The DOI matches its Crossref record; 20 of the 20 references it cites were found in a registry or an index.
Correction of a Genetic Disease in Mouse via Use of CRISPR-Cas9
Open accessAbstract
The CRISPR-Cas9 system has been employed to generate mutant alleles in a range of different organisms. However, so far there have not been reports of use of this system for efficient correction of a genetic disease. Here we show that mice with a dominant mutation in Crygc gene that causes cataracts could be rescued by coinjection into zygotes of Cas9 mRNA and a single-guide RNA (sgRNA) targeting the mutant allele. Correction occurred via homology-directed repair (HDR) based on an exogenously supplied oligonucleotide or the endogenous WT allele, with only rare evidence of off-target modifications. The resulting mice were fertile and able to transmit the corrected allele to their progeny. Thus, our study provides proof of principle for use of the CRISPR-Cas9 system to correct genetic disease.
Overview
Integrity signals
Registry
Cites
Statistics
Verification
Claims the paper makes
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?
In the citing paper
“Due to the low rate of HDR recombination [109]”
From CRISPR-Cas: Converting A Bacterial Defence Mechanism into A State-of-the-Art Genetic Manipulation Tool (2019) · cites this paper as [109]
In the citing paper
“Several groups have also demonstrated in vitro correction of genetic disease genes including CFTR (cystic fibrosis)9, Crygc (cataracts)10 and DMD (Duchenne muscular dystrophy)11 in organoids or mouse zygotes.”
From Therapeutic genome editing by combined viral and non-viral delivery of CRISPR system components in vivo (2016) · cites this paper as [10]
In the citing paper
“Furthermore, CRISPR/Cas9 system holds tremendous promise for gene therapy [46–51]. It can correct causal mutations in the original genome and rescue the disease phenotypes of monogenic disorders permanently, which currently represents the most translatable field in CRISPR/Cas9-mediated disease therapy.”
From Non-viral delivery systems for CRISPR/Cas9-based genome editing: challenges and opportunities (2018) · cites this paper as [47]
In the citing paper
“CRISPR-Cas9 genome editing has been applied to correct disease-causing mutations in mouse zygotes and human cell lines for cataract5 and cystic fibrosis6”
From Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype (2014) · cites this paper as [5]
In the citing paper
“In such a mouse model, germline-manipulation with CRISPR-Cas9 system was capable of correcting both the mutant gene and cataract phenotype in part of offsprings.”
From CRISPR-Cas9: a new and promising player in gene therapy (2015) · cites this paper as [30]
In the citing paper
“Interestingly, however, of the six offsprings undergoing NHEJ, two were also free of cataract thanks to coincidental frameshift restoration resulting from NHEJ-induced indel mutations, while the rest four remained cataract phenotype.30”
From CRISPR-Cas9: a new and promising player in gene therapy (2015) · cites this paper as [30]
In the citing paper
“These results highlighted the importance of HDR in reliable gene corrections.”
From CRISPR-Cas9: a new and promising player in gene therapy (2015) · cites this paper as [30]
In the citing paper
“Of the 22 live offsprings, 10 underwent culprit gene modifications (the editing efficiency is 45.45%) either by HDR (n=4) (figure 1C left) or by NHEJ (n=6) (figure 1C right).”
From CRISPR-Cas9: a new and promising player in gene therapy (2015) · cites this paper as [30]
In the citing paper
“However, as demonstrated in this study, zygote manipulation can only rescue part of the offsprings in an unpredictable manner.”
From CRISPR-Cas9: a new and promising player in gene therapy (2015) · cites this paper as [30]
In the citing paper
“As expected, all of the four offsprings undergoing HDR were free of cataract due to HDR-mediated gene corrections.”
From CRISPR-Cas9: a new and promising player in gene therapy (2015) · cites this paper as [30]
In the citing paper
“As shown in the study by Wu et al 30 among mice genetically edited by CRISPR-Cas9, all HDR-mediated editing corrected the causative gene defects and rescued disease phenotype while only a small part of NHEJ-mediated editing had the corrective effect.”
From CRISPR-Cas9: a new and promising player in gene therapy (2015) · cites this paper as [30]
Citations
Cites
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
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
ZFN, TALEN, and CRISPR/Cas-based methods for genome engineering
Gaj, T., Gersbach, C. A., & Barbas, C. F. (2013) · Trends in Biotechnology · doi:10.1016/j.tibtech.2013.04.004 · Cited by 3,820
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
High-Frequency off-Target Mutagenesis Induced by CRISPR-Cas Nucleases in Human Cells
Fu, Y., Foden, J. A., Khayter, C., Maeder, M. L., Reyon, D., Joung, J. K., & Sander, J. D. (2013) · Nature Biotechnology · doi:10.1038/nbt.2623 · Cited by 3,292
Efficient genome editing in zebrafish using a CRISPR-Cas system
Hwang, W. Y., Fu, Y., Reyon, D., Maeder, M. L., Tsai, S. Q., Sander, J. D., Peterson, R. T., Yeh, J.-R. J., & Joung, J. K. (2013) · Nature Biotechnology · doi:10.1038/nbt.2501 · Cited by 2,937
RNA-programmed genome editing in human cells
Jinek, M., East, A., Cheng, A., Lin, S., Ma, E., & Doudna, J. (2013) · eLife · doi:10.7554/elife.00471 · Cited by 2,098
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
Yang, H., Wang, H., Shivalila, C. S., Cheng, A. W., Shi, L., & Jaenisch, R. (2013) · Cell · doi:10.1016/j.cell.2013.08.022 · Cited by 1,596
Flagged first, then by citations
Cited by
Sorted by citations
Items 201–300 of 619
Homology-based repair induced by CRISPR-Cas nucleases in mammalian embryo genome editing
Zhang, X., Li, T., Ou, J., Huang, J., & Liang, P. (2021) · Protein & Cell · doi:10.1007/s13238-021-00838-7 · Cited by 47
“In addition to exogenous DNA donors, endogenous homologous DNA can be utilized as an HBR template in mouse and human tripronuclear zygotes (Wu et al., 2013; Liang et al., 2015).”
