Cited by 366CRISPR and Genetic EngineeringRNA Interference and Gene DeliveryVirus-based gene therapy research

The DOI matches its Crossref record; 54 of the 54 references it cites were found in a registry or an index.

Nanoparticle delivery of CRISPR into the brain rescues a mouse model of fragile X syndrome from exaggerated repetitive behaviours

Open access

Lee, Bumwhee, Lee, Kunwoo, Panda, Shree, Gonzales-Rojas, Rodrigo, Chong, Anthony, Bugay, Vladislav, Park, Hyo Min, Brenner, Robert, Murthy, Niren, Lee, Hye Young·2018·Nature Biomedical Engineering

DOI 10.1038/s41551-018-0252-8PMC PMC6544395

Open this paper in CoChat — free account

Abstract

Technologies that can safely edit genes in the brains of adult animals may revolutionize the treatment of neurological diseases and the understanding of brain function. Here, we demonstrate that intracranial injection of CRISPR-Gold, a nonviral delivery vehicle for the CRISPR-Cas9 ribonucleoprotein, can edit genes in the brains of adult mice in multiple mouse models. CRISPR-Gold can deliver both Cas9 and Cpf1 ribonucleoproteins, and can edit all of the major cell types in the brain, including neurons, astrocytes and microglia, with undetectable levels of toxicity at the doses used. We also show that CRISPR-Gold designed to target the metabotropic glutamate receptor 5 (mGluR5) gene can efficiently reduce local mGluR5 levels in the striatum after an intracranial injection. The effect can also rescue mice from the exaggerated repetitive behaviours caused by fragile X syndrome, a common single-gene form of autism spectrum disorders. CRISPR-Gold may significantly accelerate the development of brain-targeted therapeutics and enable the rapid development of focal brain-knockout animal models.

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 · 10.1038/s41551-018-0252-8

Cites

54 of 54 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
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
A robust and high-throughput Cre reporting and characterization system for the whole mouse brain
Madisen, L., Zwingman, T. A., Sunkin, S. M., Oh, S. W., Zariwala, H. A., Gu, H., Ng, L. L., Palmiter, R. D., Hawrylycz, M. J., Jones, A. R., et al. (2010) · Nature Neuroscience · doi:10.1038/nn.2467 · Cited by 6,907
Improved vectors and genome-wide libraries for CRISPR screening
Sanjana, N. E., Shalem, O., & Zhang, F. (2014) · Nature Methods · doi:10.1038/nmeth.3047 · Cited by 5,239
Cpf1 Is a Single RNA-Guided Endonuclease of a Class 2 CRISPR-Cas System
Zetsche, B., Gootenberg, J. S., Abudayyeh, O. O., Slaymaker, I. M., Makarova, K. S., Essletzbichler, P., Volz, S. E., Joung, J., van der Oost, J., Regev, A., et al. (2015) · Cell · doi:10.1016/j.cell.2015.09.038 · Cited by 4,761
Imaging Neuronal Subsets in Transgenic Mice Expressing Multiple Spectral Variants of GFP
Feng, G., Mellor, R. H., Bernstein, M., Keller-Peck, C., Nguyen, Q. T., Wallace, M., Nerbonne, J. M., Lichtman, J. W., & Sanes, J. R. (2000) · Neuron · doi:10.1016/s0896-6273(00)00084-2 · Cited by 3,042
Easy quantitative assessment of genome editing by sequence trace decomposition
Brinkman, E. K., Chen, T., Amendola, M., & van Steensel, B. (2014) · Nucleic Acids Research · doi:10.1093/nar/gku936 · Cited by 2,390
Targeted genome engineering in human cells with the Cas9 RNA-guided endonuclease
Cho, S. W., Kim, S., Kim, J. M., & Kim, J.-S. (2013) · Nature Biotechnology · doi:10.1038/nbt.2507 · Cited by 1,863
The mGluR theory of fragile X mental retardation
Bear, M. F., Huber, K. M., & Warren, S. T. (2004) · Trends in Neurosciences · doi:10.1016/j.tins.2004.04.009 · Cited by 1,580

