AAV-CRISPR Gene Editing Is Negated by Pre-existing Immunity to Cas9

Analysis of Relative Gene Expression Data Using Real-Time Quantitative PCR and the 2−ΔΔCT Method

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Mutation of the mouse klotho gene leads to a syndrome resembling ageing

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In vivo genome editing using Staphylococcus aureus Cas9

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Production of High-Titer Recombinant Adeno-Associated Virus Vectors in the Absence of Helper Adenovirus

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In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy

Nelson, C. E., Hakim, C. H., Ousterout, D. G., Thakore, P. I., Moreb, E. A., Rivera, R. M. C., Madhavan, S., Pan, X., Ran, F. A., Yan, W. X., et al. (2016) · Science · doi:10.1126/science.aad5143 · Cited by 1,144

Identification of preexisting adaptive immunity to Cas9 proteins in humans

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In vivo gene editing in dystrophic mouse muscle and muscle stem cells

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Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophy

Long, C., Amoasii, L., Mireault, A. A., McAnally, J., Li, H. Z., Sanchez‐Ortiz, E., Bhattacharyya, S., Shelton, J. M., Bassel‐Duby, R., & Olson, E. N. (2015) · Science · doi:10.1126/science.aad5725 · Cited by 973

Epitope spreading in immune-mediated diseases: implications for immunotherapy

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A Single Administration of CRISPR/Cas9 Lipid Nanoparticles Achieves Robust and Persistent In Vivo Genome Editing

Finn, J. D., Smith, A. R., Patel, M. C., Shaw, L., Youniss, M. R., van Heteren, J., Dirstine, T., Ciullo, C., Lescarbeau, R., Seitzer, J., et al. (2018) · Cell Reports · doi:10.1016/j.celrep.2018.02.014 · Cited by 878

Protection is not always a good thing: The immune system’s impact on gene therapy

de Freitas, M. V., Frâncio, L., Haleva, L., & Matte, Ú. D. S. (2022) · Genetics and Molecular Biology · doi:10.1590/1678-4685-gmb-2022-0046 · Cited by 18

Gene Editing to Tackle Facioscapulohumeral Muscular Dystrophy

Mariot, V., & Dumonceaux, J. (2022) · Frontiers in Genome Editing · doi:10.3389/fgeed.2022.937879 · Cited by 16

CD47 Knock‐Out Using CRISPR‐Cas9 RNA Lipid Nanocarriers Results in Reduced Mesenchymal Glioblastoma Growth In Vivo

Rouatbi, N., Walters, A. A., Zam, A., Lim, Y. M., Marrocu, A., Liam‐Or, R., Anstee, J. E., Arnold, J. N., Wang, J. T.-W., Pollard, S. M., et al. (2025) · Advanced Science · doi:10.1002/advs.202407262 · Cited by 15

Clinical perspective: Advancing hemophilia treatment through gene therapy approaches

Thornburg, C. D., PIPE, S. W., Cantore, A., Unzu, C., Jones, M., & Miesbach, W. (2025) · Molecular Therapy · doi:10.1016/j.ymthe.2025.04.023 · Cited by 13

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

CRISPR Gene-Editing Models Geared Toward Therapy for Hereditary and Developmental Neurological Disorders

Wong, P. K., Cheah, F., Syafruddin, S. E., Mohtar, M. A., Azmi, N., Ng, P. Y., & Chua, E. W. (2021) · Frontiers in Pediatrics · doi:10.3389/fped.2021.592571 · Cited by 14

Novel CRISPR-Associated Gene-Editing Systems Discovered in Metagenomic Samples Enable Efficient and Specific Genome Engineering

Lamothe, R. C., Storlie, M. D., Espinosa, D. A., Rudlaff, R. M., Browne, P. J., Liu, J., Rivas, A., Devoto, A. E., Oki, J., Khoubyari, A., et al. (2023) · The CRISPR Journal · doi:10.1089/crispr.2022.0089 · Cited by 12

AAV-delivered hepato-adrenal cooperativity in steroidogenesis: Implications for gene therapy for congenital adrenal hyperplasia

Graves, L. E., van Dijk, E. B., Zhu, E., Koyyalamudi, S. R., Wotton, T., Sung, D., Srinivasan, S., Ginn, S. L., & Alexander, I. E. (2024) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2024.101232 · Cited by 13

Insights into Prime Editing Technology: A Deep Dive into Fundamentals, Potentials, and Challenges

Hosseini, S. Y., Mallick, R., Mäkinen, P., & Ylä‐Herttuala, S. (2024) · Human Gene Therapy · doi:10.1089/hum.2024.043 · Cited by 12

