Delivery of CRISPR-Cas tools for in vivo genome editing therapy: Trends and challenges

A Programmable Dual-RNA–Guided DNA Endonuclease in Adaptive Bacterial Immunity

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

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A bivalent chromatin structure marks key developmental genes in embryonic stem cells

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

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Programmable base editing of A•T to G•C in genomic DNA without DNA cleavage

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CRISPR-Mediated Modular RNA-Guided Regulation of Transcription in Eukaryotes

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

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High-Frequency off-Target Mutagenesis Induced by CRISPR-Cas Nucleases in Human Cells

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Genome-Scale CRISPR-Mediated Control of Gene Repression and Activation

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In vivo genome editing using Staphylococcus aureus Cas9

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Shining light on CRISPR/Cas9 therapeutics for inherited retinal diseases

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Innovative cancer nanomedicine based on immunology, gene editing, intracellular trafficking control

Yamada, Y., Sato, Y., Nakamura, T., & Harashima, H. (2022) · Journal of Controlled Release · doi:10.1016/j.jconrel.2022.05.033 · Cited by 11

Optically Controlled CRISPR-Cas9 and Cre Recombinase for Spatiotemporal Gene Editing: A Review

Devarajan, A. (2023) · ACS Synthetic Biology · doi:10.1021/acssynbio.3c00596 · Cited by 11

Successful Gene Editing of Apolipoprotein E4 to E3 in Brain of Alzheimer Model Mice After a Single IV Dose of Synthetic Exosome-Delivered CRISPRPreprint

Teter, B., Campagna, J. J., Zhu, C., McAuley, G. E., Spilman, P. R., Kohn, D. B., & John, V. (2024) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/2024.04.23.590784 · Cited by 12

CRISPR applications in microbial World: Assessing the opportunities and challenges

Kursheed, F., Naz, E., Mateen, S., & Kulsoom, U. (2024) · Gene · doi:10.1016/j.gene.2024.149075 · Cited by 10

Improving the use of CRISPR/Cas9 gene editing machinery as a cancer therapeutic tool with the help of nanomedicine

Fatima, H., Singh, D., Muhammad, H., Acharya, S., & Aziz, M. A. (2024) · 3 Biotech · doi:10.1007/s13205-024-04186-1 · Cited by 12

Optimization of mtDNA-targeted platinum TALENs for bi-directionally modifying heteroplasmy levels in patient-derived m.3243A>G-iPSCs

Yahata, N., Goto, Y., & Hata, R. (2025) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2025.102521 · Cited by 10

RNA splicing as a therapeutic target in myelodysplastic syndromes

Tseng, C.-C., & Obeng, E. A. (2024) · Seminars in Hematology · doi:10.1053/j.seminhematol.2024.10.005 · Cited by 11

Advances in Gene Therapy for Sickle Cell Disease: From Preclinical Innovations to Clinical Implementation and Access Challenges

Butt, H., Mandava, M., & Jacobsohn, D. A. (2025) · The CRISPR Journal · doi:10.1089/crispr.2024.0101 · Cited by 9

CRISPR/cas genome editing for neurodegenerative diseases: Mechanisms, therapeutic advances, and clinical prospects

Pandya, K., & Kumar, D. (2025) · Ageing Research Reviews · doi:10.1016/j.arr.2025.102922 · Cited by 9

Gene editing strategies to treat lysosomal disorders: The example of mucopolysaccharidoses

Fachel, F. N. S., Frâncio, L., Poletto, É., Schuh, R. S., Teixeira, H. F., Giugliani, R., Baldo, G., & Matte, Ú. D. S. (2022) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2022.114616 · Cited by 9

Targeted Gene Insertion: The Cutting Edge of CRISPR Drug Development with Hemophilia as a Highlight

Zhang, Z., Zhang, S., Wong, H. T., Li, D., & Feng, B. (2024) · BioDrugs · doi:10.1007/s40259-024-00654-5 · Cited by 9

Lipopeptide-mediated delivery of CRISPR/Cas9 ribonucleoprotein complexes for gene editing and correction