Show all 54 references

Sorted by citations

Items 101–200 of 366

Suppressing the NHEJ pathway by DNA-PKcs inhibitor NU7026 prevents degradation of HBV cccDNA cleaved by CRISPR/Cas9
Kostyushev, D. S., Kostyusheva, A., Brezgin, S., Zarifyan, D., Utkina, A., Goptar, I. A., & Chulanov, V. (2019) · Scientific Reports · doi:10.1038/s41598-019-38526-6 · Cited by 62
Dual Supramolecular Nanoparticle Vectors Enable CRISPR/Cas9‐Mediated Knockin of Retinoschisin 1 Gene—A Potential Nonviral Therapeutic Solution for X‐Linked Juvenile Retinoschisis
Chou, S., Yang, P., Ban, Q., Yang, Y. P., Wang, M., Chien, C., Chen, S. J., Sun, N., Zhu, Y., Liu, H., et al. (2020) · Advanced Science · doi:10.1002/advs.201903432 · Cited by 62
Nanobiomaterial vectors for improving gene editing and gene therapy
Hu, S. W., Ding, T., Tang, H., Guo, H., Cui, W., & Shu, Y. (2023) · Materials Today · doi:10.1016/j.mattod.2023.04.011 · Cited by 58
Progress and challenges towards CRISPR/Cas clinical translation
Rosenblum, D., Gutkin, A., Dammes, N., & Peer, D. (2020) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2020.07.004 · Cited by 61
Gold Nanoparticle-Mediated Gene Therapy
Kanu, G. A., Parambath, J. B. M., Odeh, R. O. A., & Mohamed, A. A. M. (2022) · Cancers · doi:10.3390/cancers14215366 · Cited by 57
Engineering self-deliverable ribonucleoproteins for genome editing in the brain
Chen, K., Stahl, E. C., Kang, M. H., Xu, B., Allen, R., Trinidad, M. I., & Doudna, J. A. (2024) · Nature Communications · doi:10.1038/s41467-024-45998-2 · Cited by 61
Delivery of nucleic acid based genome editing platforms via lipid nanoparticles: Clinical applications
Masarwy, R., Stotsky‐Oterin, L., Elisha, A., Hazan‐Halevy, I., & Peer, D. (2024) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2024.115359 · Cited by 59
In vivo delivery of CRISPR-Cas9 genome editing components for therapeutic applications
Huang, K. L., Zapata, D. A., Tang, Y., Teng, Y., & Li, Y. (2022) · Biomaterials · doi:10.1016/j.biomaterials.2022.121876 · Cited by 61
Intelligent nanotherapeutic strategies for the delivery of CRISPR system
Chen, C., Zhong, W., Du, S., Li, Y., Zeng, Y., Liu, K., Yang, J., Guan, X., & Han, X. (2022) · Acta Pharmaceutica Sinica B · doi:10.1016/j.apsb.2022.12.013 · Cited by 56
A potential paradigm in CRISPR/Cas systems delivery: at the crossroad of microalgal gene editing and algal-mediated nanoparticles
Feng, S., Xie, X., Liu, J., Li, A., Wang, Q., Guo, D., Li, S., Li, Y., Wang, Z., Guo, T., et al. (2023) · Journal of Nanobiotechnology · doi:10.1186/s12951-023-02139-z · Cited by 59
Approach for in vivo delivery of CRISPR/Cas system: a recent update and future prospect
Chuang, Y.-F., Phipps, A. J., Lin, F., Hecht, V. F. G., Hewitt, A. W., Wang, P., & Liu, G. (2021) · Cellular and Molecular Life Sciences · doi:10.1007/s00018-020-03725-2 · Cited by 53
Precision epigenetic editing: Technological advances, enduring challenges, and therapeutic applications
Roth, G. V., Gengaro, I. R., & Qi, L. S. (2024) · Cell chemical biology · doi:10.1016/j.chembiol.2024.07.007 · Cited by 59
Recent Advances in CRISPR/Cas9 Delivery Approaches for Therapeutic Gene Editing of Stem Cells
Lotfi, M., Rad, D. M., Mashhadi, S., Ashouri, A., Mojarrad, M., Mozaffari‐Jovin, S., Farrokhi, S., Hashemi, M., Lotfi, M., Warkiani, M. E., et al. (2023) · Stem Cell Reviews and Reports · doi:10.1007/s12015-023-10585-3 · Cited by 54
Regulation of Proteins to the Cytosol Using Delivery Systems with Engineered Polymer Architecture
Kretzmann, J. A., Luther, D. C., Evans, C. W., Jeon, T., Jerome, W. P., Gopalakrishnan, S., Lee, Y.-W., Nörret, M., Iyer, K. S., & Rotello, V. M. (2021) · Journal of the American Chemical Society · doi:10.1021/jacs.1c00258 · Cited by 50
Prevention of acquired sensorineural hearing loss in mice by in vivo Htra2 gene editing
Gu, X. G., Wang, D., Xu, Z., Wang, J., Guo, L., Chai, R., Li, G., Shu, Y., & Li, H. (2021) · Genome biology · doi:10.1186/s13059-021-02311-4 · Cited by 51
