Latest Developed Strategies to Minimize the Off-Target Effects in CRISPR-Cas-Mediated Genome Editing

Ultrafast and memory-efficient alignment of short DNA sequences to the human genome.

Langmead, B., Trapnell, C., Pop, M., & Salzberg, S. L. (2009) · Genome Biology · doi:10.1186/gb-2009-10-3-r25 · Cited by 21,849

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

Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage

Komor, A. C., Kim, Y. B., Packer, M. S., Zuris, J. A., & Liu, D. R. (2016) · Nature · doi:10.1038/nature17946 · Cited by 5,437

Aligning sequence reads, clone sequences and assembly con*gs with BWA-MEMPreprint

Li, H. (2014) · arXiv (Cornell University) · doi:10.6084/m9.figshare.963153.v1 · Cited by 5,155

Repurposing CRISPR as an RNA-Guided Platform for Sequence-Specific Control of Gene Expression

Qi, L. S., Larson, M. H., Gilbert, L. A., Doudna, J. A., Weissman, J. S., Arkin, A. P., & Lim, W. A. (2013) · Cell · doi:10.1016/j.cell.2013.02.022 · Cited by 5,016

Search-and-replace genome editing without double-strand breaks or donor DNA

Anzalone, A. V., Randolph, P. B., Davis, J. R., Sousa, A. A., Koblan, L. W., Levy, J. M., Chen, P. J., Wilson, C., Newby, G. A., Raguram, A., et al. (2019) · Nature · doi:10.1038/s41586-019-1711-4 · Cited by 4,767

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

Optimized sgRNA design to maximize activity and minimize off-target effects of CRISPR-Cas9

Doench, J. G., Fusi, N., Sullender, M., Hegde, M., Vaimberg, E. W., Donovan, K. F., Smith, I., Tothova, Z., Wilen, C., Orchard, R., et al. (2016) · Nature Biotechnology · doi:10.1038/nbt.3437 · Cited by 4,637

Off-target effects in CRISPR/Cas9 gene editing

Guo, C., Ma, X., Gao, F., & Guo, Y. (2023) · Frontiers in Bioengineering and Biotechnology · doi:10.3389/fbioe.2023.1143157 · Cited by 704

Mechanism and Applications of CRISPR/Cas-9-Mediated Genome Editing

Mengstie, M. A., & Wondimu, B. Z. (2021) · Biologics: Targets and Therapy · doi:10.2147/btt.s326422 · Cited by 478

Deletion and replacement of long genomic sequences using prime editing

Jiang, T., Zhang, X.-O., Weng, Z., & Xue, W. (2021) · Nature Biotechnology · doi:10.1038/s41587-021-01026-y · Cited by 207

Comprehensive Overview of Bottom-Up Proteomics Using Mass Spectrometry

Jiang, Y., Balaya, R. D. A., Schuster, D., Neely, B. A., Rosano, G. L., Volkmar, N., Momenzadeh, A., Peters-Clarke, T. M., Egbert, S., Kreimer, S. D., et al. (2024) · ACS Measurement Science Au · doi:10.1021/acsmeasuresciau.3c00068 · Cited by 217

Stimuli-Responsive Gene Delivery Nanocarriers for Cancer Therapy

Zhang, Q., Kuang, G., Li, W., Wang, J., Ren, H., & Zhao, Y. (2023) · Nano-Micro Letters · doi:10.1007/s40820-023-01018-4 · Cited by 191

In vivo delivery of CRISPR-Cas9 using lipid nanoparticles enables antithrombin gene editing for sustainable hemophilia A and B therapy

Han, J. P., Kim, M., Choi, B. S., Lee, J. H., Lee, G. S., Jeong, M., Lee, Y., Kim, E.-A., Oh, H.-K., Go, N., et al. (2022) · Science Advances · doi:10.1126/sciadv.abj6901 · Cited by 193

Advances in CRISPR therapeutics

Chavez, M., Chen, X., Finn, P. B., & Qi, L. S. (2022) · Nature Reviews Nephrology · doi:10.1038/s41581-022-00636-2 · Cited by 184

A Critical Review: Recent Advancements in the Use of CRISPR/Cas9 Technology to Enhance Crops and Alleviate Global Food Crises