Öktem, M., Nguyen, T. H., Bosman, E., Fens, M. H. A. M., Caiazzo, M., Mastrobattista, E., Lei, Z., & de Jong, O. G. (2025) · Journal of Controlled Release · doi:10.1016/j.jconrel.2025.113854 · Cited by 8

Advances in Epigenome Engineering: Mastering Technical Approaches for Better Outcomes

Khalil, A. M. (2023) · Journal of Medical Sciences and Interdisciplinary Research · doi:10.51847/ibbxxqhvqh · Cited by 8

Genome Editing in Ferret Airway Epithelia Mediated by CRISPR/Nucleases Delivered with Amphiphilic Shuttle Peptides

Luo, M., Ma, J., Cheng, X., Wu, S., Bartels, D., Guay, D. R. P., Engelhardt, J. F., & Liu, X. (2023) · Human Gene Therapy · doi:10.1089/hum.2023.016 · Cited by 8

Deep Characterization and Comparison of Different Retrovirus-like Particles Preloaded with CRISPR/Cas9 RNPs

Wichmann, M., Maire, C. L., Nuppenau, N., Habiballa, M., Uhde, A., Kolbe, K., Schröder, T., Lamszus, K., Fehse, B., & Głów, D. (2023) · International Journal of Molecular Sciences · doi:10.3390/ijms241411399 · Cited by 9

Viral delivery of CRISPR/Cas9 genome editing for rapid crop improvement: A promising approach to enhance crop resilience against biotic and abiotic stresses

Patil, A. M., Wagh, S. G., Janvale, G. B., Pawar, B. D., & Daspute, A. A. (2024) · International Journal of Advanced Biochemistry Research · doi:10.33545/26174693.2024.v8.i3sj.885 · Cited by 9

Retinal organoid and gene editing for basic and translational research

Cheng, Y.-M., Ma, C., Jin, K., & Jin, Z. (2023) · Vision Research · doi:10.1016/j.visres.2023.108273 · Cited by 8

Genome editing in the adrenal gland: a novel strategy for treating congenital adrenal hyperplasia

van Dijk, E. B., Ginn, S. L., Alexander, I. E., & Graves, L. E. (2024) · Exploration of Endocrine and Metabolic Diseases · doi:10.37349/eemd.2024.00011 · Cited by 8

Enzyme miniaturization: Revolutionizing future biocatalysts

Ding, N., Jiang, Y., Lee, S., Cheng, Z., Ran, X., Ding, Y., Ge, R., Zhang, Y., & Yang, Z. J. (2025) · Biotechnology Advances · doi:10.1016/j.biotechadv.2025.108598 · Cited by 8

Immunoengineering via Chimeric Antigen Receptor-T Cell Therapy: Reprogramming Nanodrug Delivery

Katopodi, T., Petanidis, S., Anestakis, D., Charalampidis, C., Chatziprodromidou, I. P., Floros, G., Eskitzis, P., Zarogoulidis, P., Koulouris, C., Sevva, C. D., et al. (2023) · Pharmaceutics · doi:10.3390/pharmaceutics15102458 · Cited by 7

Viral Delivery of Compact CRISPR-Cas12f for Gene Editing Applications

Sharrar, A. M., Meacham, Z. A., Staples-Ager, J., de Tacca, L. A., Rabuka, D., Collingwood, T. N., & Schelle, M. W. (2024) · The CRISPR Journal · doi:10.1089/crispr.2024.0010 · Cited by 8

CRISPR technology in human diseases

Feng, Q., Li, Q., Zhou, H., Wang, Z., Lin, C., Jiang, Z., Liu, T., & Wang, D. (2024) · MedComm · doi:10.1002/mco2.672 · Cited by 8

Quinine-Based Polymers Are Versatile and Effective Vehicles for Intracellular pDNA, mRNA, and Cas9 Protein Delivery

Roy, P., Kreofsky, N. W., & Reineke, T. M. (2024) · Biomacromolecules · doi:10.1021/acs.biomac.4c00925 · Cited by 7

Nanomaterials as signal amplifiers in CRISPR/Cas biosensors: A path toward multiplex point-of-care diagnostics

Arshad, F., Yee, B. J., Ting, K. P., Janudin, A. A. S., bin Amir, M. N. A., & Ahmed, M. U. (2024) · Microchemical Journal · doi:10.1016/j.microc.2024.111826 · Cited by 8

