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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.
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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]
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The new frontier of genome engineering with CRISPR-Cas9
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“A dominant mutation in the Crygc gene responsible for cataracts was successfully corrected in mice (103).”
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“A previous report showed that CRISPR/Cas9-induced DSB in a mutated gene in mice could be corrected to wild-type through the HR mechanism, also termed homology-directed repair (HDR), with the endogenous wild-type allele as the template (Wu et al., 2013).”
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BackgroundCRISPR/Cas9 in Genome Editing and Beyond
Wang, H., Russa, M. F. L., & Qi, L. S. (2016) · Annual Review of Biochemistry · doi:10.1146/annurev-biochem-060815-014607 · Cited by 1,213
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BackgroundCRISPR/Cas9-mediated gene editing in human tripronuclear zygotes
Liang, P., Xu, Y., Zhang, X., Ding, C., Huang, R., Zhang, Z., Lv, J., Xie, X., Chen, Y., Li, Y., et al. (2015) · Protein & Cell · doi:10.1007/s13238-015-0153-5 · Cited by 1,135
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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
“147 – 149 These approaches reduce the risk of off-target genetic perturbations, as only the pre-selected and correctly edited cells can be implanted into patients.”
CRISPR-Based Technologies for the Manipulation of Eukaryotic Genomes
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Genome-wide binding of the CRISPR endonuclease Cas9 in mammalian cells
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“The simplicity of targeting any locus with a single protein and a programmable sgRNA has quickly led to widespread use of Cas9 11,12 in applications such as genome editing 7,8,13–16 , disease gene repair 17,18 and knock-in of specific tags 8,19 .”
Applications of CRISPR technologies in research and beyond
Barrangou, R., & Doudna, J. A. (2016) · Nature Biotechnology · doi:10.1038/nbt.3659 · Cited by 1,005
Correction of a pathogenic gene mutation in human embryos
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Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype
Yin, H., Xue, W., Chen, S., Bogorad, R. L., Benedetti, E., Grompe, M., Koteliansky, V., Sharp, P. A., Jacks, T., & Anderson, D. G. (2014) · Nature Biotechnology · doi:10.1038/nbt.2884 · Cited by 951
FGF/FGFR signaling in health and disease
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CRISPR-mediated direct mutation of cancer genes in the mouse liver
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Prevention of muscular dystrophy in mice by CRISPR/Cas9–mediated editing of germline DNA
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CRISPR/Cas9 therapeutics: progress and prospects
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“Fortunately, the chance of HDR occurring in mature cells is much lower than that of NHEJ.(68) Cas9 efficiently cleaves double-stranded DNA, but in practice, the sgRNA often mismatches with double-stranded DNA, leading to offtarget effects.”
BackgroundInfluentialGenome-editing Technologies for Gene and Cell Therapy
Maeder, M. L., & Gersbach, C. A. (2016) · Molecular Therapy · doi:10.1038/mt.2016.10 · Cited by 736
The CRISPR-Cas immune system: Biology, mechanisms and applications
Rath, D., Amlinger, L., Rath, A., & Lundgren, M. (2015) · Biochimie · doi:10.1016/j.biochi.2015.03.025 · Cited by 471
Wang, H.-X., Li, M., Lee, C. M., Chakraborty, S., Kim, H.-W., Bao, G., & Leong, K. W. (2017) · Chemical Reviews · doi:10.1021/acs.chemrev.6b00799 · Cited by 560
Expanding the genetic editing tool kit: ZFNs, TALENs, and CRISPR-Cas9
Gupta, R. M., & Musunuru, K. (2014) · Journal of Clinical Investigation · doi:10.1172/jci72992 · Cited by 577
Efficient Gene Disruption in Diverse Strains of Toxoplasma gondii Using CRISPR/CAS9
Shen, B., Brown, K. M., Lee, T. D., & Sibley, L. D. (2014) · mBio · doi:10.1128/mbio.01114-14 · Cited by 468
“CRISPR/CAS9 stimulated homologous recombination has been used to correct genetic disorders in a few disease models by replacing the mutated genes with wild-type copies through homology-directed repair (30, 31).”
