D. G. (2014) · Nature Reviews Genetics · doi:10.1038/nrg3763 · Cited by 3,123

In vivo genome editing using Staphylococcus aureus Cas9

Ran, F. A., Cong, L., Yan, W. X., Scott, D. A., Gootenberg, J. S., Kriz, A. J., Zetsche, B., Shalem, O., Wu, X., Makarova, K. S., et al. (2015) · Nature · doi:10.1038/nature14299 · Cited by 2,687

Engineering precision nanoparticles for drug delivery

Mitchell, M. J., Billingsley, M. M., Haley, R. M., Wechsler, M. E., Peppas, N. A., & Langer, R. (2021) · Nature Reviews Drug Discovery · doi:10.1038/s41573-020-0090-8 · Cited by 7,933

Applications of genome editing technology in the targeted therapy of human diseases: mechanisms, advances and prospects

Li, H., Yang, Y., Hong, W., Huang, M., Wu, M., & Zhao, X. (2020) · Signal Transduction and Targeted Therapy · doi:10.1038/s41392-019-0089-y · Cited by 1,829

Block copolymer micelles in nanomedicine applications

Cabral, H., Miyata, K., Osada, K., & Kataoka, K. (2018) · Chemical Reviews · doi:10.1021/acs.chemrev.8b00199 · Cited by 1,309

Delivering the Messenger: Advances in Technologies for Therapeutic mRNA Delivery

Kowalski, P. S., Rudra, A., Miao, L., & Anderson, D. G. (2019) · Molecular Therapy · doi:10.1016/j.ymthe.2019.02.012 · Cited by 1,127

Lysosomal storage diseases

Platt, F. M., d’Azzo, A., Davidson, B. L., Neufeld, E. F., & Tifft, C. J. (2018) · Nature Reviews Disease Primers · doi:10.1038/s41572-018-0025-4 · Cited by 1,052

Intracellular Delivery by Membrane Disruption: Mechanisms, Strategies, and Concepts

Stewart, M. P., Langer, R., & Jensen, K. F. (2018) · Chemical Reviews · doi:10.1021/acs.chemrev.7b00678 · Cited by 767

Proteolysis-targeting chimera (PROTAC) for targeted protein degradation and cancer therapy

Li, X., & Song, Y. (2020) · Journal of Hematology & Oncology · doi:10.1186/s13045-020-00885-3 · Cited by 559

Therapeutic in vivo delivery of gene editing agents

Raguram, A., Banskota, S., & Liu, D. R. (2022) · Cell · doi:10.1016/j.cell.2022.03.045 · Cited by 552

CRISPR-Cas9 DNA Base-Editing and Prime-Editing

Kantor, A., McClements, M., & MacLaren, R. (2020) · International Journal of Molecular Sciences · doi:10.3390/ijms21176240 · Cited by 462

DNA Repair Pathway Choices in CRISPR-Cas9-Mediated Genome Editing

Xue, C., & Greene, E. C. (2021) · Trends in Genetics · doi:10.1016/j.tig.2021.02.008 · Cited by 431

Non-viral delivery systems for CRISPR/Cas9-based genome editing: challenges and opportunities

Li, L., Hu, S., & Chen, X. (2018) · Biomaterials · doi:10.1016/j.biomaterials.2018.04.031 · Cited by 415

The great escape: how cationic polyplexes overcome the endosomal barrier

Buś, T., Traeger, A., & Schubert, U. S. (2018) · Journal of Materials Chemistry B · doi:10.1039/c8tb00967h · Cited by 422

Fast and Efficient CRISPR/Cas9 Genome Editing In Vivo Enabled by Bioreducible Lipid and Messenger RNA Nanoparticles

Liu, J., Chang, J., Jiang, Y., Meng, X., Sun, T., Mao, L., Xu, Q., & Wang, M. (2019) · Advanced Materials · doi:10.1002/adma.201902575 · Cited by 373

Polymeric Delivery of Therapeutic Nucleic Acids

Kumar, R., Chalarca, C. F. S., Bockman, M. R., Bruggen, C. V., Grimme, C. J., Dalal, R. J., Hanson, M. G., Hexum, J. K., & Reineke, T. M. (2021) · Chemical Reviews · doi:10.1021/acs.chemrev.0c00997 · Cited by 379

Engineering living therapeutics with synthetic biology

Cubillos-Ruiz, A., Guo, T., Sokolovska, A., Miller, P., Collins, J. J., Lu, T. K., & Lora, J. M. (2021) · Nature Reviews Drug Discovery · doi:10.1038/s41573-021-00285-3 · Cited by 387