An NGS-based approach for precise and footprint-free CRISPR-based gene editing in human stem cells

Vandendriessche, B., Huyghebaert, J., Rossem, K. E. V., Cremers, T. C., Man, K. D., Sieliwończyk, E., Boen, H. M., Akdeniz, D., Rabaut, L., Schippers, J., et al. (2025) · Methods · doi:10.1016/j.ymeth.2025.05.004 · Cited by 7

Gene Therapy for Paediatric Homozygous Familial Hypercholesterolaemia

Graves, L. E., Horton, A., Alexander, I. E., & Srinivasan, S. (2023) · Heart Lung and Circulation · doi:10.1016/j.hlc.2023.01.017 · Cited by 7

A comprehensive review on the current status of CRISPR based clinical trials for rare diseases

Badwal, A. K., & Singh, S. (2024) · International Journal of Biological Macromolecules · doi:10.1016/j.ijbiomac.2024.134097 · Cited by 10

CRISPR challenges in clinical developments

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State of the art in CAR-based therapy: In vivo CAR production as a revolution in cell-based cancer treatment

Esmaeilzadeh, A., Hadiloo, K., Yaghoubi, S., Makoui, M. H., & Mostanadi, P. (2025) · Cellular Oncology · doi:10.1007/s13402-025-01056-7 · Cited by 7

CRISPR Technology: Transforming the Future of Medicine and Diagnostics

Iyer, K. A., Tenchov, R., Diaz, L. M. L., Jain, P., Thite, T., Deng, Y., & Zhou, Q. A. (2025) · Biochemistry · doi:10.1021/acs.biochem.5c00480 · Cited by 9

CRISPR/Cas9 genome editing-based preclinical theranostics, biomarkers and drug delivery systems for cancer applications

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On the road to in vivo CAR-T success: Comparing promising viral and non-viral vectors

de la Mota, S., Suchet, L., van Wijk, M., Stibbs, D. J., Couto, P. S., & Rafiq, Q. A. (2026) · Biotechnology Advances · doi:10.1016/j.biotechadv.2026.108907 · Cited by 6

Advances in CRISPR/Cas systems-based cell and gene therapy

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Control of HSV-1 Infection: Directions for the Development of CRISPR/Cas-Based Therapeutics and Diagnostics

Sosnovtseva, A. O., Demidova, N. A., Рафаиловна, К. Р., Kovalev, M. A., Aa, K., Starodubova, E. S., Latanova, A. A., & Karpov, D. S. (2024) · International Journal of Molecular Sciences · doi:10.3390/ijms252212346 · Cited by 6

Gene targeting in adult organs using in vivo cleavable donor plasmids for CRISPR-Cas9 and CRISPR-Cas12a

Ishibashi, R., Maki, R., & Toyoshima, F. (2024) · Scientific Reports · doi:10.1038/s41598-024-57551-8 · Cited by 6

From IscB to Cas9: Engineering and advances in the next generation of miniature gene editing tools

Zhou, C., Zhu, S., Luo, C., Wang, W., Fan, H.-Q., Gao, Y., Xu, X., Wang, Q., Yu, Y., & Xie, T. (2025) · Biotechnology Advances · doi:10.1016/j.biotechadv.2025.108743 · Cited by 5

Nanocarrier-mediated CRISPR-Cas delivery: a novel approach against antibiotic-resistant superbugs

Almufarriji, F. M. (2026) · Saudi Pharmaceutical Journal · doi:10.1007/s44446-026-00064-4 · Cited by 5

Next-generation gene therapy for infectious disease: Advances, challenges, and future directions

Fattahi, M., Pirbadami, F., Motallebirad, T., Beheshti, Z., & Azadi, D. (2026) · Journal of Infection and Public Health · doi:10.1016/j.jiph.2026.103164 · Cited by 5

Nanodelivery in Gene Therapy

Eksi, O. B., Kutlu, A. Ü., Yumuk, K., Memet, B. C., Benk, R., Kurşunluoğlu, G., & Aydin, Ö. F. (2024) · Springer eBooks · doi:10.1007/978-3-030-80962-1_410-1 · Cited by 5