MethodGenome-Editing Technologies: Principles and Applications
Gaj, T., Sirk, S. J., Shui, S.-L., & Liu, J. (2016) · Cold Spring Harbor Perspectives in Biology · doi:10.1101/cshperspect.a023754 · Cited by 381
“…the rapid creation of transgenic zebrafish (Doyon et al. 2008; Sander et al. 2011a; Hwang et al. 2013), mice (Cui et al. 2011; Wang et al. 2013; Wu et al. 2013), rats (Geurts et al. 2009; Tesson et al. 2011; Li et al. 2013), monkeys (Liu et al. 2014c), and livestock (Hauschild et al. 2011;…”
BackgroundXie, S., Shen, B., Zhang, C., Huang, X., & Zhang, Y. (2014) · PLoS ONE · doi:10.1371/journal.pone.0100448 · Cited by 426
“An interesting report shows that the CRISPRCas9 system can be used successfully to correct a genetic disease in mice with cataracts [23].”
BackgroundNon-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
Xu, Y., & Li, Z. (2020) · Computational and Structural Biotechnology Journal · doi:10.1016/j.csbj.2020.08.031 · Cited by 457
Xie, F., Ye, L., Chang, J. C., Beyer, A. I., Wang, J., Muench, M. O., & Kan, Y. W. (2014) · Genome Research · doi:10.1101/gr.173427.114 · Cited by 408
“1526 Genome Research www.genome.org 24:1526–1533 Published by Cold Spring Harbor Laboratory Press; ISSN 1088-9051/14; www.genome.org locus in cultured cells, plants, and animals (Cradick et al. 2013; Nekrasov et al. 2013;Wu et al. 2013; Yang et al. 2013).”
BackgroundRNA Drugs and RNA Targets for Small Molecules: Principles, Progress, and Challenges
Yu, A., Choi, Y. H., & Tu, M. (2020) · Pharmacological Reviews · doi:10.1124/pr.120.019554 · Cited by 421
“…et al., 2006; Ivics et al., 2014a,b; Ellis et al., 2017), CRISPR-associated proteins, or endo-nucleases (e.g., Cas9 and Cas12a) (Wang et al., 2013; Wu et al., 2013; Yin et al., 2016; Ren et al., 2017; Cromer et al., 2018; Xu et al., 2018; Gurumurthy et al., 2019) to enable genome editing or…”
BackgroundNaturally Occurring Off-Switches for CRISPR-Cas9
Pawluk, A., Amrani, N., Zhang, Y., García, B., Hidalgo-Reyes, Y., Lee, J., Edraki, A., Shah, M. K., Sontheimer, E. J., Maxwell, K. L., et al. (2016) · Cell · doi:10.1016/j.cell.2016.11.017 · Cited by 392
“…gene therapy to correct inherited disease, for sequence-specific targeting of pathogens to treat infectious disease, and many other applications (Bikard et al., 2014; Ebina et al., 2013; Gomaa et al., 2014; Kaminski et al., 2016; Ousterout et al., 2015; Wu et al., 2013; Yin et al., 2014).”
Target specificity of the CRISPR‐Cas9 system
Wu, X., Kriz, A. J., & Sharp, P. A. (2014) · Quantitative Biology · doi:10.1007/s40484-014-0030-x · Cited by 388
“With exogenous template oligos, specific sequences such as HA-tag or GFP could be inserted into genes to label proteins [19,20], or to correct mutations in disease genes in human and mouse [21–23].”