A biodegradable nanocapsule delivers a Cas9 ribonucleoprotein complex for in vivo genome editing

Chen, G., Abdeen, A. A., Wang, Y., Shahi, P. K., Robertson, S., Xie, R., Suzuki, M., Pattnaik, B. R., Saha, K., & Gong, S. (2019) · Nature Nanotechnology · doi:10.1038/s41565-019-0539-2 · Cited by 361

Highly efficient DNA-free plant genome editing using virally delivered CRISPR–Cas9

Ma, X., Zhang, X., Liu, H., & Li, Z. (2020) · Nature Plants · doi:10.1038/s41477-020-0704-5 · Cited by 362

Therapies for rare diseases: therapeutic modalities, progress and challenges ahead

Tambuyzer, E., Vandendriessche, B., Austin, C. P., Brooks, P. J., Larsson, K., Miller Needleman, K. I., Valentine, J., Davies, K., Groft, S. C., Preti, R., et al. (2020) · Nature Reviews Drug Discovery · doi:10.1038/s41573-019-0049-9 · Cited by 357

DNA hydrogel-based gene editing and drug delivery systems

Mo, F., Jiang, K., Zhao, D. S., Wang, Y., Song, J., & Tan, W. (2020) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2020.07.018 · Cited by 309

Combinatorial design of nanoparticles for pulmonary mRNA delivery and genome editing

Li, B., Manan, R. S., Liang, S., Gordon, A. G. R., Jiang, A. Y., Varley, A., Gao, G., Langer, R., Xue, W., & Anderson, D. G. (2023) · Nature Biotechnology · doi:10.1038/s41587-023-01679-x · Cited by 298

Emerging non-viral vectors for gene delivery

Wang, C., Pan, C., Yong, H., Wang, F., Bo, T., Zhao, Y., Ma, B., He, W., & Li, M. (2023) · Journal of Nanobiotechnology · doi:10.1186/s12951-023-02044-5 · Cited by 325

Programmable RNA editing by recruiting endogenous ADAR using engineered RNAs

Qu, L., Yi, Z., Zhu, S., Wang, C., Cao, Z., Zhou, Z. L., Yuan, P., Yu, Y. F., Tian, F., Liu, Z., et al. (2019) · Nature Biotechnology · doi:10.1038/s41587-019-0178-z · Cited by 292

Engineering and physical sciences in oncology: challenges and opportunities

Mitchell, M. J., Jain, R. K., & Langer, R. (2017) · Nature Reviews Cancer · doi:10.1038/nrc.2017.83 · Cited by 273

Metal-organic frameworks for precise inclusion of single-stranded DNA and transfection in immune cells

Peng, S., Bie, B., Sun, Y., Liu, M., Cong, H., Zhou, W., Xia, Y., Tang, H. Y., Deng, H., & Zhou, X. (2018) · Nature Communications · doi:10.1038/s41467-018-03650-w · Cited by 269

Proteolysis targeting chimera (PROTAC) in drug discovery paradigm: Recent progress and future challenges

Zeng, S., Huang, W., Zheng, X., Cheng, L., Zhang, Z., Wang, J., & Shen, Z. (2020) · European Journal of Medicinal Chemistry · doi:10.1016/j.ejmech.2020.112981 · Cited by 264

Cancer‐Targeting Nanoparticles for Combinatorial Nucleic Acid Delivery

Vaughan, H. J., Green, J. J., & Tzeng, S. Y. (2019) · Advanced Materials · doi:10.1002/adma.201901081 · Cited by 259

Genome Editing with mRNA Encoding ZFN, TALEN, and Cas9

Zhang, H., Zhang, Y., & Yin, H. (2019) · Molecular Therapy · doi:10.1016/j.ymthe.2019.01.014 · Cited by 256

Comprehensive review of CRISPR-based gene editing: Mechanisms, challenges, and applications in cancer therapy

Chehelgerdi, M., Chehelgerdi, M., Khorramian-Ghahfarokhi, M., Shafieizadeh, M., Mahmoudi, E., Eskandari, F., Rashidi, M., Arshi, A., & Mokhtari-Farsani, A. (2024) · Molecular Cancer · doi:10.1186/s12943-023-01925-5 · Cited by 251

Chemically and Biologically Engineered Bacteria‐Based Delivery Systems for Emerging Diagnosis and Advanced Therapy