CRISPR/Cas9-mediated genome editing: from basic research to gene therapy

Goel, R. (2024) · International Journal of Research in Medical Sciences · doi:10.18203/2320-6012.ijrms20241589 · Cited by 7

Current application and future perspective of CRISPR/cas9 gene editing system mediated immune checkpoint for liver cancer treatment

Tong, L.-W., Hu, Y.-S., Yu, S.-J., Li, C., & Shao, J. (2024) · Nanotechnology · doi:10.1088/1361-6528/ad5f33 · Cited by 5

Lipopolymeric Nanocarrier Enables Effective Delivery of CRISPR/Cas9 Expressing Plasmid

Sahel, D. K., Goswami, S. G., Jatyan, R., Kumari, A., Mittal, A., Ramalingam, S., & Chitkara, D. (2023) · Macromolecular Rapid Communications · doi:10.1002/marc.202300101 · Cited by 5

exoCasMINI: A T5 exonuclease fused CRISPR-Cas12f system with enhanced gene editing efficiency

Liao, K., Chen, K., Ma, S., Yang, X., Chen, P., Li, S., Li, M., Zhang, X., & Lin, Y. (2025) · iScience · doi:10.1016/j.isci.2025.113171 · Cited by 4

Guanidyl-rich highly branched poly(β-amino ester)s for the delivery of dual CRISPR ribonucleoprotein for efficient large DNA fragment deletion

Wang, X., Li, Y., Friess, D., Yao, L., Wang, X., He, Z., He, W., Li, M., & Wang, W. (2025) · Journal of Controlled Release · doi:10.1016/j.jconrel.2025.01.032 · Cited by 4

Multi-Omics approaches in gene therapy for vascular diseases: bridging genomics, transcriptomics, and epigenetics

Dong, J., Sun, M., Li, Y., & Xie, Z. (2025) · Journal of drug targeting · doi:10.1080/1061186x.2025.2544786 · Cited by 4

Red Blood Cell-Derived Extracellular Vesicles for Gene and RNA Therapeutics: Biological, Engineering, and Translational Challenges

Shafiq, T., Khan, N., Kausar, T., Ahmed, W., Zhang, Z., Liang, Y., & Duan, L. (2026) · International Journal of Nanomedicine · doi:10.2147/ijn.s579975 · Cited by 5

Cure of Congenital Purpura Fulminans via Expression of Engineered Protein C Through Neonatal Genome Editing in Mice

Togashi, T., Baatartsogt, N., Nagao, Y., Kashiwakura, Y., Hayakawa, M., Kamoshita, N., Hiramoto, T., Fujiwara, T., Morishita, E., Nureki, O., et al. (2024) · Arteriosclerosis Thrombosis and Vascular Biology · doi:10.1161/atvbaha.123.319460 · Cited by 4

Prominent supramolecular systems for cancer Therapy: From structural design to tailored applications

Zhang, J., Zhang, Q., Li, X., Wei, Y., Qiu, M., Yang, H., & Sun, X. (2025) · European Journal of Medicinal Chemistry · doi:10.1016/j.ejmech.2025.117754 · Cited by 4

In vivo Delivery Tools for Clustered Regularly Interspaced Short Palindromic Repeat/Associated Protein 9-Mediated Inhibition of Hepatitis B Virus Infection: An Update

Kayesh, M. E. H., Hashem, M. A., Kohara, M., & Tsukiyama–Kohara, K. (2022) · Frontiers in Microbiology · doi:10.3389/fmicb.2022.953218 · Cited by 4

CRISPR: a tool with potential for genomic reprogramming in neurological disorders

Dhuriya, Y. K., & Naik, A. A. (2022) · Molecular Biology Reports · doi:10.1007/s11033-022-08136-z · Cited by 4

CRISPR Manipulation of Age-Related Macular Degeneration Haplotypes in the Complement System: Potential Future Therapeutic Applications/Avenues

Salman, A., McClements, M. E., & MacLaren, R. E. (2024) · International Journal of Molecular Sciences · doi:10.3390/ijms25031697 · Cited by 4