Rasheed, A., Gill, R. A., Hassan, M., Mahmood, A., Qari, S. H., Zaman, Q. U., Ilyas, M., Aamer, M., Batool, M., Li, H., et al. (2021) · Current Issues in Molecular Biology · doi:10.3390/cimb43030135 · Cited by 154

An Outlook on Global Regulatory Landscape for Genome-Edited Crops

Ahmad, A., Munawar, N., Khan, Z., Qumsani, A. T., Khan, S. H., Jamil, A., Ashraf, S., Ghouri, M. Z., Aslam, S., Mubarik, M. S., et al. (2021) · International Journal of Molecular Sciences · doi:10.3390/ijms222111753 · Cited by 140

Writers, readers, and erasers RNA modifications and drug resistance in cancer

Chen, D., Gu, X., Nurzat, Y., Xu, L., Li, X., Wu, L., Jiao, H., Gao, P., Zhu, X., Yan, D., et al. (2024) · Molecular Cancer · doi:10.1186/s12943-024-02089-6 · Cited by 127

Multiplex genome-editing technologies for revolutionizing plant biology and crop improvement

Abdelrahman, M., Wei, Z., Rohila, J. S., & Zhao, K. (2021) · Frontiers in Plant Science · doi:10.3389/fpls.2021.721203 · Cited by 125

Massively parallel base editing to map variant effects in human hematopoiesis

Martin-Rufino, J. D., Castano, N., Pang, M., Grody, E. I., Joubran, S. R., Caulier, A., Wahlster, L., Li, T., Qiu, X., Riera-Escandell, A. M., et al. (2023) · Cell · doi:10.1016/j.cell.2023.03.035 · Cited by 130

Mechanistic study of cancer drug delivery: Current techniques, limitations, and future prospects

Imtiaz, S., Ferdous, U. T., Nizela, A., Hasan, A., Shakoor, A., Zia, A. W., & Uddin, S. (2025) · European Journal of Medicinal Chemistry · doi:10.1016/j.ejmech.2025.117535 · Cited by 119

CRISPR-Based Crop Improvements: A Way Forward to Achieve Zero Hunger

Ahmad, S., Tang, L., Shahzad, R., Mawia, A. M., Rao, G. S., Jamil, S., Chen, W., Sheng, Z., Shao, G., Wei, X., et al. (2021) · Journal of Agricultural and Food Chemistry · doi:10.1021/acs.jafc.1c02653 · Cited by 125

Current State of Human Gene Therapy: Approved Products and Vectors

Shchaslyvyi, A. Y., Antonenko, S. V., Tesliuk, M. G., & Телегеев, Г. Д. (2023) · Pharmaceuticals · doi:10.3390/ph16101416 · Cited by 127

Using traditional machine learning and deep learning methods for on- and off-target prediction in CRISPR/Cas9: a review

Sherkatghanad, Z., Abdar, M., Charlier, J., & Makarenkov, V. (2023) · Briefings in Bioinformatics · doi:10.1093/bib/bbad131 · Cited by 123

Integration of CRISPR/Cas9 with artificial intelligence for improved cancer therapeutics

Bhat, A. A., Nisar, S., Mukherjee, S., Saha, N., Yarravarapu, N., Lone, S. N., Masoodi, T., Chauhan, R., Maacha, S., Bagga, P., et al. (2022) · Journal of Translational Medicine · doi:10.1186/s12967-022-03765-1 · Cited by 114

Off-target effects in CRISPR-Cas genome editing for human therapeutics: Progress and challenges

Kalter, N., Fuster‐García, C., Silva, A., Ronco-Díaz, V., Roncelli, S., Turchiano, G., Gorodkin, J., Cathomen, T., Benabdellah, K., Lee, C. M., et al. (2025) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2025.102636 · Cited by 121

CRISPR-based gene editing technology and its application in microbial engineering

Wei, J., & Li, Y. (2023) · Engineering Microbiology · doi:10.1016/j.engmic.2023.100101 · Cited by 106

A Review on Advanced CRISPR-Based Genome-Editing Tools: Base Editing and Prime Editing

Sichani, A. S., Ranjbar, M., Baneshi, M., Zadeh, F. T., & Fallahi, J. (2022) · Molecular Biotechnology · doi:10.1007/s12033-022-00639-1 · Cited by 105

Gene therapy for CNS disorders: modalities, delivery and translational challenges