BackgroundEfficient Correction of a Hypertrophic Cardiomyopathy Mutation by ABEmax-NG
Ma, S., Jiang, W., Liu, X., Lu, W., Qi, T., Wei, J., Wu, F., Chang, Y., Zhang, S., Song, Y., et al. (2021) · Circulation Research · doi:10.1161/circresaha.120.318674 · Cited by 45
New advances in CRISPR/Cas-mediated precise gene-editing techniques
Richardson, C., Kelsh, R. N., & Richardson, R. J. (2023) · Disease Models & Mechanisms · doi:10.1242/dmm.049874 · Cited by 48
“Despite these challenges, PGE has been achieved in several model organisms, including mice (Miura et al., 2018; Wu et al., 2013), zebrafish (Hoshijima et al., 2016; Irion et al., 2014; Wierson et al., 2020; Zhang et al., 2018) rabbits (Song et al., 2016; Song et al., 2018), pigs (Song et al., 2016;…”
Genome editing strategies: potential tools for eradicating HIV-1/AIDS
Khalili, K., Kaminski, R., Gordon, J., Cosentino, L., & Hu, W. (2015) · Journal of NeuroVirology · doi:10.1007/s13365-014-0308-9 · Cited by 51
“Although unbiased assays for off-target effects and continuous improvement of genome editing tools are still required, apparent genotoxicity, cytotoxicity, and off-target effects were not observed in most cases, particularly for Cas9-induced genome editing in both cells and animals (Cho et al. 2014; Fu et al. 2013; Ga…”
BackgroundGene Editing and Genetic Lung Disease. Basic Research Meets Therapeutic Application
Alapati, D., & Morrisey, E. E. (2016) · American Journal of Respiratory Cell and Molecular Biology · doi:10.1165/rcmb.2016-0301ps · Cited by 47
“Finally, a mouse model of cataract caused by a dominant mutation in the Crygc gene was corrected using CRISPR/Cas9 in the germline (20).”
BackgroundCRISPR/Cas9: the Jedi against the dark empire of diseases
Khan, S., Mahmood, M. S., Rahman, S. U., Zafar, H., Khan, S. H., khan, Z., & Ahmad, A. (2018) · Journal of Biomedical Science · doi:10.1186/s12929-018-0425-5 · Cited by 49
“The cataract phenotype is caused by a frame-shift mutation of one base pair deletion in exon 3 of Crygc (crystalline gamma c) [77].”
CRISPR/Cas system: A game changing genome editing technology, to treat human genetic diseases
Hussain, W., Mahmood, T., Hussain, J. S., Ali, N., Shah, T. M., Qayyum, S., & Khan, I. (2018) · Gene · doi:10.1016/j.gene.2018.10.072 · Cited by 46
A review of nanocellulose in the drug-delivery system
Tan, T. H., Lee, H. V., Yehye, W. A., & Hamid, S. B. O. A. A. (2019) · Elsevier eBooks · doi:10.1016/b978-0-12-816913-1.00005-2 · Cited by 48
Programmable Molecular Scissors: Applications of a New Tool for Genome Editing in Biotech
Saha, S. K., Saikot, F. K., Rahman, M. S., Jamal, M. A. H. M., Rahman, S. M. K., Islam, S. M. R., & Kim, K. (2018) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2018.11.016 · Cited by 48
Feng, W., Dai, Y., Mou, L., Cooper, D. K. C., Shi, D., & Cai, Z. (2015) · International Journal of Molecular Sciences · doi:10.3390/ijms16036545 · Cited by 44
“CRISPR/Cas9-mediated editing has been applied to various cells and animals [25–33].”
MethodZhao, L., Luo, Y., Huang, Q., Cao, Z., & Yang, X. (2020) · Small · doi:10.1002/smll.202004879 · Cited by 44
Position 34 of tRNA is a discriminative element for m5C38 modification by human DNMT2
Huang, Z., Li, J., Xiong, Q., Li, H., Wang, E., & Liu, R. (2021) · Nucleic Acids Research · doi:10.1093/nar/gkab1148 · Cited by 45
“Sense and antisense oligonucleotides for the sgRNAs were cloned into vector pX330-mCherry (plasmid #98750; Addgene, Watertown, MA, USA) (51).”