Clinical applications of CRISPR‐based genome editing and diagnostics
Foss, D. V., Hochstrasser, M. L., & Wilson, R. C. (2019) · Transfusion · doi:10.1111/trf.15126 · Cited by 51
Strategies for the CRISPR-Based Therapeutics
Li, B., Niu, Y., Ji, W., & Dong, Y. (2019) · Trends in Pharmacological Sciences · doi:10.1016/j.tips.2019.11.006 · Cited by 52
Enhancing CRISPR/Cas systems with nanotechnology
Chowdhry, R., Lu, S. Z., Lee, S., Godhulayyagari, S., Ebrahimi, S. B., & Samanta, D. (2023) · Trends in biotechnology · doi:10.1016/j.tibtech.2023.06.005 · Cited by 50
Nanoplatform‐Based In Vivo Gene Delivery Systems for Cancer Therapy
Luo, R., Le, H., Wu, Q., & Gong, C. (2024) · Small · doi:10.1002/smll.202312153 · Cited by 50
Therapy Approaches for Stargardt Disease
Piotter, E., McClements, M. E., & MacLaren, R. E. (2021) · Biomolecules · doi:10.3390/biom11081179 · Cited by 49
Genome editing in the mouse brain with minimally immunogenic Cas9 RNPs
Stahl, E. C., Sabo, J. K., Kang, M. H., Allen, R., Applegate, E., Kim, S., Kwon, Y., Seth, A., Lemus, N., Salinas-Rios, V., et al. (2023) · Molecular Therapy · doi:10.1016/j.ymthe.2023.06.019 · Cited by 51
CRISPR-Cas9 Gene Therapy: Non-Viral Delivery and Stimuli-Responsive Nanoformulations
Lee, H., Rho, W., Kim, Y.-H., Chang, H., & Jun, B. (2025) · Molecules · doi:10.3390/molecules30030542 · Cited by 59
CRISPR/Cas9 System: A Bacterial Tailor for Genomic Engineering
Lone, B. A., Karna, S. K. L., Ahmad, F., Shahi, N., & Pokharel, Y. R. (2018) · Genetics Research International · doi:10.1155/2018/3797214 · Cited by 48
Emerging technologies to study glial cells
Hirbec, H., Déglon, N., Foo, L. C., Goshen, I., Grutzendler, J., Hangen, E., Kreisel, T., Linck, N., Muffat, J., Regio, S., et al. (2020) · Glia · doi:10.1002/glia.23780 · Cited by 51
Binding‐Mediated Formation of Ribonucleoprotein Corona for Efficient Delivery and Control of CRISPR/Cas9
Wu, J., Peng, H., Lu, X., Lai, M., Zhang, H., & Le, X. C. (2020) · Angewandte Chemie International Edition · doi:10.1002/anie.202014162 · Cited by 45
Development of delivery strategies for CRISPR‐Cas9 genome editing
Liu, Q., Yang, J., Xing, Y., Zhao, Y., & Liu, Y. (2023) · BMEMat · doi:10.1002/bmm2.12025 · Cited by 47
CRISPR/Cas System for Genome Editing: Progress and Prospects as a Therapeutic Tool
Sahel, D. K., Mittal, A., & Chitkara, D. (2019) · Journal of Pharmacology and Experimental Therapeutics · doi:10.1124/jpet.119.257287 · Cited by 47
X‐ray‐Guided In Situ Genetic Engineering of Macrophages for Sustained Cancer Immunotherapy
Zhao, C., Cheng, Y., Huang, P., Wang, C., Wang, W., Wang, M., Shan, W., & Deng, H. (2022) · Advanced Materials · doi:10.1002/adma.202208059 · Cited by 45
Advanced theragnostics for the central nervous system (CNS) and neurological disorders using functional inorganic nanomaterials
Conklin, B., Conley, B. M., Hou, Y., Chen, M., & Lee, K.-B. (2023) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2022.114636 · Cited by 45
CRISPR/Cas9 application in cancer therapy: a pioneering genome editing tool
Baghini, S. S., Gardanova, Z. R., Abadi, S. A. H., Zaman, B. A., İlhan, A., Shomali, N., Adili, A., Moghaddar, R., & Yaseri, A. F. (2022) · Cellular & Molecular Biology Letters · doi:10.1186/s11658-022-00336-6 · Cited by 46
Translating CRISPR-Cas Therapeutics: Approaches and Challenges
Tay, L. S., Palmer, N. D., Panwala, R., Chew, W. L., & Mali, P. (2020) · The CRISPR Journal · doi:10.1089/crispr.2020.0025 · Cited by 43
Supramolecular nanosubstrate–mediated delivery system enables CRISPR-Cas9 knockin of hemoglobin beta gene for hemoglobinopathies
Yang, P., Chou, S., Li, J., Hui, W., Liu, W., Sun, N., Zhang, R. Y., Zhu, Y., Tsai, M., Lai, H. I. A. M., et al. (2020) · Science Advances · doi:10.1126/sciadv.abb7107 · Cited by 42
Nanoparticle-Based Immunoengineered Approaches for Combating HIV
Bowen, A., Sweeney, E. E., & Fernandes, R. (2020) · Frontiers in Immunology · doi:10.3389/fimmu.2020.00789 · Cited by 42