Single-guide RNA Cas9 and enhanced-deletion Cas9 rescue a recurrent USH2A-related splicing defect

Angeli, P. D., Spaag, S., Shliaga, S., Flores-Tufiño, A., Ritter, M. U., Nasri, M., Štingl, K., Kuehlewein, L., Wissinger, B., & Kohl, S. (2025) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2025.102523 · Cited by 3

Enhancer regulation in cancer: from epigenetics to m6A RNA modification

Vasukutty, A., Bhattarai, P. Y., & Choi, H. S. (2025) · Archives of Pharmacal Research · doi:10.1007/s12272-025-01561-1 · Cited by 5

Genetically Programmable Biohybrid Nanoplatform Enables Cuproptosis‐Immunotherapy via Spatiotemporal Control of Macrophage Polarization and Copper Flux

Wang, J., Li, H., Gao, M., Xie, Y., Zhu, G., Qian, Y., Lin, J., & Li, C. (2025) · Advanced Functional Materials · doi:10.1002/adfm.202510738 · Cited by 3

Non-viral delivery of genome-editing tools for treatment of genetic disorders

Yang, J., Chen, Y., Chen, X., & Ping, Y. (2026) · Acta Pharmaceutica Sinica B · doi:10.1016/j.apsb.2026.02.014 · Cited by 3

Virus-like particles: Bridging immunization and precision drug delivery

Gupta, J., Tiwari, P., & Kumar, R. (2026) · Gene · doi:10.1016/j.gene.2026.150208 · Cited by 3

CRISPR-Based Approaches for Cancer Immunotherapy

Malla, R. R., & Middela, K. (2023) · Critical Reviews™ in Oncogenesis · doi:10.1615/critrevoncog.2023048723 · Cited by 3

Recent development in CRISPR-Cas systems for cardiac disease

Munshi, I. D., Acharya, M., Mukherjee, S., & Mani, I. (2024) · Progress in molecular biology and translational science · doi:10.1016/bs.pmbts.2024.08.004 · Cited by 5

Genome Engineering of Primary and Pluripotent Stem Cell-Derived Hepatocytes for Modeling Liver Tumor Formation

Zhang, L., Wang, X., Yang, X., Chi, Y., Chu, Y., Zhang, Y., Gong, Y., Wang, F., Zhao, Q., & Zhao, D. (2024) · Biology · doi:10.3390/biology13090684 · Cited by 4

Comparative examination of the chemistry and biology of AI-driven gold NPs in Theranostics: New insights into biosensing, bioimaging, genomics, diagnostics, and therapy

Esmaeilpour, D., Zare‬, E. N., Hassanpur, M., Sher, F., & Sillanpää, M. E. T. (2025) · Nanomedicine Nanotechnology Biology and Medicine · doi:10.1016/j.nano.2025.102821 · Cited by 3

Lymphoid and CXCR4 Cell Targeted Lipid Nanoparticles Facilitate HIV‐1 Proviral DNA Excision

Panja, S., Zaman, L. A., Zhang, C., Patel, M., Gorantla, S., Dash, P. K., & Gendelman, H. E. (2025) · Advanced Healthcare Materials · doi:10.1002/adhm.202501190 · Cited by 6

Precision Delivery of CRISPR/Cas Systems via DNA Nanostructures for Gene Therapy and Intracellular Detection

Song, N., Wang, L., Zhang, L., Tian, G., Yao, C., & Yang, D. (2025) · ChemBioChem · doi:10.1002/cbic.202500357 · Cited by 3

Application of Gene Editing Technology Based on Targeted Delivery Materials in TNBC

Qu, P., Li, X., Liu, J., Su, X., Han, S., Wang, Y., Luo, Y., Li, J., Ma, C., Deng, S., et al. (2025) · ACS Omega · doi:10.1021/acsomega.5c01294 · Cited by 4

Illuminating the genome: emerging approaches in CRISPR-Cas live-cell imaging

Xiao, Z., & Sun, Y. (2026) · Nucleic Acids Research · doi:10.1093/nar/gkaf1540 · Cited by 5

Tetraspanin CD81 is expressed in human parotid cancer tissue and mediates cell proliferation