The Intrinsic and Extrinsic Effects of Tet Proteins During Gastrulation
Cheng, S., Mittnenzweig, M., Mayshar, Y., Lifshitz, A., Dunjić, M., Rais, Y., Ben-Yair, R., Gehrs, S., Chomsky, E., Mukamel, Z., et al. (2021) · Cell · doi:10.1016/j.cell.2022.06.049 · Cited by 43
Ma, H., Martí‐Gutiérrez, N., Park, S.-W., Wu, J., Hayama, T., Darby, H., Dyken, C. V., Li, Y., Koski, A., Liang, D., et al. (2018) · Nature · doi:10.1038/s41586-018-0381-y · Cited by 45
Song, M. (2017) · Biotechnology Progress · doi:10.1002/btpr.2484 · Cited by 46
“In each case, the rescued mice were fertile and could transmit the corrected allele to the next generation.(85,86)”
BackgroundKang, X., Caparas, C. I. N., Soh, B.-S., & Fan, Y. (2017) · Protein & Cell · doi:10.1007/s13238-017-0477-4 · Cited by 49
CRISPR/Cas9-mediated correction of human genetic disease
Men, K., Duan, X., He, Z., Yang, Y., Yao, S., & Wei, Y. (2017) · Science China Life Sciences · doi:10.1007/s11427-017-9032-4 · Cited by 44
Thrombopoietin knock-in augments platelet generation from human embryonic stem cells
Zhang, L., Liu, C., Wang, H., Wu, D., Su, P., Wang, M., Guo, J., Zhao, S., Dong, S., Zhou, W., et al. (2018) · Stem Cell Research & Therapy · doi:10.1186/s13287-018-0926-x · Cited by 42
Wang, Y., Yan, M., Zhang, X., Liu, X., Ding, Y., Lai, C.-P., Tong, M., & Li, J. (2021) · Asian Journal of Andrology · doi:10.4103/aja.aja_97_20 · Cited by 42
Unmasking the ciliopathies: craniofacial defects and the primary cilium
Cortés, C. R., Metzis, V., & Wicking, C. (2015) · Wiley Interdisciplinary Reviews Developmental Biology · doi:10.1002/wdev.199 · Cited by 39
Minimizing off-Target Mutagenesis Risks Caused by Programmable Nucleases
Ishida, K., Gee, P., & Hotta, A. (2015) · International Journal of Molecular Sciences · doi:10.3390/ijms161024751 · Cited by 41
“The CRISPR Cas9 system also has tremendous therapeutic potential [17] and has been shown to correct genetic mutations [18] such as β-thalassemia [19], cystic fibrosis [20], hemophilia A [21], cataracts [22], hereditary tyrosinemia type I [23], and Duchenne muscular dystrophy [24].”
BackgroundRubio, A., Luoni, M., Giannelli, S. G., Radice, I., Iannielli, A., Cancellieri, C., Berardino, C. D., Regalia, G., Lazzari, G., Menegon, A., et al. (2016) · Scientific Reports · doi:10.1038/srep37540 · Cited by 41
The Roles of TGF-β Signaling in Cerebrovascular Diseases
Zhang, Y., & Yang, X. Y. (2020) · Frontiers in Cell and Developmental Biology · doi:10.3389/fcell.2020.567682 · Cited by 45
CRISPR-based strategies for targeted transgene knock-in and gene correction
Lau, C., Tin, C., & Suh, Y. (2020) · Faculty Reviews · doi:10.12703/r/9-20 · Cited by 46
The AAV-mediated and RNA-guided CRISPR/Cas9 system for gene therapy of DMD and BMD
Wang, J.-Z., Wu, P., Shi, Z.-M., Xu, Y.-L., & Liu, Z.-J. (2017) · Brain and Development · doi:10.1016/j.braindev.2017.03.024 · Cited by 41
“could be deleted by utilizing Cas9 and a number of welldesigned gRNAs [1,21,29,32,33].”
Sato, M., Takabayashi, S., Akasaka, E., & Nakamura, S. (2020) · Cells · doi:10.3390/cells9040799 · Cited by 40
The Bibliometric Landscape of Gene Editing Innovation and Regulation in the Worldwide
Wei, X., Pu, A., Liu, Q., Hou, Q., Zhang, Y., An, X., Long, Y., Jiang, Y., Dong, Z., Wu, S., et al. (2022) · Cells · doi:10.3390/cells11172682 · Cited by 39
“In addition, CRISPR/Cas9 has made remarkable progress in the gene therapy of human hereditary tyrosinemia [123], hemophilia [124], intestinal neoplasia [36,37], lung adenocarcinoma [38], cataracts [39], obesity [30] and other diseases.”
BackgroundBelmadi, N., Midoux, P., Loyer, P., Passirani, C., Pichon, C., Gall, T. L., Jaffrès, P., Lehn, P., & Montier, T. (2015) · Biotechnology Journal · doi:10.1002/biot.201400841 · Cited by 46
“This technique was also effectively applied to mouse eggs to correct a dominant mutation in the Crygc gene known to cause cataracts [121].”