A temporally resolved DNA framework state machine in living cells
Zhao, Y., Cao, S., Wang, Y., Li, F., Lin, L., Guo, L., Wang, F., Chao, J., Zuo, X., Zhu, Y. J., et al. (2023) · Nature Machine Intelligence · doi:10.1038/s42256-023-00707-4 · Cited by 44
FMRP(1–297)-tat restores ion channel and synaptic function in a model of Fragile X syndrome
Zhan, X., Asmara, H., Cheng, N., Sahu, G., Sanchez, E., Zhang, F., Zamponi, G. W., Rho, J. M., & Turner, R. W. (2020) · Nature Communications · doi:10.1038/s41467-020-16250-4 · Cited by 39
Hematopoietic Stem Cell-Targeted Gene-Addition and Gene-Editing Strategies for β-hemoglobinopathies
Drysdale, C. M., Nassehi, T., Gamer, J., Yapundich, M., Tisdale, J. F., & Uchida, N. (2021) · Cell stem cell · doi:10.1016/j.stem.2021.01.001 · Cited by 39
Nano-vectors for CRISPR/Cas9-mediated genome editing
Yang, P., Lee, A. Y.-T., Xue, J., Chou, S., Lee, C., Tseng, P., Zhang, T. X., Zhu, Y., Lee, J., Chiou, S., et al. (2022) · Nano Today · doi:10.1016/j.nantod.2022.101482 · Cited by 41
Non-viral delivery of CRISPR/Cas cargo to the retina using nanoparticles: current possibilities, challenges, and limitations
Salman, A., Kantor, A., McClements, M. E., Marfany, G., Trigueros, S., & MacLaren, R. E. (2022) · Pharmaceutics · doi:10.3390/pharmaceutics14091842 · Cited by 37
Advances in CRISPR Delivery Methods: Perspectives and Challenges
Demirci, S., Essawi, K., Germino-Watnick, P., Liu, X., Hakami, W., & Tisdale, J. F. (2022) · The CRISPR Journal · doi:10.1089/crispr.2022.0051 · Cited by 37
Delivery Platforms for CRISPR/Cas9 Genome Editing of Glial Cells in the Central Nervous System
Meneghini, V., Peviani, M., Luciani, M., Zambonini, G., & Gritti, A. (2021) · Frontiers in Genome Editing · doi:10.3389/fgeed.2021.644319 · Cited by 36
Nanoparticle delivery systems for substance use disorder
Kasina, V., Mownn, R. J., Bahal, R., & Sartor, G. C. (2022) · Neuropsychopharmacology · doi:10.1038/s41386-022-01311-7 · Cited by 39
A Genome-Editing Nanomachine Constructed with a Clustered Regularly Interspaced Short Palindromic Repeats System and Activated by Near-Infrared Illumination
Peng, H., Le, C., Wu, J., Li, X., Zhang, H., & Le, X. C. (2020) · ACS Nano · doi:10.1021/acsnano.9b05276 · Cited by 34
Genome Editing for CNS Disorders
Duarte, F. T., & Déglon, N. (2020) · Frontiers in Neuroscience · doi:10.3389/fnins.2020.579062 · Cited by 36
High-Throughput Automation of Endosomolytic Polymers for mRNA Delivery
Ulkoski, D., Munson, M. J., Jacobson, M. E., Palmer, C. R., Carson, C. S., Sabirsh, A., Wilson, J. T., & Krishnamurthy, V. R. (2021) · ACS Applied Bio Materials · doi:10.1021/acsabm.0c01463 · Cited by 34
Efficient spatially targeted gene editing using a near-infrared activatable protein-conjugated nanoparticle for brain applications
Rebelo, C., Reis, T., Guedes, J. R., Saraiva, C., Rodrigues, A. F., Simões, S. M. N., Bernardino, L. I., Peça, J., Pinho, S. L. C., & Ferreira, L. (2022) · Nature Communications · doi:10.1038/s41467-022-31791-6 · Cited by 33
Efficient in vivo neuronal genome editing in the mouse brain using nanocapsules containing CRISPR-Cas9 ribonucleoproteins
Metzger, J. M., Wang, Y., Neuman, S. S., Snow, K. J., Murray, S. A., Lutz, C. M., Bondarenko, V., Felton, J., Gimse, K., Xie, R., et al. (2022) · Biomaterials · doi:10.1016/j.biomaterials.2022.121959 · Cited by 38
Lipid Nanoparticles for In Vivo Lung Delivery of CRISPR-Cas9 Ribonucleoproteins Allow Gene Editing of Clinical Targets
Haley, R. M., Padilla, M. S., El-Mayta, R. D., Joseph, R. A., Weber, J. A., Figueroa‐Espada, C. G., Mukalel, A. J., Ricciardi, A. S., Palanki, R., Geisler, H. C., et al. (2025) · ACS Nano · doi:10.1021/acsnano.4c16617 · Cited by 38
Engineering CRISPR mouse models of cancer
Weber, J., & Rad, R. (2019) · Current Opinion in Genetics & Development · doi:10.1016/j.gde.2019.04.001 · Cited by 33