ssODN-mediated knock-in with CRISPR-Cas for large genomic regions in zygotes
Yoshimi, K., Kunihiro, Y., Kaneko, T., Nagahora, H., Voigt, B., & Mashimo, T. (2016) · Nature Communications · doi:10.1038/ncomms10431 · Cited by 363
Canver, M. C., Bauer, D. E., Dass, A., Yien, Y. Y., Chung, J., Masuda, T., Maeda, T., Paw, B. H., & Orkin, S. H. (2014) · Journal of Biological Chemistry · doi:10.1074/jbc.m114.564625 · Cited by 357
In vivo CRISPR editing with no detectable genome-wide off-target mutations
Akçakaya, P., Bobbin, M. L., Guo, J. A., Malagon-Lopez, J., Clement, K., Garcia, S. P., Fellows, M. D., Porritt, M. J., Firth, M. A., Carreras, A., et al. (2018) · Nature · doi:10.1038/s41586-018-0500-9 · Cited by 324
A Mouse Geneticist’s Practical Guide to CRISPR Applications
Singh, P., Schimenti, J. C., & Bolcun‐Filas, E. (2014) · Genetics · doi:10.1534/genetics.114.169771 · Cited by 312
“causing mutations and reverse the phenotypes (Wu et al. 2013; Yoshimi et al. 2014), but the biggest obstacle for therapeutic use of CRISPRs in humans would be the delivery of editing reagents for efficient allele correction in vivo or in stem cells that can be re-introduced into people.”
BackgroundDistinct Processing of lncRNAs Contributes to Non-conserved Functions in Stem Cells
Guo, C., Ma, X.-K., Xing, Y., Zheng, C.-C., Xu, Y.-F., Shan, L., Zhang, J., Wang, S., Wang, Y., Carmichael, G., et al. (2020) · Cell · doi:10.1016/j.cell.2020.03.006 · Cited by 315
Cloning-free CRISPR/Cas system facilitates functional cassette knock-in in mice
Aida, T., Chiyo, K., Usami, T., Ishikubo, H., Imahashi, R., Wada, Y., Tanaka, K. F., Sakuma, T., Yamamoto, T., & Tanaka, K. (2015) · Genome biology · doi:10.1186/s13059-015-0653-x · Cited by 298
“In addition to the generation of knock-in mice carrying complex gene cassettes, our method can be directly applied to the generation of knockout mice [23] and knock-in mice carrying single nucleotide substitutions with oligo DNA donors [5,7,8], as well as to other species [39-41] and cultured cells [22,28], and in viv…”
BackgroundMethodEuPaGDT: a web tool tailored to design CRISPR guide RNAs for eukaryotic pathogens
Peng, D., & Tarleton, R. L. (2015) · Microbial Genomics · doi:10.1099/mgen.0.000033 · Cited by 293
“In addition to relying on MMEJ-induced deletions to mutate specific sequences, single-stranded oligonucleotides (ssODNs) bearing homology arms bracketing gRNA-guided DSBs can be used to introduce modifications at specific target positions by homology-directed repair (HDR; Wang et al., 2013; Wu et al., 2013; Yang et al…”
MethodTALEN and CRISPR/Cas Genome Editing Systems: Tools of Discovery
Nemudryi, A. A., Valetdinova, K. R., Medvedev, S. P., & Zakian, S. M. (2014) · Acta Naturae · doi:10.32607/20758251-2014-6-3-19-40 · Cited by 287
Machine Learning in Genomic Medicine: A Review of Computational Problems and Data Sets
Leung, M. K. K., Delong, A., Alipanahi, B., & Frey, B. J. (2015) · Proceedings of the IEEE · doi:10.1109/jproc.2015.2494198 · Cited by 284
“The system has been used to control gene expression by deactivating the endonuclease domain [201], study gene functions in neural cells [202], develop synthetic biology applications [203], fix pathogenic mutations causing -thalassemia [204], and to repair cystic fibrosis transmembrane conductor receptor mutations [205…”
MethodGenome modification by CRISPR /Cas9
Ma, Y., Zhang, L., & Huang, X. (2014) · FEBS Journal · doi:10.1111/febs.13110 · Cited by 304
Kang, X., He, W., Huang, Y., Qian, Y., Chen, Y., Gao, X., Sun, X., & Fan, Y. (2016) · Journal of Assisted Reproduction and Genetics · doi:10.1007/s10815-016-0710-8 · Cited by 271
“causing mutations, leading to the production of healthy adult animals [8, 9].”