Multiplex precise base editing in cynomolgus monkeys
Zhang, W., Aida, T., del Rosario, R. C. H., Wilde, J. J., Ding, C., Zhang, X., Baloch, Z., Huang, Y., Tang, Y., Li, D., et al. (2020) · Nature Communications · doi:10.1038/s41467-020-16173-0 · Cited by 42
Myoediting: Toward Prevention of Muscular Dystrophy by Therapeutic Genome Editing
Zhang, Y., Long, C., Bassel‐Duby, R., & Olson, E. N. (2018) · Physiological Reviews · doi:10.1152/physrev.00046.2017 · Cited by 42
“A ssODN can also serve as a repair template for HDR, especially for introducing small DNA modifications (231, 329, 441, 469).”
BackgroundAllele-specific genome targeting in the development of precision medicine
Wu, J., Tang, B., & Tang, Y. (2020) · Theranostics · doi:10.7150/thno.43298 · Cited by 43
Human cell based directed evolution of adenine base editors with improved efficiency
Fu, J., Li, Q., Liu, X., Tu, T., Lv, X., Yin, X., Lv, J., Song, Z., Qu, J., Zhang, J., et al. (2021) · Nature Communications · doi:10.1038/s41467-021-26211-0 · Cited by 35
Modeling a cataract disorder in mice with prime editing
Lin, J., Liu, X., Lu, Z., Huang, S., Wu, S., Yu, W., Liu, Y., Zheng, X., Huang, X., Sun, Q., et al. (2021) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2021.06.020 · Cited by 38
“Interestingly, twomice that were born bymice #3 and #15, respectively, were observed with cataract disorders as expected, whereas the other three mice carrying heterozygous G-deletion displayed normal lenses (Figure 3A), which was also observed in a previous study.(5) Moreover, the heterozygous genotype from Sanger se…”
MethodResultApplications of CRISPR-Cas9 mediated genome engineering
Yang, X. P. (2015) · Military Medical Research · doi:10.1186/s40779-015-0038-1 · Cited by 38
“The CRISPRCas9 system has been shown to efficiently correct a dominant Crygc gene mutation in a cataracts mouse model by co-injecting Cas9 mRNA and sgRNA targeting the mutant Crygc allele into zygotes [66].”
Epstein-Barr virus-driven B cell lymphoma mediated by a direct LMP1-TRAF6 complex
Giehler, F., Ostertag, M. S., Sommermann, T. G., Weidl, D., Sterz, K. R., Kutz, H., Moosmann, A., Feller, S. M., Geerlof, A., Biesinger, B., et al. (2024) · Nature Communications · doi:10.1038/s41467-023-44455-w · Cited by 36
High-Throughput Silencing Using the CRISPR-Cas9 System: A Review of the Benefits and Challenges
Wade, M. R. (2015) · SLAS DISCOVERY · doi:10.1177/1087057115587916 · Cited by 39
A possible aid in targeted insertion of large DNA elements by CRISPR/Cas in mouse zygotes
Nakao, H., Harada, T., Nakao, K., Kiyonari, H., Inoue, K., Furuta, Y., & Aiba, A. (2015) · genesis · doi:10.1002/dvg.22914 · Cited by 36
“We next examined potential off-target (OT) mutations in five of the obtained founder mice according to two methods described in previous reports (Wang et al., 2013; Yang et al., 2013).”
BackgroundMethodResultInfluentialThe expanding footprint of CRISPR/Cas9 in the plant sciences
Schaeffer, S. M., & Nakata, P. A. (2016) · Plant Cell Reports · doi:10.1007/s00299-016-1987-x · Cited by 37
The application and progression of CRISPR/Cas9 technology in ophthalmological diseases
Hu, X., Zhang, B., Li, X., Li, M., Wang, Y., Dan, H., Zhou, J., Wei, Y., Ge, K., Li, P., et al. (2022) · Eye · doi:10.1038/s41433-022-02169-1 · Cited by 37
“[38] were the first to report a mouse model of congenital cataract caused by a specific mutation in the CRYGC; these mice carried a 1-bp deletion mutation in exon 3 of the CRYGC gene, which the translation arrest at the 76th amino acid.”
BackgroundBaker, M. (2014) · Nature Biotechnology · doi:10.1038/nbt.2863 · Cited by 36
Programmable Site-Specific Nucleases for Targeted Genome Engineering in Higher Eukaryotes
Govindan, G., & Ramalingam, S. (2016) · Journal of Cellular Physiology · doi:10.1002/jcp.25367 · Cited by 38
“The gene corrected mice are fertile and the corrected allele was transmitted to their progeny (Wu et al., 2013).”
BackgroundCRISPR-Cas9 Mediated Genome Editing in Bicyclus anynana Butterflies
Banerjee, T. D., & Monteiro, A. (2018) · Methods and Protocols · doi:10.3390/mps1020016 · Cited by 30
“CRISPR-Cas9 has also been applied to correct genetic disorders in mice [45,46] and humans [47,48].”