A traceless linker for aliphatic amines that rapidly and quantitatively fragments after reduction
He, M., Li, J., Han, H., Borges, C. A., Neiman, G., Røise, J. J., Hadaczek, P., Mendonsa, R., Holm, V. R., Wilson, R. C., et al. (2020) · Chemical Science · doi:10.1039/d0sc00929f · Cited by 33
Dosage-sensitive genes in autism spectrum disorders: From neurobiology to therapy
Javed, S., Selliah, T., Lee, Y., & Huang, W. (2020) · Neuroscience & Biobehavioral Reviews · doi:10.1016/j.neubiorev.2020.08.009 · Cited by 32
Design of efficacious somatic cell genome editing strategies for recessive and polygenic diseases
Carlson-Stevermer, J., Das, A., Abdeen, A. A., Fiflis, D., Grindel, B. I., Saxena, S., Akcan, T., Alam, T., Kletzien, H., Kohlenberg, L., et al. (2020) · Nature Communications · doi:10.1038/s41467-020-20065-8 · Cited by 32
Genome and base editing for genetic hearing loss
Niggemann, P., György, B., & Chen, Z. (2020) · Hearing Research · doi:10.1016/j.heares.2020.107958 · Cited by 32
Pre-clinical non-viral vectors exploited for in vivo CRISPR/Cas9 gene editing: an overview
Rouatbi, N., McGlynn, T., & Al‐Jamal, K. T. (2022) · Biomaterials Science · doi:10.1039/d1bm01452h · Cited by 33
Techniques for investigating lncRNA transcript functions in neurodevelopment
Srinivas, T., Soares, E. S., & Guil, S. (2023) · Molecular Psychiatry · doi:10.1038/s41380-023-02377-5 · Cited by 37
Non-viral nanocarriers for CRISPR-Cas9 gene editing system delivery
Tang, X., Wang, Z., Zhang, Y., Mu, W., & Han, X. (2022) · Chemical Engineering Journal · doi:10.1016/j.cej.2022.135116 · Cited by 30
Nonviral Nanoparticles for CRISPR-Based Genome Editing: Is It Just a Simple Adaption of What Have Been Developed for Nucleic Acid Delivery?
Qiu, M., Glass, Z., & Xu, Q. (2019) · Biomacromolecules · doi:10.1021/acs.biomac.9b00783 · Cited by 30
Nucleic acid therapies for CNS diseases: Pathophysiology, targets, barriers, and delivery strategies
Padmakumar, S., D’souza, A. A., Parayath, N. N., Bleier, B. S., & Amiji, M. M. (2022) · Journal of Controlled Release · doi:10.1016/j.jconrel.2022.10.018 · Cited by 30
The potential of gene editing for Huntington’s disease
Duan, W., Urani, E., & Mattson, M. P. (2023) · Trends in Neurosciences · doi:10.1016/j.tins.2023.02.005 · Cited by 31
Widespread Gene Editing in the Brain via In Utero Delivery of mRNA Using Acid-Degradable Lipid Nanoparticles
Gao, K., Han, H., Cranick, M. G., Zhao, S., Xu, S., Yin, B., Song, H., Hu, Y., Clarke, M. T., Wang, D., et al. (2024) · ACS Nano · doi:10.1021/acsnano.4c05169 · Cited by 33
Advance genome editing technologies in the treatment of human diseases: CRISPR therapy (Review)
Alagöz, M., & Kherad, N. (2020) · International Journal of Molecular Medicine · doi:10.3892/ijmm.2020.4609 · Cited by 32
Protocol for assessment of the efficiency of CRISPR/Cas RNP delivery to different types of target cells
Tyumentseva, M. A., Tyumentsev, A. I., & Акимкин, В. Г. (2021) · PLoS ONE · doi:10.1371/journal.pone.0259812 · Cited by 30
Nanoparticle technologies: Recent state of the art and emerging opportunities
Parupudi, A., Mulagapati, S. H. R., & Subramony, J. A. (2021) · Elsevier eBooks · doi:10.1016/b978-0-12-820757-4.00009-0 · Cited by 34
Synergy of nanocarriers with CRISPR-Cas9 in an emerging technology platform for biomedical appliances: Current insights and perspectives
Bhattacharjee, R., Jana, A., Nandi, A., Sinha, A., Bhattacharjee, A., Mitra, S., Kar, S., Dey, A., Singh, S. K., Varma, R. S., et al. (2022) · Materials & Design · doi:10.1016/j.matdes.2022.111415 · Cited by 29
Clinical progress in genome-editing technology and in vivo delivery techniques
Khirallah, J., Eimbinder, M., Li, Y., & Xu, Q. (2023) · Trends in Genetics · doi:10.1016/j.tig.2022.12.001 · Cited by 31
Mouse models of fragile X-related disorders
Willemsen, R., & Kooy, R. F. (2023) · Disease Models & Mechanisms · doi:10.1242/dmm.049485 · Cited by 30
Genome Editing in a Wide Area of the Brain Using Dendrimer-Based Ternary Polyplexes of Cas9 Ribonucleoprotein