BackgroundAdvances in therapeutic CRISPR/Cas9 genome editing
Savić, N., & Schwank, G. (2016) · Translational Research · doi:10.1016/j.trsl.2015.09.008 · Cited by 257
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
“For a proof of concept of potential therapeutic applications, such method can be applied to correct genetic mutations in genes in mouse zygotes as well as spermatogonial stem cells, leading to bear healthy animals.(157,158) Pigs bearing biallelic knockout have been generated in one step by an injection of Cas9 mRNA an…”
MethodWu, Y., Zhou, H., Fan, X., Zhang, Y., Zhang, M., Wang, Y., Xie, Z., Bai, M., Yin, Q., Liang, D., et al. (2015) · Cell Research · doi:10.1038/cr.2014.160 · Cited by 247
Artificial Virus Delivers CRISPR-Cas9 System for Genome Editing of Cells in Mice
Li, L., Song, L., Liu, X., Yang, X., Li, X., He, T., Wang, N., Yang, S., Yu, C., Yin, T., et al. (2017) · ACS Nano · doi:10.1021/acsnano.6b04261 · Cited by 244
CRISPR-mediated Genome Editing Restores Dystrophin Expression and Function in mdx Mice
Xu, L., Park, K. H., Zhao, L., Xu, J., El Refaey, M., Gao, Y., Zhu, H., Ma, J., & Han, R. (2016) · Molecular Therapy · doi:10.1038/mt.2015.192 · Cited by 244
The rapidly evolving view of lysosomal storage diseases
Parenti, G., Medina, D. L., & Ballabio, A. (2021) · EMBO Molecular Medicine · doi:10.15252/emmm.202012836 · Cited by 230
“Additionally, CRISPR/Cas9 can be by itself an alternative therapeutic option for treating LSDs by correcting disease-causing mutations, both in vitro and in vivo (Schwank et al, 2013; Wu et al, 2013; Xie et al, 2014).”
BackgroundCRISPR-Cpf1 correction of muscular dystrophy mutations in human cardiomyocytes and mice
Zhang, Y., Long, C., Li, H. Z., McAnally, J., Baskin, K. K., Shelton, J. M., Bassel‐Duby, R., & Olson, E. N. (2017) · Science Advances · doi:10.1126/sciadv.1602814 · Cited by 228
CRISPR-Cas9 delivery to hard-to-transfect cells via membrane deformation
Han, X., Liu, Z., Jo, M. C., Zhang, K., Li, Y., Zeng, Z., Li, N., Zu, Y., & Qin, L. (2015) · Science Advances · doi:10.1126/sciadv.1500454 · Cited by 235
A CRISPR view of developmentPreprint
Harrison, M. M., Jenkins, B., O’Connor-Giles, K. M., & Wildonger, J. (2014) · Genes & Development · doi:10.1101/gad.248252.114 · Cited by 234
“RGNs were used in mouse embryos to correct a dominant mutation in the Crygc gene, which causes cataracts, and those mice with the corrected locus were free of cataracts (Wu et al. 2013).”
MethodMacrophage-Specific in Vivo Gene Editing Using Cationic Lipid-Assisted Polymeric Nanoparticles
Luo, Y., Xu, C., Li, H., Cao, Z., Liu, J., Wang, J., Du, X., Yang, X., Gu, Z., & Wang, J. (2018) · ACS Nano · doi:10.1021/acsnano.7b07874 · Cited by 223
CRISPR-based rapid and ultra-sensitive diagnostic test for Mycobacterium tuberculosis
Ai, J., Zhou, X., Xu, T., Yang, M., Chen, Y., He, G., Pan, N., Cai, Y., Li, Y., Wang, X., et al. (2019) · Emerging Microbes & Infections · doi:10.1080/22221751.2019.1664939 · Cited by 224
“Cas9 was the first member of the Cas family which had been used for gene editing since 2013 [7,8].”