Gao, J., Gunasekar, S., Xia, Z. J., Shalin, K., Jiang, C., Chen, H., Lee, D., Lee, S., Pisal, N. D., Luo, J. N., et al. (2024) · Nature reviews. Neuroscience · doi:10.1038/s41583-024-00829-7 · Cited by 100

Gold nanorods etching as a powerful signaling process for plasmonic multicolorimetric chemo-/biosensors: Strategies and applications

Kermanshahian, K., Yadegar, A., & Ghourchian, H. (2021) · Coordination Chemistry Reviews · doi:10.1016/j.ccr.2021.213934 · Cited by 95

Recent advancements in reducing the off-target effect of CRISPR/Cas9 genome editing

Asmamaw Mengstie, M., Teshome Azezew, M., Asmamaw Dejenie, T., Teshome, A. A., Tadele Admasu, F., Behaile Teklemariam, A., Tilahun Mulu, A., Mekonnen Agidew, M., Adugna, D. G., Geremew, H., et al. (2024) · Biologics: Targets and Therapy · doi:10.2147/btt.s429411 · Cited by 91

Biomaterials-mediated CRISPR/Cas9 delivery: recent challenges and opportunities in gene therapy

Dubey, A. K., & Mostafavi, E. (2023) · Frontiers in Chemistry · doi:10.3389/fchem.2023.1259435 · Cited by 96

Recent advances in the delivery and applications of nonviral CRISPR/Cas9 gene editing

Sinclair, F., Begum, A. A., Dai, C. C., Tóth, I., & Moyle, P. M. (2023) · Drug Delivery and Translational Research · doi:10.1007/s13346-023-01320-z · Cited by 91

Applications and Potential of Genome-Editing Systems in Rice Improvement: Current and Future Perspectives

Tabassum, J., Ahmad, S., Hussain, B., Mawia, A. M., Zeb, A., & Ju, L. (2021) · Agronomy · doi:10.3390/agronomy11071359 · Cited by 88

Assessment of Biofortification Approaches Used to Improve Micronutrient-Dense Plants That Are a Sustainable Solution to Combat Hidden Hunger

Koç, E., & Karayiğit, B. (2021) · Journal of soil science and plant nutrition · doi:10.1007/s42729-021-00663-1 · Cited by 86

Engineering approaches for RNA-based and cell-based osteoarthritis therapies

DeJulius, C. R., Walton, B. L., Colazo, J. M., d’Arcy, R., Francini, N., Brunger, J. M., & Duvall, C. L. (2024) · Nature Reviews Rheumatology · doi:10.1038/s41584-023-01067-4 · Cited by 85

CRISPR Variants for Gene Editing in Plants: Biosafety Risks and Future Directions

Movahedi, A., Dargiri, S. A., Li, H., Zhuge, Q., & Sun, W. (2023) · International Journal of Molecular Sciences · doi:10.3390/ijms242216241 · Cited by 83

Current and future concepts for the generation and application of genetically engineered CAR-T and TCR-T cells

Hiltensperger, M., & Krackhardt, A. M. (2023) · Frontiers in Immunology · doi:10.3389/fimmu.2023.1121030 · Cited by 79

Recent Advances in the Application of CRISPR/Cas9 Gene Editing System in Poultry Species

Khwatenge, C. N., & Nahashon, S. N. (2021) · Frontiers in Genetics · doi:10.3389/fgene.2021.627714 · Cited by 76

Applicability of the EFSA Opinion on site‐directed nucleases type 3 for the safety assessment of plants developed using site‐directed nucleases type 1 and 2 and oligonucleotide‐directed mutagenesis

Panel), E. P. O. G. M. O. (. G., Naegeli, H., Bresson, J., Dalmay, T., Dewhurst, I. C., Epstein, M. M., Firbank, L. G., Guerche, P., Hejátko, J., Moreno, F. J., et al. (2020) · EFSA Journal · doi:10.2903/j.efsa.2020.6299 · Cited by 77

Current Bioinformatics Tools to Optimize CRISPR/Cas9 Experiments to Reduce Off-Target Effects

Naeem, M., & Alkhnbashi, O. S. (2023) · International Journal of Molecular Sciences · doi:10.3390/ijms24076261 · Cited by 70