Li, Z., Wang, Y., Liu, J., Rawding, P. A., Bu, J., Hong, S., & Hu, Q. (2021) · Advanced Materials · doi:10.1002/adma.202102580 · Cited by 247

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

Behr, M., Zhou, J., Xu, B., & Zhang, H. (2021) · Acta Pharmaceutica Sinica B · doi:10.1016/j.apsb.2021.05.020 · Cited by 210

Appraisal for the Potential of Viral and Nonviral Vectors in Gene Therapy: A Review

Butt, M. H., Zaman, M., Ahmad, A., Khan, R., Mallhi, T. H., Hasan, M. M., Khan, Y. H., Hafeez, S., Massoud, E. E. S., Rahman, M. H., et al. (2022) · Genes · doi:10.3390/genes13081370 · Cited by 230

Exosome-mediated delivery of CRISPR/Cas9 for targeting of oncogenic Kras G12D in pancreatic cancer

McAndrews, K. M., Xiao, F., Chronopoulos, A., LeBleu, V. S., Kugeratski, F. G., & Kalluri, R. (2021) · Life Science Alliance · doi:10.26508/lsa.202000875 · Cited by 216

Emergence of synthetic mRNA: In vitro synthesis of mRNA and its applications in regenerative medicine

Kwon, H., Kim, M., Seo, Y., Moon, Y. S., Lee, H. J., Lee, K., & Lee, H. (2018) · Biomaterials · doi:10.1016/j.biomaterials.2017.11.034 · Cited by 208

Engineered materials for in vivo delivery of genome-editing machinery

Tong, S., Moyo, B., Lee, C. M., Leong, K. W., & Bao, G. (2019) · Nature Reviews Materials · doi:10.1038/s41578-019-0145-9 · Cited by 208

In vivo somatic cell base editing and prime editing

Newby, G. A., & Liu, D. R. (2021) · Molecular Therapy · doi:10.1016/j.ymthe.2021.09.002 · Cited by 195

Targeting of NLRP3 inflammasome with gene editing for the amelioration of inflammatory diseases

Xu, C., Lu, Z.-D., Luo, Y., Liu, Y., Cao, Z., Shen, S., Li, H., Liu, J., Chen, K., Chen, Z., et al. (2018) · Nature Communications · doi:10.1038/s41467-018-06522-5 · Cited by 199

CAR T cell therapy: A new era for cancer treatment (Review)

Mohanty, R., Chowdhury, C. R., Arega, S., Sen, P., Ganguly, P., & Ganguly, N. (2019) · Oncology Reports · doi:10.3892/or.2019.7335 · Cited by 203

Rational designs of in vivo CRISPR-Cas delivery systems

Xu, C., Chen, G., Luo, Y., Zhang, Y., Zhao, G., Lu, Z.-D., Czarna, A., Gu, Z., & Wang, J. (2019) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2019.11.005 · Cited by 187

Lung SORT LNPs enable precise homology-directed repair mediated CRISPR/Cas genome correction in cystic fibrosis models

Wei, T., Sun, Y., Cheng, Q., Chatterjee, S., Traylor, Z., Johnson, L. T., Coquelin, M. L., Wang, J., Torres, M. J., Lian, X., et al. (2023) · Nature Communications · doi:10.1038/s41467-023-42948-2 · Cited by 207

Smart Biomaterials: Recent Advances and Future Directions

Kowalski, P. S., Bhattacharya, C., Afewerki, S., & Langer, R. (2018) · ACS Biomaterials Science & Engineering · doi:10.1021/acsbiomaterials.8b00889 · Cited by 189

CRISPR–Cas: a tool for cancer research and therapeutics

Yin, H., Xue, W., & Anderson, D. G. (2019) · Nature Reviews Clinical Oncology · doi:10.1038/s41571-019-0166-8 · Cited by 185

Adeno-associated viral vector-mediated immune responses: Understanding barriers to gene delivery

Nidetz, N. F., McGee, M. C., Tse, L. V., Li, C., Cong, L., Li, Y., & Huang, W. (2020) · Pharmacology & Therapeutics · doi:10.1016/j.pharmthera.2019.107453 · Cited by 174

Single siRNA Nanocapsules for Effective siRNA Brain Delivery and Glioblastoma Treatment

Zou, Y., Sun, X., Wang, Y., Yan, C., Liu, Y., Li, J., Zhang, D., Zheng, M., Chung, R. S., & Shi, B. (2020) · Advanced Materials · doi:10.1002/adma.202000416 · Cited by 178