Taharabaru, T., Yokoyama, R., Higashi, T., Mohammed, A. F. A., Inoue, M., Maeda, Y. K., Niidome, T., Onodera, R., & Motoyama, K. (2020) · ACS Applied Materials & Interfaces · doi:10.1021/acsami.9b21667 · Cited by 27
Primary Ciliary Deficits in the Dentate Gyrus of Fragile X Syndrome
Lee, B., Panda, S., & Lee, H. Y. (2020) · Stem Cell Reports · doi:10.1016/j.stemcr.2020.07.001 · Cited by 27
Key considerations in designing CRISPR/Cas9-carrying nanoparticles for therapeutic genome editing
Xu, Y., Liu, R., & Dai, Z. (2020) · Nanoscale · doi:10.1039/d0nr05452f · Cited by 27
CRISPR-Cas technology a new era in genomic engineering
Parsaeimehr, A., Ebirim, R. I., & Ozbay, G. (2022) · Biotechnology Reports · doi:10.1016/j.btre.2022.e00731 · Cited by 27
Rationally designed nanoparticle delivery of Cas9 ribonucleoprotein for effective gene editing
Chae, S.-Y., Jeong, E., Kang, S., Yim, Y., Kim, J., & Min, D. (2022) · Journal of Controlled Release · doi:10.1016/j.jconrel.2022.02.035 · Cited by 27
Overview of genetic models of autism spectrum disorders
Patel, J., Lukkes, J. L., & Shekhar, A. (2018) · Progress in brain research · doi:10.1016/bs.pbr.2018.10.002 · Cited by 29
Live-Animal Epigenome Editing: Convergence of Novel Techniques
Gomez, J. A., Beitnere, U., & Segal, D. J. (2019) · Trends in Genetics · doi:10.1016/j.tig.2019.04.007 · Cited by 24
MicroRNAs in β-thalassemia
Wang, F., Ling, L., & Yu, D. (2021) · The American Journal of the Medical Sciences · doi:10.1016/j.amjms.2021.02.011 · Cited by 27
Early and Late Corrections in Mouse Models of Autism Spectrum Disorder
Chung, C., Shin, W., & Kim, E. (2021) · Biological Psychiatry · doi:10.1016/j.biopsych.2021.07.021 · Cited by 23
Exploring Advanced CRISPR Delivery Technologies for Therapeutic Genome Editing
Rostami, N., Gomari, M. M., Choupani, E., Abkhiz, S., Fadaie, M., Eslami, S. S., Mahmoudi, Z., Zhang, Y., Puri, M., Monfared, F. N., et al. (2024) · Small Science · doi:10.1002/smsc.202400192 · Cited by 27
Lipid nanoparticle-mediated intracameral mRNA delivery facilitates gene expression and editing in the anterior chamber of the eye
Vasudevan, A., Jozić, A., Curtis, A. G., Bodi, E., Ryals, R. C., & Sahay, G. (2025) · Journal of Controlled Release · doi:10.1016/j.jconrel.2025.01.068 · Cited by 28
Prime Editing: A Revolutionary Technology for Precise Treatment of Genetic Disorders
Li, M., Lin, Y., Cheng, Q., & Wei, T. (2025) · Cell Proliferation · doi:10.1111/cpr.13808 · Cited by 28
CRISPR/Cas systems combined with DNA nanostructures for biomedical applications
Sun, S., Yang, H., Wu, Z., Zhang, S., Xu, J., & Shi, P. (2024) · Chemical Communications · doi:10.1039/d4cc00290c · Cited by 23
Therapeutic application of the CRISPR system: current issues and new prospects
Lee, M., & Kim, S. (2019) · Human Genetics · doi:10.1007/s00439-019-02028-2 · Cited by 23
Gene Therapy: The Next-Generation Therapeutics and Their Delivery Approaches for Neurological Disorders
Paul, A., Collins, M. G., & Lee, H. Y. (2022) · Frontiers in Genome Editing · doi:10.3389/fgeed.2022.899209 · Cited by 24
Revisiting gene delivery to the brain: silencing and editing
Conniot, J., Talebian, S., Simões, S. M. N., Ferreira, L., & Conde, J. (2020) · Biomaterials Science · doi:10.1039/d0bm01278e · Cited by 25
Spatiotemporal Delivery of CRISPR/Cas9 Genome Editing Machinery Using Stimuli‐Responsive Vehicles
Cai, W., Luo, T., Mao, L., & Wang, M. (2020) · Angewandte Chemie · doi:10.1002/ange.202005644 · Cited by 25
Targeted core-shell nanoparticles for precise CTCF gene insert in treatment of metastatic breast cancer
Duan, J., Bao, C., Xie, Y., Guo, H., Liu, Y., Li, J., Liu, R., Li, P., Bai, J., Yan, Y., et al. (2021) · Bioactive Materials · doi:10.1016/j.bioactmat.2021.10.007 · Cited by 21
Nanoparticles targeting hematopoietic stem and progenitor cells: Multimodal carriers for the treatment of hematological diseases