CRISPR technology for genome editing
Bhattacharjee, G., Mani, I., Gohil, N., Khambhati, K., Braddick, D., Panchasara, H., & Singh, V. (2019) · Elsevier eBooks · doi:10.1016/b978-0-12-819178-1.00007-1 · Cited by 36
AKT1/FOXP3 axis-mediated expression of CerS6 promotes p53 mutant pancreatic tumorigenesis
Qi, D., Song, X., Xue, C., Yao, W., Shen, P., Yu, H. T., & Zhang, Z. (2021) · Cancer Letters · doi:10.1016/j.canlet.2021.06.024 · Cited by 35
Bacterial Delivery of TALEN Proteins for Human Genome Editing
Jia, J., Jin, Y., Bian, T., Wu, D., Yang, L., Terada, N., Wu, W., & Jin, S. (2014) · PLoS ONE · doi:10.1371/journal.pone.0091547 · Cited by 35
“Recently,Wu et al. have shown in mice that a dominant mutation in Crygc gene that causes cataracts could be rescued by Cas9 mediated DSB on the mutant allele which triggered homology-directed repair based on the endogenous WT allele, raising the hope for the application of current TALEN PLOS ONE | www.plosone.org 5 Ma…”
BackgroundInfluentialExosomes for CRISPR-Cas9 Delivery: The Cutting Edge in Genome Editing
Aslan, C., Zolbanin, N. M., Faraji, F., & Jafari, R. (2023) · Molecular Biotechnology · doi:10.1007/s12033-023-00932-7 · Cited by 35
“By targeting zygotes and spermatogonial stem cells, this study proved the concept of curing genetic diseases in offspring [138, 139].”
Li, Y., Wang, R., Qiao, N., Peng, G., Zhang, K., Tang, K., Han, J. J., & Jing, N. (2017) · Journal of Biological Chemistry · doi:10.1074/jbc.m117.796383 · Cited by 37
Stem cell transplantation for Huntington’s diseases
Choi, K., Choi, Y., & Hong, S. (2017) · Methods · doi:10.1016/j.ymeth.2017.08.017 · Cited by 34
Henser‐Brownhill, T., Monserrat, J., & Scaffidi, P. (2017) · Epigenetics · doi:10.1080/15592294.2017.1395121 · Cited by 33
Rysenkova, K. D., Semina, E. V., Karagyaur, M. N., Shmakova, A., Dyikanov, D. T., Vasiluev, P. A., Rubtsov, Y. P., Rubina, K. A., & Va, T. (2018) · Oncotarget · doi:10.18632/oncotarget.25647 · Cited by 33
“The oligonucleotides sg1, sg1c, sg2, sg2c were phosphorylated, denatured and annealed according to the protocol [37].”
BackgroundMethodDosage effect of multiple genes accounts for multisystem disorder of myotonic dystrophy type 1
Yin, Q., Wang, H., Li, N., Ding, Y., Xie, Z., Jin, L., Li, Y., Wang, Q., Liu, X., Xu, L., et al. (2019) · Cell Research · doi:10.1038/s41422-019-0264-2 · Cited by 33
“2a).(53) Ten stable cell lines carrying mutant Dmwd gene (termed ΔDmwd-O48) were obtained (Fig.”
BackgroundMethodSuper-Mendelian inheritance mediated by CRISPR/Cas9 in the female mouse germlinePreprint
Grunwald, H. A., Gantz, V. M., Poplawski, G. H. D., Xu, X., Bier, E., & Cooper, K. L. (2018) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/362558 · Cited by 33
Su, J., Huang, F., Yu, T., Tian, R., Qianqian, G., Bello, S. T., Zeng, D., Jendrichovsky, P., Lau, C. G., Xiong, W., et al. (2023) · Cell Reports · doi:10.1016/j.celrep.2023.113467 · Cited by 36
Engineered CRISPR Systems for Next Generation Gene Therapies
Pineda, M., Moghadam, F., Ebrahimkhani, M. R., & Kiani, S. (2017) · ACS Synthetic Biology · doi:10.1021/acssynbio.7b00011 · Cited by 30
Recent Advances in the Production of Genome-Edited Rats
Sato, M., Nakamura, S., Inada, E., & Takabayashi, S. (2022) · International Journal of Molecular Sciences · doi:10.3390/ijms23052548 · Cited by 31
“Numerous reports have demonstrated that CRISPR/Cas9 editors combined with ~100 bp ssODNs, instead of using a relatively long template (~1 kb), allow for precise HDR-mediated GE in mice [156,157,193].”