Hamada, W., Kato‐Kogoe, N., Yamanegi, K., Kanetake, H., Hirata, A., Terada, T., Kurisu, Y., Une, H., Hirose, Y., Nakanishi, T., et al. (2023) · Journal of Oral and Maxillofacial Surgery Medicine and Pathology · doi:10.1016/j.ajoms.2023.08.012 · Cited by 3

Genome Engineering as a Therapeutic Approach in Cancer Therapy: A Comprehensive Review

Gemayel, J., Chebly, A., Kourié, H. R., Hanna, C., Mheidly, K., Mhanna, M., Karam, F., Ghoussaini, D., Najjar, P. E., & Khalil, C. (2024) · Advanced Genetics · doi:10.1002/ggn2.202300201 · Cited by 2

Evaluation of subretinally delivered Cas9 ribonucleoproteins in murine and porcine animal models highlights key considerations for therapeutic translation of genetic medicines

Wei, S. C., Cantor, A. J., Walleshauser, J., Mepani, R. J., Melton, K., Bans, A., Khekare, P., Gupta, S., Wang, J., Soares, C., et al. (2025) · PLoS ONE · doi:10.1371/journal.pone.0317387 · Cited by 3

Cardiovascular Therapeutics at the Crossroads: Pharmacological, Genetic, and Digital Frontiers

Vetrano, E., Caturano, A., Nilo, D., Lorenzo, G. D., Tagliaferri, G., Piacevole, A., Donnarumma, M., Iadicicco, I., Picco, S., Moretto, S. M., et al. (2025) · Pharmaceuticals · doi:10.3390/ph18111703 · Cited by 2

eVLP‐Mediated Cas9 Delivery for Preventing IBMIR in Islet Transplantation

Shrestha, M., Kim, Y., Park, S. B., Jiang, H., Lee, J., Kim, D., & Jeong, J. (2025) · Small · doi:10.1002/smll.202508128 · Cited by 2

Engineered miniature CRISPR activation system promotes dodecanedioic acid production in Candida viswanathii

Pham, N. N., Chien, C.-H., Chang, C.-W., & Hu, Y. (2025) · Journal of the Taiwan Institute of Chemical Engineers · doi:10.1016/j.jtice.2025.106548 · Cited by 2

Exploration of the potential of genomic editing in the treatment of congenital adrenal hyperplasia

Graves, L. E., Christina, S., Mullany, K., Alexander, I. E., & Falhammar, H. (2025) · Frontiers in Endocrinology · doi:10.3389/fendo.2025.1719376 · Cited by 2

Characterization and engineering of highly efficient Cas12j genome editors

Rao, G. S., Jiang, W., Aouida, M., Wang, Q., Kazlak, A. M., Elbehery, A. H. A., Saleh, A., Masood, M., Ghouneimy, A., & Mahfouz, M. E. (2026) · Trends in biotechnology · doi:10.1016/j.tibtech.2026.02.001 · Cited by 2

Next-generation gene editing strategies in cancer: Integrating CRISPR, PROTACs, and advanced molecular technologies

Vikal, A., Maurya, R., Kumar, P., Verma, D. N., & Mishra, A. K. (2026) · Life Sciences · doi:10.1016/j.lfs.2026.124434 · Cited by 2

Recent advances in CRISPR-Cas system for the treatment of genetic hearing loss.

Yin, G., Wang, X., & Sun, Y. (2023) · PubMed Central · Cited by 3

Virus-Like Particles as a CRISPR-Cas9 Delivery Tool

Shindadkar, S., & Severson, K. M. (2024) · Journal of Undergraduate Life Sciences · doi:10.33137/juls.v16i1.42386 · Cited by 2

Expanded toolkits for RNA circularization

Wang, X., Huang, Y., & Chen, L. (2024) · Nature Biomedical Engineering · doi:10.1038/s41551-024-01262-y · Cited by 3

A conceptual model and practical guidance for the development, administration, and evaluation of individualized therapies

Perillat, L. O. M., McFadyen, A., Furlong, P., & Anderson, J. (2025) · Frontiers in Medicine · doi:10.3389/fmed.2025.1493832 · Cited by 2