Cruz, L. J., Rezaei, S., Grosveld, F. G., Philipsen, S., & Eich, C. (2022) · Frontiers in Genome Editing · doi:10.3389/fgeed.2022.1030285 · Cited by 23
Reactivation of FMR1 gene expression is a promising strategy for fragile X syndrome therapy
Shitik, E. M., Velmiskina, A. A., Dolskiy, A. A., & Yudkin, D. V. (2020) · Gene Therapy · doi:10.1038/s41434-020-0141-0 · Cited by 24
Non-Invasive Delivery of CRISPR/Cas9 Ribonucleoproteins (Cas9 RNPs) into Cells via Nanoparticles for Membrane Transport
Tashima, T. (2025) · Pharmaceutics · doi:10.3390/pharmaceutics17020201 · Cited by 22
Targeting the Inside of Cells with Biologicals: Chemicals as a Delivery Strategy
Marschall, A. L. J. (2021) · BioDrugs · doi:10.1007/s40259-021-00500-y · Cited by 22
MOF effectively deliver CRISPR and enhance gene-editing efficiency via MOF’s hydrolytic activity of phosphate ester bonds
Wang, Y., Liu, Z., Zhang, H., Liu, J., Dai, H., Ji, T., Liu, F., Cao, P., Zou, J., Wang, S., et al. (2022) · Chemical Engineering Journal · doi:10.1016/j.cej.2022.134992 · Cited by 21
Cyclodextrin-based tailored polyrotaxanes for highly efficient delivery of the genome-editing molecule
Taharabaru, T., Kihara, T., Obata, A., Onodera, R., Wen, Y., Li, J., Motoyama, K., & Higashi, T. (2023) · Carbohydrate Polymers · doi:10.1016/j.carbpol.2023.121443 · Cited by 22
Systems of Delivery of CRISPR/Cas9 Ribonucleoprotein Complexes for Genome Editing
Амирханов, Р. Н., & Stepanov, G. A. (2019) · Russian Journal of Bioorganic Chemistry · doi:10.1134/s1068162019060025 · Cited by 21
Versatile modification of the CRISPR/Cas9 ribonucleoprotein system to facilitate in vivo application
Sun, B., Chen, H., & Gao, X. (2021) · Journal of Controlled Release · doi:10.1016/j.jconrel.2021.08.007 · Cited by 20
CRISPR–Cas technologies in neurodegenerative disorders: mechanistic insights, therapeutic potential, and translational challenges
Yashooa, R. K., Nabi, A. Q., Smail, S. W., Azeez, S. S., Nooh, W. A., Mustafa, S. A., Al-Farha, A. A.-B., Capitanio, N., & Shekha, M. S. (2026) · Frontiers in Neurology · doi:10.3389/fneur.2025.1737468 · Cited by 21
Making gene editing a therapeutic reality
Conboy, I. M., Murthy, N., Etienne, J., & Robinson, Z. R. (2018) · F1000Research · doi:10.12688/f1000research.16106.1 · Cited by 19
Nanoscale metal–organic frameworks for the intracellular delivery of CRISPR/Cas9 genome editing machinery
Zheng, Q., Li, W., Mao, L., & Wang, M. (2021) · Biomaterials Science · doi:10.1039/d1bm00790d · Cited by 19
Understanding the Potential of Genome Editing in Parkinson’s Disease
Arango, D., Bittar, A., Esmeral, N. P., Ocasión, C., Muñoz-Camargo, C., Cruz, J. C., Reyes, L. H., & Bloch, N. I. (2021) · International Journal of Molecular Sciences · doi:10.3390/ijms22179241 · Cited by 24
CRISPR Systems Suitable for Single AAV Vector Delivery
Stevanovic, M., Piotter, E., McClements, M. E., & MacLaren, R. E. (2021) · Current Gene Therapy · doi:10.2174/1566523221666211006120355 · Cited by 19
Genome editing in cancer: Challenges and potential opportunities
Breier, D., & Peer, D. (2022) · Bioactive Materials · doi:10.1016/j.bioactmat.2022.08.013 · Cited by 21
Gold Nanoparticle-Incorporated Chitosan Nanogels as a Theranostic Nanoplatform for CT Imaging and Tumour Chemotherapy
Liu, Z., Zhou, D., Yan, X., Xiao, L., Wang, P., Wei, J., & Liao, L. (2022) · International Journal of Nanomedicine · doi:10.2147/ijn.s375999 · Cited by 19
Control of Selective mRNA Translation in Neuronal Subcellular Compartments in Health and Disease
Cagnetta, R., Flanagan, J. G., & Sonenberg, N. (2023) · Journal of Neuroscience · doi:10.1523/jneurosci.2240-22.2023 · Cited by 22
Self-delivering, chemically modified CRISPR RNAs for AAV co-delivery and genome editing in vivo
Zhang, H., Kelly, K. L., Lee, J. L. C., Echeverria, D., Cooper, D., Panwala, R., Amrani, N., Chen, Z., Gaston, N., Wagh, A. A., et al. (2023) · Nucleic Acids Research · doi:10.1093/nar/gkad1125 · Cited by 19