BackgroundTremblay, J. P. (2015) · médecine/sciences · doi:10.1051/medsci/20153111016 · Cited by 39
CRISPR/Cas9—Advancing Orthopoxvirus Genome Editing for Vaccine and Vector Development
Okoli, A. S., Okeke, M. I., Tryland, M., & Moens, U. L. (2018) · Viruses · doi:10.3390/v10010050 · Cited by 33
FREQUENT GENE CONVERSION IN HUMAN EMBRYOS INDUCED BY DOUBLE STRAND BREAKSPreprint
Liang, D., Gutiérrez, N. M., Chen, T., Lee, Y., Park, S.-W., Ma, H., Koski, A., Ahmed, R., Darby, H., Li, Y., et al. (2020) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/2020.06.19.162214 · Cited by 31
Temporal BMP4 effects on mouse embryonic and extraembryonic development
Hadas, R., Rubinstein, H., Mittnenzweig, M., Mayshar, Y., Ben-Yair, R., Cheng, S., Aguilera-Castrejon, A., Reines, N., Orenbuch, A.-H., Lifshitz, A., et al. (2024) · Nature · doi:10.1038/s41586-024-07937-5 · Cited by 32
Lipid nanoparticles: a promising tool for nucleic acid delivery in cancer immunotherapy
Abaza, T., Mohamed, E., & Zaky, M. Y. (2025) · Medical Oncology · doi:10.1007/s12032-025-02939-3 · Cited by 35
“CRISPR/Cas9 has become a potent instrument for comprehending and addressing the genetic origins of diverse diseases [202, 203].”
The Rise of CRISPR/Cas for Genome Editing in Stem Cells
Shui, B., Hernández-Matías, L., Guo, Y., & Peng, Y. (2016) · Stem Cells International · doi:10.1155/2016/8140168 · Cited by 33
“The Cas9 proteins of Streptococcus thermophilus , Neisseria meningitidis , and Treponema denticola demonstrated comparable genome-editing efficiency to spCas9 (Table 1) [24–27].”
福田, 展., Senga, Y., & Honda, S. (2019) · Biotechnology Progress · doi:10.1002/btpr.2820 · Cited by 29
Rabl2 GTP hydrolysis licenses BBSome‐mediated export to fine‐tune ciliary signaling
Duan, S., Li, H., Zhang, Y., Yang, S., Chen, Y., Qiu, B., Huang, C., Wang, J., Li, J., Zhu, X., et al. (2020) · The EMBO Journal · doi:10.15252/embj.2020105499 · Cited by 27
“Rabl2 Q80L knock-in ( Rabl2 KI / KI ) mice that endogenously expressed Rabl2 Q80L were generated using a CRISPR/Cas9 genome editing protocol as described previously (Wu et al , 2013).”
CRISPR-Cas9 for treating hereditary diseases
Mani, I. (2021) · Progress in molecular biology and translational science · doi:10.1016/bs.pmbts.2021.01.017 · Cited by 33
Off- and on-target effects of genome editing in mouse embryos
Ayabe, S., NAKASHIMA, K., & Yoshiki, A. (2018) · Journal of Reproduction and Development · doi:10.1262/jrd.2018-128 · Cited by 31
Liu, P., Dou, X., Liu, C., Wang, L., Xing, C., Peng, G., Chen, J., Yu, F., Qiao, Y., Song, L., et al. (2015) · Nature Communications · doi:10.1038/ncomms7830 · Cited by 28
La révolution des CRISPR est en marche
Gilgenkrantz, H. (2014) · médecine/sciences · doi:10.1051/medsci/20143012004 · Cited by 27
CRISPR/Cas9 system: a powerful technology for in vivo and ex vivo gene therapy
Zhang, X., Wang, L., Liu, M., & Li, D. (2017) · Science China Life Sciences · doi:10.1007/s11427-017-9057-2 · Cited by 28
“The corrected SSCs were used to generate healthy offspring (Wu et al., 2013; Wu et al., 2015).”
BackgroundMethodCRISPR/Cas9 increases mitotic gene conversion in human cells
Javidi-Parsijani, P., Lyu, P., Makani, V. S., Sarhan, W. M., Yoo, K. W., El-Korashi, L. A., Atala, A., & Lu, B. (2020) · Gene Therapy · doi:10.1038/s41434-020-0126-z · Cited by 29
Rabinowitz, R., Almog, S., Darnell, R., & Offen, D. (2020) · Frontiers in Genetics · doi:10.3389/fgene.2020.00851 · Cited by 29
“Moreover, studies have shown that allele-specific targeting in heterozygous cells results in high rates of gene conversion (14–47%), where the homologous chromosome serves as a template for homologous recombination to resolve the double-strand break (Wu et al., 2013; Filler Hayut et al., 2017).”
BackgroundPhaneuf, C. R., Seamon, K. J., Eckles, T., Sinha, A., Schoeniger, J. S., Harmon, B., Meagher, R. J., Abhyankar, V. V., & Koh, C. (2019) · Analytical Methods · doi:10.1039/c8ay02726a · Cited by 27
Development and Characterization of MDR1 (Mdr1a/b) CRISPR/Cas9 Knockout Rat Model
Liang, C., Zhao, J., Lu, J., Zhang, Y., Ma, X., Shang, X., Li, Y., Ma, X., Liu, M., & Wang, X. (2018) · Drug Metabolism and Disposition · doi:10.1124/dmd.118.084277 · Cited by 27
“Compared with ZFN and transcription activator–like effector nuclease (TALEN), the CRISPR/Cas9 system can edit multiple genes simultaneously and has few species limitations (Wu et al., 2013; Ceasar et al., 2016).”