Characterization of diverse Cas9 orthologs for genome and epigenome editing

Butterfield, G. L., Rohm, D., Roberts, A., Nethery, M. A., Rizzo, A. J., Morone, D. J., Garnier, L., Iglesias, N., Barrangou, R., & Gersbach, C. A. (2025) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.2417674122 · Cited by 14

In vivo delivery systems for CRISPR genome editing: Viral and non-viral carriers

Lee, Y., Kim, B., Lee, D., Cheon, S. Y., Lim, S. G., Kim, Y., & Koo, H. (2025) · Applied Physics Reviews · doi:10.1063/5.0228874 · Cited by 13

The history, use, and challenges of therapeutic somatic cell and germline gene editing

Ryu, J., Adashi, E. Y., & Hennebold, J. D. (2023) · Fertility and Sterility · doi:10.1016/j.fertnstert.2023.02.040 · Cited by 12

Advances in Therapeutic Applications of CRISPR Genome Editing for Spinal Pain Management

Kang, C. Y., Been, K. W., Kang, M. R., Choi, M. S., Kang, R. H., Hur, J. W., & Hur, J. K. (2025) · Neurospine · doi:10.14245/ns.2550462.231 · Cited by 15

CRISPR/Cas9-Mediated Gene Editing in Porcine Models for Medical Research

Gao, M., Zhu, X., Yang, G., Bao, J., & Bu, H. (2021) · DNA and Cell Biology · doi:10.1089/dna.2020.6474 · Cited by 10

CRISPR/Cas-based Human T cell Engineering: Basic Research and Clinical Application

Bernard, B. E., Landmann, E., Jeker, L. T., & Schumann, K. (2022) · Immunology Letters · doi:10.1016/j.imlet.2022.03.005 · Cited by 12

rAAV expressing a COBRA-designed influenza hemagglutinin generates a protective and durable adaptive immune response with a single dose

Wiggins, K., Winston, S. M., Reeves, I. L., Gaevert, J. A., Spence, Y., Brimble, M. A., Livingston, B., Morton, C. L., Thomas, P. G., Sant, A. J., et al. (2024) · Journal of Virology · doi:10.1128/jvi.00781-24 · Cited by 10

In Vivo Gene Editing in Lipid and Atherosclerosis Research

Giorgi, M. D., Jarrett, K. E., Vallim, T. Q. D. A., & Lagor, W. R. (2022) · Methods in molecular biology · doi:10.1007/978-1-0716-1924-7_42 · Cited by 10

Hepatitis B virus: promising drug targets and therapeutic implications

Maepa, M. B., Bloom, K. M., Ely, A., & Arbuthnot, P. B. (2021) · Expert Opinion on Therapeutic Targets · doi:10.1080/14728222.2021.1915990 · Cited by 10

Integrating Biomaterials and Genome Editing Approaches to Advance Biomedical Science

Abdeen, A. A., Cosgrove, B. D., Gersbach, C. A., & Saha, K. (2021) · Annual Review of Biomedical Engineering · doi:10.1146/annurev-bioeng-122019-121602 · Cited by 11

Generation of allogeneic and xenogeneic functional muscle stem cells for intramuscular transplantation

Lenardič, A., Domenig, S. A., Zvick, J., Bundschuh, N., Tarnowska-Sengül, M., Furrer, R., Noé, F., Trautmann, C. L., Ghosh, A., Bacchin, G., et al. (2024) · Journal of Clinical Investigation · doi:10.1172/jci166998 · Cited by 9

Genome editing strategies for targeted correction of β-globin mutation in sickle cell disease: From bench to bedside

Butt, H., Sathish, S., London, E., Johnson, T. L., Essawi, K., Leonard, A. K., Tisdale, J. F., & Demirci, S. (2025) · Molecular Therapy · doi:10.1016/j.ymthe.2025.03.047 · Cited by 12

Gene regulatory and gene editing tools and their applications for retinal diseases and neuroprotection: From proof-of-concept to clinical trial

Altay, H. Y., Özdemir, F., Afghah, F., Kilinç, Z., Ahmadian, M., Tschopp, M., & Ağca, C. (2022) · Frontiers in Neuroscience · doi:10.3389/fnins.2022.924917 · Cited by 9

Temporal restriction of Cas9 expression improves CRISPR-mediated deletion efficacy and fidelity

Weber, J. A., Lang, J. F., Carrell, E. M., Alameh, M., & Davidson, B. L. (2024) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2024.102172 · Cited by 10

CRISPR-Cas9 Targeting PCSK9: A Promising Therapeutic Approach for Atherosclerosis

Gu, B., Li, M., Li, D., & Huang, K. (2025) · Journal of Cardiovascular Translational Research · doi:10.1007/s12265-024-10587-7 · Cited by 13