Authors: Lee, B., Lee, K., Panda, S., Gonzales-Rojas, R., Chong, A., Bugay, V., Park, H. M., Brenner, R., Murthy, N., & Lee, H. Y.
Affiliations: The University of Texas at San Antonio Health Science Center (US)
University of California, Berkeley (US)
Corresponding author: Murthy, Niren
Lee, Hye Young
ORCID: Lee, Kunwoo
Chong, Anthony
Brenner, Robert
Murthy, Niren
Lee, Hye Young
Venue: Nature Biomedical Engineering
Publisher: Springer Science and Business Media LLC
Published: 2018-06-25
Volume: 2
Issue: 7
Pages: 497-507
ISSN: 2157-846X (ISSN-L)
DOI: 10.1038/s41551-018-0252-8
PMC: PMC6544395
OpenAlex: W2810249184
Cited by: 361
References: 54
Citations by year: 2026: 22 · 2025: 28 · 2024: 36 · 2023: 42 · 2022: 58 · 2021: 56 · 2020: 66 · 2019: 38 · 2018: 15
FWCI: 16.00
Type: journal article
Language: en
Open access: green
www.ncbi.nlm.nih.gov
Licence: www.springernature.com/gp/researchers/text-and-data-mining (tdm)
www.springernature.com/gp/researchers/text-and-data-mining (vor)
Locations: Nature Biomedical Engineering (publishedVersion)
PubMed (publishedVersion)
PubMed Central (acceptedVersion)
eScholarship (California Digital Library) (submittedVersion)
Funders: National Institute of Allergy and Infectious Diseases (R01AI117064)
National Institute of Biomedical Imaging and Bioengineering (R01EB023776, R01EB020008)
Topics: Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › CRISPR and Genetic Engineering
Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
Keywords: CRISPR/Cas9 delivery, non-viral gene delivery, intracranial injection, fragile X syndrome, repetitive behavior, metabotropic glutamate receptor 5, mGluR5, CRISPR/Cas12a, striatum, autism spectrum disorder, astrocytes, microglia, neurons
MeSH terms: Animals, Behavior, Animal, Brain, Corpus Striatum, Disease Models, Animal, Fragile X Syndrome, Gold, Humans, Mice, Inbred C57BL, Neurons, Mice, Knockout, Patch-Clamp Techniques, Thy-1 Antigens, Mice, Fragile X Messenger Ribonucleoprotein 1, Nanoparticles, HEK293 Cells, Clustered Regularly Interspaced Short Palindromic Repeats, CRISPR-Cas Systems, Receptor, Metabotropic Glutamate 5

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

Nanoparticle delivery of CRISPR into the brain rescues a mouse model of fragile X syndrome from exaggerated repetitive behaviours

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