BackgroundUnraveling the potential of CRISPR-Cas9 for gene therapy
Barrangou, R., & May, A. P. (2014) · Expert Opinion on Biological Therapy · doi:10.1517/14712598.2015.994501 · Cited by 27
“For example, a model of Crygc-associated cataract, linked to a 1 bp deletion in exon 3, can be rescued in mice by co-injection of Cas9 mRNA, target sgRNA and WT allele HDR template into embryos [5].”
BackgroundThe pathogenesis of common Gjb2 mutations associated with human hereditary deafness in mice
Li, Q., Cui, C., Liao, R., Yin, X., Wang, D., Cheng, Y., Huang, B., Wang, L., Yan, M., Zhou, J., et al. (2023) · Cellular and Molecular Life Sciences · doi:10.1007/s00018-023-04794-9 · Cited by 26
The evolution of CRISPR/Cas9 and their cousins: hope or hype?
Bhushan, K., Chattopadhyay, A., & Pratap, D. (2018) · Biotechnology Letters · doi:10.1007/s10529-018-2506-7 · Cited by 27
“Cas9 technology has been used for genome editing in mouse (Li et al. 2013; Wu et al. 2013; Yin et al. 2013),”
MethodInto the Fourth Dimension: Dysregulation of Genome Architecture in Aging and Alzheimer’s Disease
Winick‐Ng, W., & Rylett, R. J. (2018) · Frontiers in Molecular Neuroscience · doi:10.3389/fnmol.2018.00060 · Cited by 28
“…nuclease 9 (CRISPR-Cas9) genome editing system has been used to explore gene therapy potential for many diseases, including HIV (Ye et al., 2014), several cancers (reviewed by Xiao-Jie et al., 2015), cataracts in mouse (Wu et al., 2013) and muscular dystrophy (Long et al., 2014), among others.”
BackgroundMethodIn vivo targeted single-nucleotide editing in zebrafish
Tanaka, S., Yoshioka, S., Nishida, K., Hosokawa, H., Kakizuka, A., & Maegawa, S. (2018) · Scientific Reports · doi:10.1038/s41598-018-29794-9 · Cited by 25
Pancreatic Progenitors and Organoids as a Prerequisite to Model Pancreatic Diseases and Cancer
Hohwieler, M., Müller, M., Frappart, P., & Heller, S. (2019) · Stem Cells International · doi:10.1155/2019/9301382 · Cited by 27
Humanin vitrofertilisation and developmental biology: a mutually influential history
Johnson, M. H. (2019) · Development · doi:10.1242/dev.183145 · Cited by 26
“The possiblewider treatment of genetic diseases is being studied using modern techniques of gene editing, such as CRISPR/ Cas9, which have been successfully applied in mouse eggs and zygotes (Wu et al., 2013; Wang et al., 2013) as well as in human triploid eggs (Liang et al.”
BackgroundMianné, J., Nasri, A., Van, C. N., Bourguignon, C., Fieldès, M., Ahmed, E., Duthoit, C. T., Martin, N., Parrinello, H., Louis, A., et al. (2022) · BMC Biology · doi:10.1186/s12915-021-01214-x · Cited by 23
“[31], rat [36], and human embryos [37–39].”
BackgroundA Transgenic Core Facility’s Experience in Genome Editing Revolution
Yuan, C. L., & Hu, Y. (2017) · Advances in experimental medicine and biology · doi:10.1007/978-3-319-63904-8_4 · Cited by 26
Cottle, R. N., Lee, C. M., Archer, D. R., & Bao, G. (2015) · Scientific Reports · doi:10.1038/srep16031 · Cited by 26
“Compared with other delivery methods for genome editing, such as cationic lipid transfection, nucleofection, viral-based delivery and receptor-mediated protein uptake42, microinjection has potential advantages, including: (1) precise control of the amount delivered into single cells, (2) applicable to a wide range of…”
BackgroundTherapeutic applications of CRISPR RNA-guided genome editing
Koo, T., & Kim, J. (2016) · Briefings in Functional Genomics · doi:10.1093/bfgp/elw032 · Cited by 24
Bae, T., Hur, J. W., Kim, D., & Hur, J. K. (2019) · Genes & Genomics · doi:10.1007/s13258-019-00830-w · Cited by 26
Enhancer and super-enhancer landscape in polycystic kidney disease
Lakhia, R., Mishra, A., Biggers, L. M., Malladi, V. S., Cobo-Stark, P., Hajarnis, S. S., & Patel, V. (2021) · Kidney International · doi:10.1016/j.kint.2022.08.039 · Cited by 23
Breaking bad: The mutagenic effect of DNA repair
Chen, J., & Furano, A. V. (2015) · DNA repair · doi:10.1016/j.dnarep.2015.04.012 · Cited by 27
“As DNA nicking is essential forgenome editing and APOBECSs are commonly expressed in most cells and tissues [106, 107], the possibility of APOBEC-mediated mutationsis an important concern for the highly precise Cas9-directed genome editing that is necessary for therapeutic gene correction [108-110].”