MethodA single-embryo, single-cell time-resolved model for mouse gastrulation
Mittnenzweig, M., Mayshar, Y., Cheng, S., Ben-Yair, R., Hadas, R., Rais, Y., Chomsky, E., Reines, N., Uzonyi, A., Lumerman, L., et al. (2021) · Cell · doi:10.1016/j.cell.2021.04.004 · Cited by 214
CRISPR‐Cas Biochemistry and CRISPR‐Based Molecular Diagnostics
Weng, Z., You, Z., Yang, J., Mohammad, N., Lin, M., Wei, Q., Gao, X., & Zhang, Y. (2023) · Angewandte Chemie International Edition · doi:10.1002/anie.202214987 · Cited by 213
CRISPR-Cas: New Tools for Genetic Manipulations from Bacterial Immunity Systems
Jiang, W., & Marraffini, L. A. (2015) · Annual Review of Microbiology · doi:10.1146/annurev-micro-091014-104441 · Cited by 219
CRISPR-Cas9–based treatment of myocilin-associated glaucoma
Jain, A., Zode, G. S., Kasetti, R. B., Ran, F. A., Yan, W. X., Sharma, T. P., Bugge, K., Searby, C. C., Fingert, J. H., Zhang, F., et al. (2017) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1706193114 · Cited by 205
“Previously reported in vivo uses of CRISPR-Cas9 include correction of a dominant loss-of-function cataract-causing mutation in mouse embryos (39), HDR in a mouse model of type I tyrosinemina (40), correction of retinal dystrophy in a rat model of autosomal dominant retinitis pigmentosa (41), NHEJ-mediated gene disrupt…”
BackgroundCRISPR-Cas9: a new and promising player in gene therapy
Xiao-Jie, L., Hui-Ying, X., Zun-Ping, K., Jin-Lian, C., & Li-Juan, J. (2015) · Journal of Medical Genetics · doi:10.1136/jmedgenet-2014-102968 · Cited by 200
Comparison of non-canonical PAMs for CRISPR/Cas9-mediated DNA cleavage in human cells
Zhang, Y., Ge, X., Yang, F., Zhang, L., Zheng, J., Tan, X., Jin, Z., Qu, J., & Gu, F. (2014) · Scientific Reports · doi:10.1038/srep05405 · Cited by 205
CRISPR-Cas Tools and Their Application in Genetic Engineering of Human Stem Cells and Organoids
Hendriks, D. F. G., Clevers, H. C., & Artegiani, B. (2020) · Cell stem cell · doi:10.1016/j.stem.2020.10.014 · Cited by 202
A review of emerging physical transfection methods for CRISPR/Cas9-mediated gene editing
Fajrial, A. K., He, Q. Q., Wirusanti, N. I., Slansky, J. E., & Ding, X. (2020) · Theranostics · doi:10.7150/thno.43465 · Cited by 185
“For example, a mutation on the Crygc gene caused cataract in mice [144].”
BackgroundCRISPR/Cas9-mediated targeted chromosome elimination
Zuo, E., Huo, X., Yao, X., Hu, X., Sun, Y., Yin, J., He, B., Wang, X., Shi, L., Ping, J., et al. (2017) · Genome biology · doi:10.1186/s13059-017-1354-4 · Cited by 194
Off-target predictions in CRISPR-Cas9 gene editing using deep learning
Lin, J., & Wong, K.-C. (2018) · Bioinformatics · doi:10.1093/bioinformatics/bty554 · Cited by 185
Development of CRISPR-Cas systems for genome editing and beyond
Zhang, F. (2019) · Quarterly Reviews of Biophysics · doi:10.1017/s0033583519000052 · Cited by 188
“Before the year’s end, the first reports were published on the use of Cas9 to correct a cataractcausing mutation in a mouse, leading to reversal of the disease phenotype (Wu et al., 2013).”