Liver gene transfer for metabolite detoxification in inherited metabolic diseases

D’Alessio, A. M., Boffa, I., Stefano, L. D., Soria, L. R., & Brunetti‐Pierri, N. (2024) · FEBS Letters · doi:10.1002/1873-3468.14957 · Cited by 8

CRISPR/Cas and Hepatitis B Therapy: Technological Advances and Practical Barriers

Kostyushev, D. S., Kostyusheva, A., Ponomareva, N. I., Brezgin, S., & Chulanov, V. (2021) · Nucleic Acid Therapeutics · doi:10.1089/nat.2021.0075 · Cited by 8

Lipids and lipid derivatives for delivery of the CRISPR/Cas9 system

Madhi, Z. S., Shallan, M. A., Almaamuri, A. M., Alhussainy, A. A., AL-Salih, S. S. H., Raheem, A. K., Alwan, H. J., & Jalil, A. T. (2022) · Journal of Drug Delivery Science and Technology · doi:10.1016/j.jddst.2022.103948 · Cited by 8

Transgenerational Epigenetic DNA Methylation Editing and Human Disease

Tompkins, J. D. (2023) · Biomolecules · doi:10.3390/biom13121684 · Cited by 13

AAV2 delivery of the saCas9 gene results in presentation of an HLA-A∗02:01-restricted T cell epitope potent to induce T cell cytotoxicity

Najera, S. S., Nicastri, A., Bing, S. J., Sajib, A. M., Ternette, N., & Mazor, R. (2025) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2025.101506 · Cited by 7

Immunogenicity of Therapeutic Biological Modalities - Lessons from Hemophilia A Therapies

Nguyen, N. H., Jarvi, N. L., & Balu‐Iyer, S. V. (2023) · Journal of Pharmaceutical Sciences · doi:10.1016/j.xphs.2023.05.014 · Cited by 7

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

Genome editing in the human liver: Progress and translational considerations

Ginn, S. L., Christina, S., & Alexander, I. E. (2021) · Progress in molecular biology and translational science · doi:10.1016/bs.pmbts.2021.01.030 · Cited by 7

Multiplexed bioluminescence-mediated tracking of DNA double-strand break repairs in vitro and in vivo

Chien, J. C.-Y., Badr, C. E., & Lai, C. P. (2021) · Nature Protocols · doi:10.1038/s41596-021-00564-8 · Cited by 6

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

Pre-existing immunity does not impair the engraftment of CRISPR-Cas9-edited cells in rhesus macaques conditioned with busulfan or radiation

Essawi, K., Hakami, W., Khan, M. B. N., Martin, R., Zeng, J., Chu, R., Uchida, N., Bonifacino, A. C., Krouse, A. E., Linde, N. S., et al. (2023) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2023.04.004 · Cited by 9

PlmCas12e Utilizes Glu662 to Prevent Cleavage Site Occupation by Positively Charged Residues Before Target Strand Cleavage

Liu, J., & Zhu, L. (2024) · Molecules · doi:10.3390/molecules29215036 · Cited by 5

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

Enabling Precision Medicine with CRISPR-Cas Genome Editing Technology: A Translational Perspective

Ilahibaks, N. F., Hulsbos, M. J., Lei, Z., Vader, P., & Sluijter, J. P. G. (2022) · Advances in experimental medicine and biology · doi:10.1007/978-981-19-5642-3_20 · Cited by 7

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

In vitro gene editing using primary cells derived from Cas9-expressing pigs

Kim, S., No, J.-G., Lee, S., Choi, A., Hyung, N., Lee, J. Y., Kwak, T., Ju, W. S., Lee, J.-Y., Lee, P., et al. (2024) · Journal of Animal Science and Technology · doi:10.5187/jast.2024.e77 · Cited by 6

CRISPR-SCReT (CRISPR-Stop Codon Read Through) method to control Cas9 expression for gene editing

Yaméogo, P., Duchêne, B. L., Majeau, N., & Tremblay, J. P. (2021) · Gene Therapy · doi:10.1038/s41434-021-00297-z · Cited by 6

CRISPR/Cas9 Editing: Sparking Discussion on Safety in Light of the Need for New Therapeutics

Carlaw, T., Zhang, L., & Ross, C. J. (2020) · Human Gene Therapy · doi:10.1089/hum.2020.111 · Cited by 7

CRISPR/Cas13-Mediated Inhibition of EBNA1 for Suppression of Epstein–Barr Virus Transcripts and DNA Load in Nasopharyngeal Carcinoma Cells