Recent progress in sono-photodynamic cancer therapy: From developed new sensitizers to nanotechnology-based efficacy-enhancing strategies

Zheng, Y., Ye, J., Li, Z., Chen, H., & Gao, Y. (2020) · Acta Pharmaceutica Sinica B · doi:10.1016/j.apsb.2020.12.016 · Cited by 175

Efficient targeted insertion of large DNA fragments without DNA donors

Wang, J., Zhou, H., Wang, G., Zhang, R., Duan, J., Gao, P., Lei, X., Qiu, H.-Y., Zhang, C., Zhang, Y., et al. (2022) · Nature Methods · doi:10.1038/s41592-022-01399-1 · Cited by 178

An overview of nanoparticles in drug delivery: Properties and applications

Hsu, C., Rheima, A. M., Kadhim, M. M., Ahmed, N. N., Mohammed, S. H., Abbas, F. H., Abed, Z. T., Mahdi, Z. M., Abbas, Z. S., Hachim, S. K., et al. (2023) · South African Journal of Chemical Engineering · doi:10.1016/j.sajce.2023.08.009 · Cited by 180

In vivo genome editing in animals using AAV-CRISPR system: applications to translational research of human disease

Lau, C.-H., & Suh, Y. (2017) · F1000Research · doi:10.12688/f1000research.11243.1 · Cited by 161

Engineered CRISPR-Cas systems for the detection and control of antibiotic-resistant infections

Wu, Y., Battalapalli, D., Hakeem, M. J., Selamneni, V., Zhang, P., Draz, M. S., & Ruan, Z. (2021) · Journal of Nanobiotechnology · doi:10.1186/s12951-021-01132-8 · Cited by 166

Nontoxic nanopore electroporation for effective intracellular delivery of biological macromolecules

Cao, Y., Ma, E., Cestellos-Blanco, S., Zhang, B., Qiu, R., Su, Y., Doudna, J. A., & Yang, P. (2019) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1818553116 · Cited by 150

Why and where an HIV cure is needed and how it might be achieved

Ndung’u, T., McCune, J. M., & Deeks, S. G. (2019) · Nature · doi:10.1038/s41586-019-1841-8 · Cited by 153

Highly efficient prime editing by introducing same-sense mutations in pegRNA or stabilizing its structure

Li, X., Zhou, L., Gao, B., Li, G., Wang, X., Wang, Y., Wei, J., Han, W., Wang, Z., Li, J., et al. (2022) · Nature Communications · doi:10.1038/s41467-022-29339-9 · Cited by 142

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

Cell‐Based Delivery Systems: Emerging Carriers for Immunotherapy

Li, Z., Wang, Y., Ding, Y., Repp, L., Kwon, G. S., & Hu, Q. (2021) · Advanced Functional Materials · doi:10.1002/adfm.202100088 · 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

Microfluidic synthesis of lipid-based nanoparticles for drug delivery: recent advances and opportunities

Mehraji, S., & DeVoe, D. L. (2024) · Lab on a Chip · doi:10.1039/d3lc00821e · Cited by 144

Cationic Liposomes as Vectors for Nucleic Acid and Hydrophobic Drug Therapeutics

Ewert, K. K., Scodeller, P., Simón‐Gracia, L., Steffes, V. M., Wonder, E. A., Teesalu, T., & Safinya, C. R. (2021) · Pharmaceutics · doi:10.3390/pharmaceutics13091365 · Cited by 128

Self-assembled peptide–poloxamine nanoparticles enable in vitro and in vivo genome restoration for cystic fibrosis

Guan, S., Munder, A., Hedtfeld, S., Braubach, P., Glage, S., Zhang, L., Lienenklaus, S., Schultze, A., Hasenpusch, G., Garrels, W., et al. (2019) · Nature Nanotechnology · doi:10.1038/s41565-018-0358-x · Cited by 130

Design of Polymeric Gene Carriers for Effective Intracellular Delivery

Lai, W., & Wong, W. (2018) · Trends in biotechnology · doi:10.1016/j.tibtech.2018.02.006 · Cited by 128

Next-Generation Chimeric Antigen Receptor T-Cell Therapy: Going off the Shelf

Ruella, M., & Kenderian, S. S. (2017) · BioDrugs · doi:10.1007/s40259-017-0247-0 · Cited by 129