Strategies and challenges of cytosolic delivery of proteins

Li, Y., Du, B., Yu, L., Luo, H., Rong, H., Gao, X., & Yin, J. (2025) · Journal of drug targeting · doi:10.1080/1061186x.2025.2458616 · Cited by 2

Nano-carrier-based Delivery of CRISPR-Cas9 for Oncolytic Gene Therapy: Insights from Xenograft Models

Bahmani, M. (2025) · Journal of Lab Animal Research · doi:10.58803/jlar.v4i2.66 · Cited by 2

A comprehensive review on CRISPR-Cas9 in revolutionizing food and agricultural sectors

Haddad, M. A., Abu-Romman, S. M., Rather, J. A., Akhter, N., Khan, B. A., & Dalaeen, S. (2025) · Food and Humanity · doi:10.1016/j.foohum.2025.100679 · Cited by 2

Safe delivery of AAV vectors to the liver of small weaned pigs by ultrasound-guided percutaneous transhepatic portal vein injectionPreprint

Rothgangl, T., Hruzova, M., Gnannt, R., Aeberhard, N., Kissling, L., Grisch‐Chan, H. M., Klassa, S., Rimann, N., Marquart, K. F., Ioannidi, E. I., et al. (2023) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/2023.04.05.535660 · Cited by 2

Efficient in vivo mammalian neuron editing using peptide-mediated CRISPR enzyme deliveryPreprint

Manohar, B. D., Travis, M., Munjal, V., Baehr, C. M., Rahnama, L. M. A., Kang, M. H., Haddad, R. F., Asija, K., Noel, E. A., Peddireddy, N., et al. (2025) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/2025.11.26.690638 · Cited by 2

RNA N4-acetylcytidine modification in gastrointestinal cancer: Novel functions, mechanisms, and therapeutic implications

Lu, J., Gao, Y., Li, Q., Jiao, Y., Bian, Y., Lin, H., Lei, X., Wang, W., Li, Z., & Wang, L. (2025) · Critical Reviews in Oncology/Hematology · doi:10.1016/j.critrevonc.2025.104867 · Cited by 1

RNA-Seq Identification of Peanut Callus-Specific Promoters and Evaluation of Base-Editing Efficiency

Xue, L., Liu, H., Zhao, H., Qu, P., Li, X., Wang, X., Huang, B., Sun, Z., Han, S., Dai, X., et al. (2025) · Plants · doi:10.3390/plants14152290 · Cited by 2

Charge-engineered lipid nanoparticles with poly (γ-glutamic acid) for improved CRISPR/Cas9 genome editing

Zhang, M., Gao, X., Zhang, H., Liu, W., Lv, H., & Yang, C. (2025) · Colloids and Surfaces A Physicochemical and Engineering Aspects · doi:10.1016/j.colsurfa.2025.138287 · Cited by 1

Multifunctional nano-polymer-based targeted delivery system for CRISPR/Cas9-Mediated hepatocellular carcinoma therapy

Huang, G., Tang, Y. J., Zhang, S., Ning, H., Xu, J., Li, F., & Zhang, X. (2025) · International Journal of Pharmaceutics · doi:10.1016/j.ijpharm.2025.126392 · Cited by 1

An overview of gene and cell therapy approaches for Alzheimer’s disease

Heidari, Z., Zakaee, A., Vafadar, A., Alavimanesh, S., Charami, H., Jamali, Z., Jahromi, A. H., Rakhsha, A., & Savardashtaki, A. (2025) · Metabolic Brain Disease · doi:10.1007/s11011-025-01763-y · Cited by 1

Recent Advances in Microemulsion Technology and Their Various Pharmaceutical Applications

Tiwari, J., Kumar, D., Anand, P., & Singh, S. K. (2026) · Recent Innovations in Chemical Engineering (Formerly Recent Patents on Chemical Engineering) · doi:10.2174/0124055204419934251123132954 · Cited by 1

CRISPR/Cas9 in cancer research and therapeutics: Clinical applications, optimization strategies, and future perspectives

Shariati, A., Homaei, A., Khezrpour, A., Azizi, N., Khani, P., Shariati, F. S., Rafiee, M., Afkhami, H., Yousefi, M. H., Saeidi, F., et al. (2026) · Current Research in Biotechnology · doi:10.1016/j.crbiot.2026.100382 · Cited by 1