BackgroundLipid and polymer mediated CRISPR/Cas9 gene editing
Gong, Y., Tian, S., Yang, X., & Zhang, S. (2020) · Journal of Materials Chemistry B · doi:10.1039/d0tb00207k · Cited by 25
2015 Guidelines for establishing genetically modified rat models for cardiovascular research
Flister, M. J., Prokop, J. W., Lazar, J., Shimoyama, M., Dwinell, M., & Geurts, A. (2015) · Journal of Cardiovascular Translational Research · doi:10.1007/s12265-015-9626-4 · Cited by 24
A Doxycycline-Inducible System for Genetic Correction of iPSC Disease Models
Sim, X., Cardenas‐Diaz, F. L., French, D. L., & Gadue, P. J. (2014) · Methods in molecular biology · doi:10.1007/7651_2014_179 · Cited by 24
Leitão, A. L., & Enguita, F. J. (2014) · Microbiological Research · doi:10.1016/j.micres.2014.02.007 · Cited by 25
Versatile High-Throughput Fluorescence Assay for Monitoring Cas9 Activity
Seamon, K. J., Light, Y. K., Saada, E. A., Schoeniger, J. S., & Harmon, B. (2018) · Analytical Chemistry · doi:10.1021/acs.analchem.8b01155 · Cited by 24
Yuan, Y., Zhou, Q., WAN, H., Shen, B., Wang, X., Wang, M., Feng, C., Xie, M., Gu, T., Zhou, T., et al. (2015) · Cell Research · doi:10.1038/cr.2015.74 · Cited by 24
In vivo gene therapy potentials of CRISPR-Cas9
Xue, H.-Y., Zhang, X., Wang, Y., Lu, X., Dai, W.-J., & Xu, Y. C. (2016) · Gene Therapy · doi:10.1038/gt.2016.25 · Cited by 24
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
The Impact of CRISPR-Cas9 on Age-related Disorders: From Pathology to Therapy
Caobi, A., Dutta, R. K., Garbinski, L. D., Esteban-López, M., Ceyhan, Y., Andre, M., Manevski, M., Ojha, C. R., Lapierre, J., Tiwari, S., et al. (2020) · Aging and Disease · doi:10.14336/ad.2019.0927 · Cited by 24
“the gene could be rescued by targeting the mutant allele with a sgRNA co-injected with Cas9 mRNA into zygotes [43].”
BackgroundAljanahi, A. A., Lazzarotto, C. R., Chen, S., Shin, T., Cordes, S., Fan, X., Jabara, I., Zhou, Y., Young, D. J., Lee, B., et al. (2021) · Molecular Therapy · doi:10.1016/j.ymthe.2021.06.016 · Cited by 27
CRISPR-sub: Analysis of DNA substitution mutations caused by CRISPR-Cas9 in human cells
Hwang, G., Yu, J., Yang, S., Son, W. J., Lim, K., Kim, H. S., Kim, J., & Bae, S. (2020) · Computational and Structural Biotechnology Journal · doi:10.1016/j.csbj.2020.06.026 · Cited by 23
CRISPR-Cas9-mediated gene editing in human MPS I fibroblasts
de Carvalho, T. G., Schuh, R. S., Pasqualim, G., Pellenz, F. M., Filippi‐Chiela, E. C., Giugliani, R., Baldo, G., & Matte, Ú. D. S. (2018) · Gene · doi:10.1016/j.gene.2018.08.004 · Cited by 22
The evolving CRISPR technology
Yan, M., & Li, J. (2019) · Protein & Cell · doi:10.1007/s13238-019-0645-9 · Cited by 22
Metadata
- Authors
- Wu, Y., Liang, D., Wang, Y., Bai, M., Tang, W., Bao, S., Yan, Z., Li, D., & Li, J.
- Affiliations
- Chinese Academy of Sciences (CN)
- Corresponding author
- Li, Jinsong
- Venue
- Cell stem cell
- Publisher
- Elsevier BV
- Published
- 2013-12
- Volume
- 13
- Issue
- 6
- Pages
- 659-662
- ISSN
- 1875-9777 (ISSN-L)
- 1934-5909
- OpenAlex
- W2058792860
- Cited by
- 614
- References
- 20
- Citations by year
- 2026: 14 · 2025: 19 · 2024: 26 · 2023: 34 · 2022: 34 · 2021: 51 · 2020: 51 · 2019: 61 · 2018: 67 · 2017: 64 · 2016: 69 · 2015: 68 · 2014: 54 · 2013: 2
- Type
- journal article
- Language
- en
- Open access
- bronze
- doi.org
- Licence
- Locations
- Cell stem cell (publishedVersion)
- PubMed (publishedVersion)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › CRISPR and Genetic Engineering
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Animal Genetics and Reproduction
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA regulation and disease
- Keywords
- CRISPR/Cas9, gene correction, cataract, dominant mutations, homology-directed repair, single guide RNA, zygote injection, off-target effects, germline transmission, mouse model
- MeSH terms
- Animals, Base Sequence, Cataract, Disease Models, Animal, Molecular Sequence Data, RNA, Messenger, Genetic Therapy, gamma-Crystallins, Mice, Clustered Regularly Interspaced Short Palindromic Repeats, CRISPR-Associated Proteins
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