Plant Viruses: From Targets to Tools for CRISPR

Varanda, C. M. R., Félix, M. D. R., Campos, M. D., Patanita, M., & Materatski, P. (2021) · Viruses · doi:10.3390/v13010141 · Cited by 70

Recent Advances in Improving Gene-Editing Specificity through CRISPR–Cas9 Nuclease Engineering

Huang, X., Yang, D., Zhang, J., Xu, J., & Chen, Y. E. (2022) · Cells · doi:10.3390/cells11142186 · Cited by 68

Evolution of CRISPR/Cas Systems for Precise Genome Editing

Hryhorowicz, M., Lipiński, D., & Zeyland, J. (2023) · International Journal of Molecular Sciences · doi:10.3390/ijms241814233 · Cited by 69

Stimuli-responsive nanoformulations for CRISPR-Cas9 genome editing

Fang, T., Cao, X., Ibnat, M., & Chen, G. (2022) · Journal of Nanobiotechnology · doi:10.1186/s12951-022-01570-y · Cited by 63

CRISPR/Cas12a-based biosensors for ultrasensitive tobramycin detection with single- and double-stranded DNA activators

Li, D., Ling, S., Wu, H., Yang, Z., & Lv, B. (2021) · Sensors and Actuators B Chemical · doi:10.1016/j.snb.2021.131329 · Cited by 59

CRISPR-Cas9-Mediated Gene Therapy in Neurological Disorders

Guan, L., Han, Y., Yang, C., Lu, S., Du, J., Li, H., & Lin, J. (2021) · Molecular Neurobiology · doi:10.1007/s12035-021-02638-w · Cited by 58

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

Immune responses to gene editing by viral and non-viral delivery vectors used in retinal gene therapy

Ren, D., Fisson, S., Dalkara, D., & Ail, D. (2022) · Pharmaceutics · doi:10.3390/pharmaceutics14091973 · Cited by 52

CRISPR-derived genome editing technologies for metabolic engineering

Nishida, K., & Kondo, A. (2020) · Metabolic Engineering · doi:10.1016/j.ymben.2020.12.002 · Cited by 54

Hypercompact adenine base editors based on a Cas12f variant guided by engineered RNA

Kim, D. Y., Chung, Y., Lee, Y., Jeong, D., Park, K., Chin, H. J., Lee, J. M., Park, S., Ko, S., Ko, J., et al. (2022) · Nature Chemical Biology · doi:10.1038/s41589-022-01077-5 · Cited by 52

Stimulus-Responsive Smart Nanoparticles-Based CRISPR-Cas Delivery for Therapeutic Genome Editing

Naeem, M., Hoque, M. Z., Ovais, M., Basheer, C., & Ahmad, I. (2021) · International Journal of Molecular Sciences · doi:10.3390/ijms222011300 · Cited by 51

Effective use of sequence information to predict CRISPR-Cas9 off-target

Zhang, Z.-R., & Jiang, Z. (2022) · Computational and Structural Biotechnology Journal · doi:10.1016/j.csbj.2022.01.006 · Cited by 51

The CRISPR-Cas system as a tool for diagnosing and treating infectious diseases

Lou, J., Wang, B., Li, J., Ni, P., Jin, Y., Chen, S., Xi, Y., Zhang, R., & Duan, G. (2022) · Molecular Biology Reports · doi:10.1007/s11033-022-07752-z · Cited by 50

The paths toward non-viral CAR-T cell manufacturing: A comprehensive review of state-of-the-art methods

Metanat, Y., Viktor, P., Amajd, A., Kaur, I., Hamed, A. M., Al-Abadi, N. K. A., Alwan, N. H., Chaitanya, M., Natrayan, L., Ghildiyal, P., et al. (2024) · Life Sciences · doi:10.1016/j.lfs.2024.122683 · Cited by 49

Current status of the application of gene editing in pigs

Tanihara, F., Hirata, M., & Otoi, T. (2021) · Journal of Reproduction and Development · doi:10.1262/jrd.2021-025 · Cited by 49

Revolutionizing in vivo therapy with CRISPR/Cas genome editing: breakthroughs, opportunities and challenges

Palacios, A. M., Korus, P., Wilkens, B. G. C., Heshmatpour, N., & Patnaik, S. R. (2024) · Frontiers in Genome Editing · doi:10.3389/fgeed.2024.1342193 · Cited by 49

In vivo processing of digital information molecularly with targeted specificity and robust reliability

Liu, Y., Ren, Y., Li, J., Wang, F., Wang, F., Ma, C., Chen, D., Jiang, X., Fan, C., Zhang, H., et al. (2022) · Science Advances · doi:10.1126/sciadv.abo7415 · Cited by 47

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

Therapy Development by Genome Editing of Hematopoietic Stem Cells

Koniali, L., Lederer, C. W., & Kleanthous, M. (2021) · Cells · doi:10.3390/cells10061492 · Cited by 50

CRISPR-Cas technology based genome editing for modification of salinity stress tolerance responses in rice (Oryza sativa L.)