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

Computational Tools and Resources for CRISPR/Cas Genome Editing

Li, C., Chu, W., Gill, R. A., Sang, S., Shi, Y., Hu, X., Yang, Y., Zaman, Q. U., & Zhang, B. (2022) · Genomics Proteomics & Bioinformatics · doi:10.1016/j.gpb.2022.02.006 · Cited by 134

The roles of polymers in mRNA delivery

Huang, P., Deng, H., Zhou, Y., & Chen, X. S. (2022) · Matter · doi:10.1016/j.matt.2022.03.006 · Cited by 131

Efficient Polymer-Mediated Delivery of Gene-Editing Ribonucleoprotein Payloads through Combinatorial Design, Parallelized Experimentation, and Machine Learning

Kumar, R., Le, N., Tan, Z., Brown, M. E., Jiang, S., & Reineke, T. M. (2020) · ACS Nano · doi:10.1021/acsnano.0c08549 · Cited by 132

Universal intracellular biomolecule delivery with precise dosage control

Cao, Y., Chen, H., Qiu, R., Hanna, M.-E., Ma, E., Hjort, M., Zhang, A., Lewis, R. S., Wu, J. C., & Melosh, N. A. (2018) · Science Advances · doi:10.1126/sciadv.aat8131 · Cited by 128

The NIH Somatic Cell Genome Editing program

Saha, K., Sontheimer, E. J., Brooks, P. J., Dwinell, M. R., Gersbach, C. A., Liu, D. R., Murray, S. A., Tsai, S. Q., Wilson, R. C., Anderson, D. G., et al. (2021) · Nature · doi:10.1038/s41586-021-03191-1 · Cited by 127

CRISPR/Cas9 for Cancer Therapy: Hopes and Challenges

Martínez-Lage, M., Puig-Serra, P., Menéndez, P. R., Torres-Ruiz, R., & Rodríguez, S. (2018) · Biomedicines · doi:10.3390/biomedicines6040105 · Cited by 128

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

Bivalent Ligands for Protein Degradation in Drug Discovery

Scheepstra, M., Hekking, K. F. W., Hijfte, L. V., & Folmer, R. H. A. (2019) · Computational and Structural Biotechnology Journal · doi:10.1016/j.csbj.2019.01.006 · Cited by 125

Genome editing and its applications in genetic improvement in aquaculture

Yang, Z., Yu, Y., Tay, Y. X., & Yue, G. H. (2021) · Reviews in Aquaculture · doi:10.1111/raq.12591 · Cited by 122

Combinatorial design of siloxane-incorporated lipid nanoparticles augments intracellular processing for tissue-specific mRNA therapeutic delivery

Xue, L., Zhao, G., Gong, N., Han, X., Shepherd, S. J., Xiong, X., Xiao, Z., Palanki, R., Xu, J., Swingle, K. L., et al. (2024) · Nature Nanotechnology · doi:10.1038/s41565-024-01747-6 · Cited by 141

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

Engineered amphiphilic peptides enable delivery of proteins and CRISPR-associated nucleases to airway epithelia

Krishnamurthy, S., Wohlford-Lenane, C., KANDIMALLA, S., Sartre, G., Meyerholz, D. K., Théberge, V., Hallée, S., Duperré, A.-M., Del’Guidice, T., Lepetit-Stoffaes, J.-P., et al. (2019) · Nature Communications · doi:10.1038/s41467-019-12922-y · Cited by 116

Efficient gene editing via non-viral delivery of CRISPR–Cas9 system using polymeric and hybrid microcarriers

Timin, A. S., Muslimov, A. R., Lepik, K. V., Epifanovskaya, O. S., Shakirova, A. I., Mock, U., Riecken, K., Okilova, M. V., Sergeev, V. S., Afanasyev, B. V., et al. (2017) · Nanomedicine Nanotechnology Biology and Medicine · doi:10.1016/j.nano.2017.09.001 · Cited by 116

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

All-in-one adeno-associated virus delivery and genome editing by Neisseria meningitidis Cas9 in vivo

Ibraheim, R., Song, C., Mir, A., Amrani, N., Xue, W., & Sontheimer, E. J. (2018) · Genome biology · doi:10.1186/s13059-018-1515-0 · Cited by 111

CRISPR-Cas12a delivery by DNA-mediated bioresponsive editing for cholesterol regulation

Sun, W., Wang, J., Hu, Q., Zhou, X., Khademhosseini, A., & Gu, Z. (2020) · Science Advances · doi:10.1126/sciadv.aba2983 · Cited by 115