BackgroundYoshimi, K., Kaneko, T., Voigt, B., & Mashimo, T. (2014) · Nature Communications · doi:10.1038/ncomms5240 · Cited by 178
“In mice, the high efficiency of HDR-mediated KIs has also been reported with ssODN donors 26,27,33 , compared with those of KIs with dsDNA donor vectors 39 .”
Guan, Y., Ma, Y., Li, Q., Sun, Z., Ma, L., Wu, L., Wang, L., Zeng, L., Shao, Y., Chen, Y., et al. (2016) · EMBO Molecular Medicine · doi:10.15252/emmm.201506039 · Cited by 179
“…have shown potential applications of the CRISPR/Cas9 system for correction of genetic disorders in cells or mouse embryos (Schwank et al, 2013; Wu et al, 2013; Long et al, 2014), as well as prevention of cardiovascular disease in adult mice through disruption of the PCSK9 gene (Ding et al,…”
BackgroundRecent Progress in CRISPR/Cas9 Technology
Mei, Y., Wang, Y., Chen, H., Sun, Z. S., & Ju, X.-D. (2016) · Journal of Genetics and Genomics · doi:10.1016/j.jgg.2016.01.001 · Cited by 148
“…Cas9 RNA/DSB Rosa26 7/7 Cytoplasm Cas9 RNA/DSB Fujita and Fujii, 2013 Hprt 17/18 Cytoplasm Cas9 RNA/DSB Crygc 13/23 Cytoplasm Cas9 px330/DSB Wu et al., 2013 Cetn1 10/17 Pronucleus Cas9 px330/DSB Mashiko et al., 2014 Cetn1 5/20 Cytoplasm Cas9 RNA/DSB Prm1 2/3 Pronucleus Cas9 px330/DSB Prm1…”
Efficient and Allele-Specific Genome Editing of Disease Loci in Human iPSCs
Smith, C. J., Abalde-Atristain, L., He, C., Brodsky, B. R., Braunstein, E. M., Chaudhari, P., Jang, Y.-Y., Cheng, L., & Ye, Z. (2014) · Molecular Therapy · doi:10.1038/mt.2014.226 · Cited by 180
Advances in CRISPR-Cas9 genome engineering: lessons learned from RNA interference
Barrangou, R., Birmingham, A., Wiemann, S., Beijersbergen, R. L., Hornung, V., & Smith, A. V. B. (2015) · Nucleic Acids Research · doi:10.1093/nar/gkv226 · Cited by 173
“Cataracts Rescue of a dominant mutation in the Crygc gene that causes cataracts (108).”
BackgroundGenome Editing in Induced Pluripotent Stem Cells using CRISPR/Cas9
Jehuda, R. B., Shemer, Y., & Binah, O. (2018) · Stem Cell Reviews and Reports · doi:10.1007/s12015-018-9811-3 · Cited by 171
Lipid nanoparticles for short interfering RNA delivery
Leung, A. K. K., Tam, Y. Y. C., & Cullis, P. R. (2014) · Advances in genetics · doi:10.1016/b978-0-12-800148-6.00004-3 · Cited by 158
“Cataract-free mice have been generated by injecting Cas9 mRNA and gRNA targeting the mutated Crygc gene locus into mouse zygotes followed by implantation in surrogate female mice (Wu et al., 2013).”
Generation of Genomic Deletions in Mammalian Cell Lines via CRISPR/Cas9
Bauer, D. E., Canver, M. C., & Orkin, S. H. (2014) · Journal of Visualized Experiments · doi:10.3791/52118 · Cited by 176
A general strategy to construct small molecule biosensors in eukaryotes
Feng, J., Jester, B. W., Tinberg, C. E., Mandell, D. J., Antunes, M. S., Chari, R., Morey, K. J., Rios, X., Medford, J. I., Church, G. M., et al. (2015) · eLife · doi:10.7554/elife.10606 · Cited by 158
“This switch-like control over CRISPR/Cas9 activity could reduce background activity and off-target editing, a critical feature for safer gene therapies (Mandal, et al., 2014; Wu et al., 2013; Schwank et al., 2013).”