Khan, I., Khan, S., Zhang, Y., Zhou, J., Akhoundian, M., & Jan, S. A. (2021) · Molecular Biology Reports · doi:10.1007/s11033-021-06375-0 · Cited by 45

Deciphering the recent trends in pesticide bioremediation using genome editing and multi-omics approaches: a review

Hassan, S., & Ganai, B. A. (2023) · World Journal of Microbiology and Biotechnology · doi:10.1007/s11274-023-03603-6 · Cited by 45

Development of a base editor for protein evolution via in situ mutation in vivo

Hao, W., Cui, W., Cheng, Z., Han, L., Suo, F., Liu, Z., Zhou, L., & Zhou, Z. (2021) · Nucleic Acids Research · doi:10.1093/nar/gkab673 · Cited by 43

Exploiting the CRISPR‐Cas9 gene‐editing system for human cancers and immunotherapy

Afolabi, L. O., Afolabi, M. O., Sani, M. M., Okunowo, W. O., Yan, D., Chen, L., Zhang, Y., & Wan, X. (2021) · Clinical & Translational Immunology · doi:10.1002/cti2.1286 · Cited by 46

Gene Therapy and Gene Editing for β-Thalassemia

Christakopoulos, G. E., Telange, R., Yen, J. S., & Weiss, M. J. (2023) · Hematology/Oncology Clinics of North America · doi:10.1016/j.hoc.2022.12.012 · Cited by 43

Human iPSCs and Genome Editing Technologies for Precision Cardiovascular Tissue Engineering

Gähwiler, E. K. N., Motta, S. E., Martin, M., Nugraha, B., Hoerstrup, S. P., & Emmert, M. Y. (2021) · Frontiers in Cell and Developmental Biology · doi:10.3389/fcell.2021.639699 · Cited by 42

CRISPR/Cas9 as a therapeutic tool for triple negative breast cancer: from bench to clinics

Tiwari, P. K., Ko, T.-H., Dubey, R., Chouhan, M., Tsai, L.-W., Singh, H. N., Chaubey, K. K., Dayal, D., Chiang, C., & Kumar, S. (2023) · Frontiers in Molecular Biosciences · doi:10.3389/fmolb.2023.1214489 · Cited by 42

R-CRISPR: A Deep Learning Network to Predict Off-Target Activities with Mismatch, Insertion and Deletion in CRISPR-Cas9 System

Niu, R., Peng, J., Zhang, Z., & Shang, X. (2021) · Genes · doi:10.3390/genes12121878 · Cited by 43

Massively Parallel CRISPR‐Based Genetic Perturbation Screening at Single‐Cell Resolution

Cheng, J., Lin, G., Wang, T., Wang, Y., Guo, W., Liao, J., Yang, P., Chen, J., Shao, X., Lu, X., et al. (2022) · Advanced Science · doi:10.1002/advs.202204484 · Cited by 42

Gene Therapy Targeting PCSK9

Katzmann, J. L., Cupido, A. J., & Laufs, U. (2022) · Metabolites · doi:10.3390/metabo12010070 · Cited by 41

Recent Advances in Genome-Engineering Strategies

Boti, M. A., Athanasopoulou, K., Adamopoulos, P. G., Sideris, D. C., & Scorilas, A. (2023) · Genes · doi:10.3390/genes14010129 · Cited by 44

Integrating physiological and multi-omics methods to elucidate heat stress tolerance for sustainable rice production

Singh, S., Praveen, A., Dudha, N., & Bhadrecha, P. (2024) · Physiology and Molecular Biology of Plants · doi:10.1007/s12298-024-01480-3 · Cited by 39

One-Step Generation of Multiple Gene-Edited Pigs by Electroporation of the CRISPR/Cas9 System into Zygotes to Reduce Xenoantigen Biosynthesis