Therapeutic genome editing of triple-negative breast tumors using a noncationic and deformable nanolipogel

Guo, P., Yang, J., Huang, J., Auguste, D. T., & Moses, M. A. (2019) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1904697116 · Cited by 112

Quantitating Endosomal Escape of a Library of Polymers for mRNA Delivery

Jiang, Y., Qiao, L., Wang, Y., Xu, E., Ho, A. S. L., Singh, P., Wang, Y., Jiang, Z., Yang, F., Tietjen, G. T., et al. (2020) · Nano Letters · doi:10.1021/acs.nanolett.9b04426 · Cited by 111

Current Challenges in Delivery and Cytosolic Translocation of Therapeutic RNAs

Johannes, L., & Lucchino, M. (2018) · Nucleic Acid Therapeutics · doi:10.1089/nat.2017.0716 · Cited by 110

A glance at genome editing with CRISPR–Cas9 technology

Barman, A., Deb, B., & Chakraborty, S. (2019) · Current Genetics · doi:10.1007/s00294-019-01040-3 · Cited by 107

Ex vivo cell-based CRISPR/Cas9 genome editing for therapeutic applications

Li, Y., Glass, Z., Huang, M., Chen, Z., & Xu, Q. (2020) · Biomaterials · doi:10.1016/j.biomaterials.2019.119711 · Cited by 118

Recent advances in lipid nanoparticles for delivery of nucleic acid, mRNA, and gene editing-based therapeutics

Mukai, H., Ogawa, K., Kato, N., & Kawakami, S. (2022) · Drug Metabolism and Pharmacokinetics · doi:10.1016/j.dmpk.2022.100450 · Cited by 108

Hierarchical Self‐assembly of Discrete Metal–Organic Cages into Supramolecular Nanoparticles for Intracellular Protein Delivery

Liu, J., Luo, T., Xue, Y., Mao, L., Stang, P. J., & Wang, M. (2020) · Angewandte Chemie International Edition · doi:10.1002/anie.202013904 · Cited by 106

Extension of the crRNA enhances Cpf1 gene editing in vitro and in vivo

Park, H. M., Liu, H., Wu, J., Chong, A., Mackley, V., Fellmann, C., Rao, A., Jiang, F., Chu, H., Murthy, N., et al. (2018) · Nature Communications · doi:10.1038/s41467-018-05641-3 · Cited by 102

CRISPR technologies for stem cell engineering and regenerative medicine

Hsu, M., Chang, Y., Truong, V. A., Lai, P., Nguyen, T. K. N., & Hu, Y. (2019) · Biotechnology Advances · doi:10.1016/j.biotechadv.2019.107447 · Cited by 106

Efficient engineering of human and mouse primary cells using peptide-assisted genome editing

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Carrier strategies boost the application of CRISPR/Cas system in gene therapy

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Oral delivery of IL-22 mRNA-loaded lipid nanoparticles targeting the injured intestinal mucosa: A novel therapeutic solution to treat ulcerative colitis

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Safety Landscape of Therapeutic Nanozymes and Future Research Directions

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Endoglin and alk1 as therapeutic targets for hereditary hemorrhagic telangiectasia

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Overcoming the delivery problem for therapeutic genome editing: Current status and perspective of non-viral methods

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Delivery technologies for T cell gene editing: Applications in cancer immunotherapy

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Ready for Repair? Gene Editing Enters the Clinic for the Treatment of Human Disease

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Challenges in delivery systems for CRISPR-based genome editing and opportunities of nanomedicine

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Optimizing 5’UTRs for mRNA-delivered gene editing using deep learning

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An engineered hypercompact CRISPR-Cas12f system with boosted gene-editing activity

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A non-cationic nucleic acid nanogel for the delivery of the CRISPR/Cas9 gene editing tool

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An overview of treatment strategies for Hutchinson-Gilford Progeria syndrome

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Messenger RNA-Based Vaccines Against Infectious Diseases

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Non-Viral Delivery To Enable Genome Editing

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Delivery Systems for Therapeutic Genome Editing: Challenges, Innovations, and Future Perspectives

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Balancing efficiency, safety, and scalability in therapeutic genome editing delivery

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Nonviral Nanoparticles for CRISPR-Based Genome Editing: Is It Just a Simple Adaption of What Have Been Developed for Nucleic Acid Delivery?

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Exploring Advanced CRISPR Delivery Technologies for Therapeutic Genome Editing

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