Oligonucleotide‐directed mutagenesis for precision gene editing
Sauer, N. J., Mozoruk, J., Miller, R. B., Warburg, Z. J., Walker, K. A. M., Beetham, P. R., Schöpke, C. R., & Gocal, G. F. W. (2015) · Plant Biotechnology Journal · doi:10.1111/pbi.12496 · Cited by 155
“…editing in combination with oligonucleotides, primarily in eukaryotic systems (Chen et al., 2011; Connelly et al., 2010; DiCarlo et al., 2013; Gratz et al., 2013; Strouse et al., 2014; Suzuki et al., 2003; Svitashev et al., 2015; Voytas, 2013; Wang et al., 2015; Wu et al., 2013; Zhao et al., 2014).”
BackgroundGenome editing using CRISPR/Cas9-based knock-in approaches in zebrafish
Albadri, S., Bene, F. D., & Revenu, C. (2017) · Methods · doi:10.1016/j.ymeth.2017.03.005 · Cited by 152
Araki, M., & Ishii, T. (2014) · Reproductive Biology and Endocrinology · doi:10.1186/1477-7827-12-108 · Cited by 150
“Genome editing technology is more likely to develop into medicine for preventing a genetic disease if corrective genome editing is integrated into assisted reproductive technology (ART), including IVF and ICSI.”
BackgroundInfluentialGraphene oxide-mediated Cas9/sgRNA delivery for efficient genome editing
Yue, H., Zhou, X., Cheng, M., & Xing, D. (2018) · Nanoscale · doi:10.1039/c7nr07999k · Cited by 150
CRISPR/Cas9 technology as a potent molecular tool for gene therapy
Karimian, A., Azizian, K., Parsian, H., Rafieyan, S., Shafiei‐Irannejad, V., Kheyrollah, M., Yousefi, M., Majidinia, M., & Yousefi, B. (2019) · Journal of Cellular Physiology · doi:10.1002/jcp.27972 · Cited by 148
Complementary Alu sequences mediate enhancer–promoter selectivity
Liang, L., Cao, C., Ji, L., Cai, Z., Wang, D., Ye, R., Chen, J., Yu, X., Zhou, J., Bai, Z., et al. (2023) · Nature · doi:10.1038/s41586-023-06323-x · Cited by 147
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
CRISPR-Based Diagnosis of Infectious and Noninfectious Diseases
vangah, S. J., Katalani, C., Boone, H. A., Hajizade, A., Sijerčić, A., & Ahmadian, G. (2020) · Biological Procedures Online · doi:10.1186/s12575-020-00135-3 · Cited by 142
“cluding mice [86], Drosophila [8], Caenorhabditis elegans [21], humans [15, 17, 39, 69], zebrafish [32],”
BackgroundDelivery and therapeutic applications of gene editing technologies ZFNs, TALENs, and CRISPR/Cas9
LaFountaine, J. S., Fathe, K., & Smyth, H. D. C. (2015) · International Journal of Pharmaceutics · doi:10.1016/j.ijpharm.2015.08.029 · Cited by 111
Weninger, A., Fischer, J. E., Raschmanová, H., Kniely, C., Vogl, T., & Glieder, A. (2017) · Journal of Cellular Biochemistry · doi:10.1002/jcb.26474 · Cited by 133
Responsive Nanocarriers as an Emerging Platform for Cascaded Delivery of Nucleic Acids to Cancer
Liu, Y., Xu, C., Iqbal, S., Yang, X., & Wang, J. (2017) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2017.03.004 · Cited by 130
“tivemutations and the disruption of viral DNA inmany diseases, such as tumor [48], cataracts [49], cystic fibrosis [50], hereditary tyrosinemia [51,52], human immunodeficiency virus [53] et al.”
Loureiro, A., & da Silva, G. J. (2019) · Antibiotics · doi:10.3390/antibiotics8010018 · Cited by 129
“Due to the low rate of HDR recombination [109], and because it is only readily available in dividing cells [110,111], this method needs to become more robust and flexible in order to see use in disease therapy.”