Tanihara, F., Hirata, M., Nguyen, N. T., Sawamoto, O., Kikuchi, T., & Otoi, T. (2021) · International Journal of Molecular Sciences · doi:10.3390/ijms22052249 · Cited by 39

Unlocking the potential of cultivated meat through cell line engineering

Riquelme‐Guzmán, C., Stout, A. J., Kaplan, D. L., & Flack, J. E. (2024) · iScience · doi:10.1016/j.isci.2024.110877 · Cited by 38

Implementation of CRISPR/Cas9 Genome Editing to Generate Murine Lung Cancer Models That Depict the Mutational Landscape of Human Disease

Hartmann, O., Reissland, M., Maier, C. R., Fischer, T., Prieto‐Garcia, C., Baluapuri, A., Schwarz, J. D., Schmitz, W., Garrido‐Rodríguez, M., Pahor, N., et al. (2021) · Frontiers in Cell and Developmental Biology · doi:10.3389/fcell.2021.641618 · Cited by 38

Understanding and overcoming adverse consequences of genome editing on hematopoietic stem and progenitor cells

Lee, B., Lozano, R. J., & Dunbar, C. E. (2021) · Molecular Therapy · doi:10.1016/j.ymthe.2021.09.001 · Cited by 39

Nrf2 pathways in neuroprotection: Alleviating mitochondrial dysfunction and cognitive impairment in aging

Bhat, A. A., Moglad, E. H. O., Goyal, A., Afzal, M., Thapa, R., Almalki, W. H., Kazmi, I. A., Alzarea, S. I., Ali, H., Gaur, A., et al. (2024) · Life Sciences · doi:10.1016/j.lfs.2024.123056 · Cited by 38

CRISPR/Cas9 based genome editing for targeted transcriptional control in triple-negative breast cancer

Singh, D. D., Han, I., Choi, E. H., & Yadav, D. K. (2021) · Computational and Structural Biotechnology Journal · doi:10.1016/j.csbj.2021.04.036 · Cited by 39

CRISPR-targetedMAGT1insertion restores XMEN patient hematopoietic stem cells and lymphocytes

Brault, J., Liu, T., Bello, E. A., Liu, S., Sweeney, C. L., Meis, R. J., Koontz, S. M., Corsino, C. I., Choi, U., Vayssière, G., et al. (2021) · Blood · doi:10.1182/blood.2021011192 · 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

Applications of CRISPR/Cas genome editing in economically important fruit crops: recent advances and future directions

Ma, Z., Ma, L., & Zhou, J. (2023) · Molecular Horticulture · doi:10.1186/s43897-023-00049-0 · Cited by 37

Exploring Parkinson-associated kinases for CRISPR/Cas9-based gene editing: beyond alpha-synuclein

Mansour, H. M., & El-Khatib, A. S. (2023) · Ageing Research Reviews · doi:10.1016/j.arr.2023.102114 · Cited by 36

Emerging strategies for nanomedicine in autoimmunity

Thatte, A. S., Billingsley, M. M., Weissman, D., Melamed, J. R., & Mitchell, M. J. (2024) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2024.115194 · Cited by 36

Optimizing CRISPR/Cas9 precision: Mitigating off-target effects for safe integration with photodynamic and stem cell therapies in cancer treatment

Merlin, J. P. J., & Abrahamse, H. (2024) · Biomedicine & Pharmacotherapy · doi:10.1016/j.biopha.2024.117516 · Cited by 35

Simultaneous knockout of multiple LHCF genes using single sgRNAs and engineering of a high‐fidelity Cas9 for precise genome editing in marine algae

Sharma, A. K., Nymark, M., Flo, S., Sparstad, T., Bones, A. M., & Winge, P. (2021) · Plant Biotechnology Journal · doi:10.1111/pbi.13582 · Cited by 36

CRISPR-Cas system: from diagnostic tool to potential antiviral treatment

Rajan, A., Shrivastava, S., Janhawi, Kumar, A., Singh, A. K., & Arora, P. K. (2022) · Applied Microbiology and Biotechnology · doi:10.1007/s00253-022-12135-2 · Cited by 36

Integrative Approaches to Soybean Resilience, Productivity, and Utility: A Review of Genomics, Computational Modeling, and Economic Viability