Silica-based nanoplatforms for nucleic acid delivery: Emerging strategies and biomedical applications

Li, X., Bai, Y., Zhang, D., & Li, Y. (2026) · Nano Today · doi:10.1016/j.nantod.2026.103090 · Cited by 1

Light-controlled CRISPR–dCas9 epigenome editing: advanced drug-delivery strategies and oncology applications

Eskandani, N. A., Mirzaee, D., Farani, M. R., Hatami, A., Hatami, M., Ghasemzaei, M., Ghoreishian, S. M., Hwang, S.-K., & Huh, Y. S. (2026) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2026.115921 · Cited by 2

Lipid nanoparticles for Cas9 ribonucleoprotein delivery: design and evaluation of ionisable oligoamine-lipidoids

Lummerstorfer, M., Xue, Z., Zheng, D., König, L., Brunner, L., & Lächelt, U. (2026) · European Journal of Pharmaceutical Sciences · doi:10.1016/j.ejps.2026.107596 · Cited by 1

The future of pediatric gene therapy: CRISPR-Cas9, AI, and personalized medicine

Pan, Y., Ding, J., Wang, W., Xu, D., Fan, J., & Zhang, W. (2026) · Pediatric Research · doi:10.1038/s41390-026-05270-3 · Cited by 2

Biophysical considerations for designing viruses, lipid nanoparticles and virus-like particles for CRISPR-based genome editing

Ghosh, U., & Tay, A. K. P. (2026) · Journal of Controlled Release · doi:10.1016/j.jconrel.2026.115204 · Cited by 1

Epigenome Engineering: Understanding, Managing, and Improving Technical Aspects

Khalil, A. M. (2023) · Pharmacophore · doi:10.51847/wv4pydatfp · Cited by 1

Ocular Gene Therapy: Literature Review

Naser, M., Nasr, M., & Shehata, L. H. (2023) · International Journal of Progressive Sciences and Technologies · doi:10.52155/ijpsat.v40.2.5623 · Cited by 1

Reagents and their delivery systems in CRISPR/Cas

Khan, S., Zafar, H., & Ahmad, A. (2024) · Elsevier eBooks · doi:10.1016/b978-0-443-18444-4.00002-8 · Cited by 1

Novel Approaches for Greener Synthesis of Extremozymes Using Agro/Food Waste

Shah, F., Ranawat, B., Patel, V., Patidar, S. K., Thomas, R. M., & Mishra, S. (2024) · Springer eBooks · doi:10.1007/978-3-031-58441-1_10 · Cited by 1

Deletion of exons 45 to 55 in the DMD gene: from the therapeutic perspective to the in vitro model

Poyatos‐García, J., Soblechero‐Martín, P., Liquori, A., López‐Martínez, A., Maestre, P., González-Romero, E., Vázquez‐Manrique, R. P., Muelas, N., García‐García, G., Ohana, J., et al. (2024) · Skeletal Muscle · doi:10.1186/s13395-024-00353-3 · Cited by 2

Engineering adeno-associated viral vectors for CRISPR/Cas based in vivo therapeutic genome editing

Moyo, B., Brown, L. B. C., Khondaker, I., & Bao, G. (2025) · Biomaterials · doi:10.1016/j.biomaterials.2025.123314 · Cited by 13

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

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

Drug delivery systems for CRISPR-based genome editors

Madigan, V. J., Zhang, F., & Dahlman, J. E. (2023) · Nature Reviews Drug Discovery · doi:10.1038/s41573-023-00762-x · Cited by 193

Adenoviral vectors for in vivo delivery of CRISPR-Cas gene editors

Rice-Boucher, P. J., Cui, X., & Curiel, D. T. (2020) · Journal of Controlled Release · doi:10.1016/j.jconrel.2020.09.003 · Cited by 62

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

Yang, M., Song, Y., Wang, Z., Chao, K., Li, L., Zhang, X., Duan, X., Yu, C., Xue, R., & Zhao, J. (2026) · MedComm · doi:10.1002/mco2.70791 · Cited by 3