CRISPR/Cas9‐mediated genome editing: From basic research to translational medicine
Jacinto, F. V., Link, W., & Ferreira, B. I. (2020) · Journal of Cellular and Molecular Medicine · doi:10.1111/jcmm.14916 · Cited by 135
Zhong, C., Yin, Q., Xie, Z., Bai, M., Dong, R., Tang, W., Xing, Y., Zhang, H., Yang, S., Chen, L., et al. (2015) · Cell stem cell · doi:10.1016/j.stem.2015.06.005 · Cited by 118
“To test this, we used CRISPRCas9 technology (Wu et al., 2013).”
MethodCourtney, D., Moore, J. E., Atkinson, S. D., Maurizi, E., Allen, E. H. A., Pedrioli, D. M. L., McLean, W. H. I., Nesbit, M. A., & Moore, T. (2015) · Gene Therapy · doi:10.1038/gt.2015.82 · Cited by 124
Genome editing with CRISPR/Cas9 in postnatal mice corrects PRKAG2 cardiac syndrome
Xie, C., Zhang, Y., Song, L., Luo, J., Qi, W., Hu, J., Lu, D., Yang, Z. H., Zhang, J., Xiao, J., et al. (2016) · Cell Research · doi:10.1038/cr.2016.101 · Cited by 132
Protospacer adjacent motif (PAM)-distal sequences engage CRISPR Cas9 DNA target cleavage
Cencic, R., Miura, H., Malina, A., Robert, F., Ethier, S., Schmeing, T. M., Dostie, J., & Pelletier, J. (2014) · PLoS ONE · doi:10.1371/journal.pone.0109213 · Cited by 117
In utero gene editing for monogenic lung disease
Alapati, D., Zacharias, W. J., Hartman, H. A., Rossidis, A. C., Stratigis, J. D., Ahn, N. J., Coons, B., Zhou, S., Li, H., Singh, K., et al. (2019) · Science Translational Medicine · doi:10.1126/scitranslmed.aav8375 · Cited by 123
Temporal regulation of prenatal embryonic development by paternal imprinted loci
Li, Q., Li, Y., Yin, Q., Huang, S., Wang, K., Zhuo, L., Li, W., Chang, B., & Li, J. (2019) · Science China Life Sciences · doi:10.1007/s11427-019-9817-6 · Cited by 120
Ma, M., Zhuang, F., Hu, X., Wang, B., Wen, X., Ji, J., & Xi, J. (2017) · Cell Research · doi:10.1038/cr.2017.29 · Cited by 116
CUT-PCR: CRISPR-mediated, ultrasensitive detection of target DNA using PCR
Lee, S. H., Yu, J., Hwang, G.-H., Kim, S., Kim, H. S., Ye, S., Kim, K., Park, J., Park, D. Y., Cho, Y.-K., et al. (2017) · Oncogene · doi:10.1038/onc.2017.281 · Cited by 111
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
PE-Designer and PE-Analyzer: web-based design and analysis tools for CRISPR prime editing
Hwang, G., Jeong, Y. K., Habib, O., Hong, S.-A., Lim, K., Kim, J., & Bae, S. (2021) · Nucleic Acids Research · doi:10.1093/nar/gkab319 · Cited by 122
Slijkerman, R. W. N., Song, F., Astuti, G., Huynen, M. A., van Wijk, E., Stieger, K., & Collin, R. W. J. (2015) · Progress in Retinal and Eye Research · doi:10.1016/j.preteyeres.2015.04.004 · Cited by 123
“First tests in a mouse model for cataracts showed successful repair of the mutated Crygc gene by using its healthy counterpart as a template, which led to fertile wildtype mice (Wu et al., 2013).”
BackgroundCRISPR/Cas9 Genome-Editing System in Human Stem Cells: Current Status and Future Prospects
Zhang, Z., Zhang, Y., Gao, F., Han, S., Cheah, K. S. E., Tse, H., & Lian, Q. (2017) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2017.09.009 · Cited by 122
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
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