Yuhong, G., Liu, S.-H., Zhang, Z., Wei, J., Wang, H., Liu, L. N., Bai, Q., Qin, Q., Zhao, C., Zhang, S., et al. (2025) · Plants · doi:10.3390/plants14050671 · Cited by 38

One-pot synthesized three-way junction based multiple strand displacement amplification for sensitive assay of H5N1 DNA

Wu, Z., Li, J., Zhang, T., Zhang, K., Liu, X., Yang, Z., Li, X., & Han, K. (2025) · The Analyst · doi:10.1039/d4an01586j · Cited by 34

Novel Insights into the Therapeutic Potential of Lung-Targeted Gene Transfer in the Most Common Respiratory Diseases

Bisserier, M., Sun, X., Fazal, S., Turnbull, I. C., Bonnet, S., & Hadri, L. (2022) · Cells · doi:10.3390/cells11060984 · Cited by 36

Delivery strategies for CRISPR/Cas genome editing tool for retinal dystrophies: Challenges and opportunities

Lohia, A., Sahel, D. K., Salman, M., Singh, V., Mariappan, I., Mittal, A., & Chitkara, D. (2022) · Asian Journal of Pharmaceutical Sciences · doi:10.1016/j.ajps.2022.02.001 · Cited by 33

Nanoparticle Targeting Strategies for Lipid and Polymer‐Based Gene Delivery to Immune Cells In Vivo

Jain, M., Yu, X., Schneck, J. P., & Green, J. J. (2024) · Small Science · doi:10.1002/smsc.202400248 · Cited by 35

CRISPR/Cas9-Based therapeutics as a promising strategy for management of Alzheimer’s disease: progress and prospects

Khan, M. S., Qureshi, N., Khan, R., Son, Y., & Maqbool, T. (2025) · Frontiers in Cellular Neuroscience · doi:10.3389/fncel.2025.1578138 · Cited by 37

CRISPR-Assisted Probiotic and In Situ Engineering of Gut Microbiota: A Prospect to Modification of Metabolic Disorders

Rahmati, R., Zarimeidani, F., Boroujeni, M. R. G., Sadighbathi, S., Kashaniasl, Z., Saleh, M., & Alipourfard, I. (2025) · Probiotics and Antimicrobial Proteins · doi:10.1007/s12602-025-10561-y · Cited by 36

Current advances in overcoming obstacles of CRISPR/Cas9 off-target genome editing

Aquino‐Jarquín, G. (2021) · Molecular Genetics and Metabolism · doi:10.1016/j.ymgme.2021.08.002 · Cited by 34

Gene Therapy for Polyglutamine Spinocerebellar Ataxias: Advances, Challenges, and Perspectives

Vázquez‐Mojena, Y., León‐Arcia, K., González-Zaldívar, Y., Rodríguez‐Labrada, R., & Velázquez‐Pérez, L. (2021) · Movement Disorders · doi:10.1002/mds.28819 · Cited by 34

Regulatory Considerations for Clinical Trial Applications with CRISPR-Based Medicinal Products

Anliker, B., Childs, L. H., Rau, J., Renner, M., Schüle, S., Schuessler‐Lenz, M., & Sebe, A. (2022) · The CRISPR Journal · doi:10.1089/crispr.2021.0148 · Cited by 33

Overview of advances in CRISPR/deadCas9 technology and its applications in human diseases

Kanafi, M. M., & Tavallaei, M. (2022) · Gene · doi:10.1016/j.gene.2022.146518 · Cited by 37

Oligodendrogenesis and myelination tracing in a CRISPR/Cas9-engineered brain microphysiological system

Romero, J. C., Berlinicke, C. A., Chow, S., Duan, Y., Wang, Y., Chamling, X., & Smirnova, L. (2023) · Frontiers in Cellular Neuroscience · doi:10.3389/fncel.2022.1094291 · Cited by 34

A Promising RNA Nanotechnology in Clinical Therapeutics: a Future Perspective Narrative Review

Tolba, M. M., Jabbar, A., Afzal, S., Mahmoud, M., Zulfiqar, F., El-Soudany, I., Samir, S., Wadan, A. S., Ellakwa, T. E., & Ellakwa, D. E. (2023) · Future Science OA · doi:10.2144/fsoa-2023-0067 · Cited by 30

Nanotechnology‐based CRISPR/Cas9 delivery system for genome editing in cancer treatment

Zhou, S., Li, Y., Wu, Q., & Gong, C. (2024) · MedComm – Biomaterials and Applications · doi:10.1002/mba2.70 · Cited by 30

The Interface of Gene Editing with Regenerative Medicine

Farag, V., Devey, E. A., & Leong, K. W. (2024) · Engineering · doi:10.1016/j.eng.2024.10.019 · Cited by 30

Engineering Useful Microbial Species for Pharmaceutical Applications

САДАНОВ, А. К., Baimakhanova, B. B., Orasymbet, S., Ратникова, И. А., TURLYBAYEVA⃰, Z. Z., Baimakhanova, G. B., Amitova, A. A., Omirbekova, A. A., Aitkaliyeva, G., Kossalbayev, B. D., et al. (2025) · Microorganisms · doi:10.3390/microorganisms13030599 · Cited by 32

A risk-based approach for cell line development, manufacturing and characterization of genetically engineered, induced pluripotent stem cell–derived allogeneic cell therapies

Dashnau, J. L., Xue, Q., Nelson, M., Law, E., Cao, L., & Hei, D. J. (2022) · Cytotherapy · doi:10.1016/j.jcyt.2022.08.001 · Cited by 30

CRISPR/Cas9—A Promising Therapeutic Tool to Cure Blindness: Current Scenario and Future Prospects

Ahmad, I. (2022) · International Journal of Molecular Sciences · doi:10.3390/ijms231911482 · Cited by 30

State of the Art and Perspectives of Gene Therapy in Heart Failure. A Scientific Statement of the Heart Failure Association of the ESC, the ESC Council on Cardiovascular Genomics and the ESC Working Group on Myocardial & Pericardial Diseases

Linthout, S. V., Stellos, K., Giacca, M., Bertero, E., Cannata, A. N., Carrier, L., García‐Pavía, P., Ghigo, A., González, A., Haugaa, K. H., et al. (2024) · European Journal of Heart Failure · doi:10.1002/ejhf.3516 · Cited by 29

Genetic therapies for the first molecular disease

Doerfler, P. A., Sharma, A., Porter, J. S., Zheng, Y., Tisdale, J. F., & Weiss, M. J. (2021) · Journal of Clinical Investigation · doi:10.1172/jci146394 · Cited by 30

In vivo peptide-based delivery of a gene-modifying enzyme into cells of the central nervous system

Allen, J. K., Sutherland, T. C., Prater, A. R., Geoffroy, C. G., & Pellois, J. (2022) · Science Advances · doi:10.1126/sciadv.abo2954 · Cited by 30

Gene Therapy Approaches for the Treatment of Hemophilia B

Soroka, A. B., Feoktistova, S. G., Mityaeva, O. N., & Volchkov, P. (2023) · International Journal of Molecular Sciences · doi:10.3390/ijms241310766 · Cited by 29

Off-target effects in CRISPR/Cas9 gene editing

Guo, C., Ma, X., Gao, F., & Guo, Y. (2023) · Frontiers in Bioengineering and Biotechnology · doi:10.3389/fbioe.2023.1143157 · Cited by 704

Recent advancements in reducing the off-target effect of CRISPR/Cas9 genome editing

Asmamaw Mengstie, M., Teshome Azezew, M., Asmamaw Dejenie, T., Teshome, A. A., Tadele Admasu, F., Behaile Teklemariam, A., Tilahun Mulu, A., Mekonnen Agidew, M., Adugna, D. G., Geremew, H., et al. (2024) · Biologics: Targets and Therapy · doi:10.2147/btt.s429411 · Cited by 91

Assessing off-target effects in CRISPR/Cas9: challenges and strategies for precision DNA editing

Basit, A., Zhu, J., & Zheng, W. (2026) · Archives of Microbiology · doi:10.1007/s00203-025-04687-4 · Cited by 11

Progress of application and off-target effects of CRISPR/Cas9.

Zheng, W., & Gu, F. (2015) · PubMed · doi:10.16288/j.yczz.15-070 · Cited by 10

Emerging strategies to minimize the off-target effects in CRISPR/Cas9 system

Ouyang, C. (2024) · International Conference on Biological Engineering and Medical Science (ICBioMed) · doi:10.1117/12.3013214 · Cited by 1