Lin, L., Lui, W., Ong, C. P., Yau, M. Y. C., Jin, D., & Yuen, K. (2025) · Viruses · doi:10.3390/v17070899 · Cited by 5

Integrase-Deficient Lentiviral Vector as a Platform for Efficient CRISPR/Cas9-Mediated Gene Editing for Mucopolysaccharidosis IVA

Nidhi, F., & Tomatsu, S. (2025) · International Journal of Molecular Sciences · doi:10.3390/ijms26146616 · Cited by 6

CRISPR-Cas editing technologies for viral-mediated gene therapies of human diseases: Mechanisms, progress, and challenges

Kantor, B., Duke, L. C., & Bhide, P. G. (2025) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2025.102786 · Cited by 6

CRISPR Cas-9 for treatment and control of Avian viral diseases: challenges and opportunities

Ullah, A., Khan, M., Akram, M., Abdullah, M., Sayka, S., Iqbal, I., Siddique, F., Arain, M. A., Hassan, F., & Shafi, M. (2024) · World s Poultry Science Journal · doi:10.1080/00439339.2024.2344124 · Cited by 6

Preexisting maternal immunity to AAV but not Cas9 impairs in utero gene editing in mice

Riley, J. S., Luks, V. L., Berkowitz, C. L., Dumitru, A. M., Kus, N. J., Dave, A., Menon, P. V., Paepe, M. E. D., Jain, R., Li, L., et al. (2024) · Journal of Clinical Investigation · doi:10.1172/jci179848 · Cited by 5

When size matters: A novel compact Cas12a variant for in vivo genome editing

Bubeck, F., & Grimm, D. (2024) · PLoS Biology · doi:10.1371/journal.pbio.3002637 · Cited by 5

Single-cut gene therapy in a one-step generated rhesus monkey model of Duchenne muscular dystrophy

Bai, R., Guo, W., Zhang, T., Ren, S., Liu, J., Xiao, P., Zhang, J., Sun, W., Yang, J., Ma, Y., et al. (2025) · Cell Reports Medicine · doi:10.1016/j.xcrm.2025.102037 · Cited by 5

Lung stem cells and therapy for cystic fibrosis

Vaidyanathan, S., McCarra, M. B., & Desai, T. (2021) · European Respiratory Society eBooks · doi:10.1183/2312508x.10010520 · Cited by 5

Intracellular RNase activity dampens zinc finger nuclease-mediated gene editing in hematopoietic stem and progenitor cells

Peterson, C. W., Venkataraman, R., Reddy, S. S., Pande, D., Enstrom, M., Radtke, S., Humbert, O., & Kiem, H. (2021) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2021.11.010 · Cited by 7

Pre-Existing Immunity to a Nucleic Acid Contaminant-Derived Antigen Mediates Transaminitis and Resultant Diminished Transgene Expression in a Mouse Model of Hepatic Recombinant Adeno-Associated Virus-Mediated Gene Transfer

Brimble, M. A., Morton, C. L., Winston, S. M., Reeves, I. L., Spence, Y., Cheng, P.-H., Zhou, J., Nathwani, A. C., Thomas, P. G., Souquette, A. H., et al. (2024) · Human Gene Therapy · doi:10.1089/hum.2023.188 · Cited by 5

Ex vivo gene editing and cell therapy for hereditary tyrosinemia type 1

Ates, I., Stuart, C., Rathbone, T., Barzi, M., He, G., Major, A. M., Shankar, V., Lyman, R. A., Angner, S. S., Mackay, T. F. C., et al. (2024) · Hepatology Communications · doi:10.1097/hc9.0000000000000424 · Cited by 5

CRISPR-Cpf1 system and its applications in animal genome editing

Han, Y., Jia, Z., Xu, K., Li, Y., Lu, S., & Guan, L. (2024) · Molecular Genetics and Genomics · doi:10.1007/s00438-024-02166-x · Cited by 5

Early postnatal expression mitigates immune responses to Cas9 in the murine central nervous system

Duba-Kiss, R., & Hampson, D. R. (2025) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2025.101536 · Cited by 5

A compact base editor rescues AATD-associated liver and lung disease in mouse models

Gao, J., Bamidele, N., Pires-Ferreira, D., Zheng, C., Destefano, A., Cheng, H., Tang, Q., Cao, Y., Xie, J., Gao, G., et al. (2025) · Molecular Therapy · doi:10.1016/j.ymthe.2025.08.040 · Cited by 5

Decoding Undesirable Inflammatory Responses of Nucleic Acid‐Delivering Lipid Nanoparticles

Hu, R., Dou, Y., Li, C., Mu, Y., Ouyang, H., Shen, W., & Zhang, J. (2026) · Advanced Science · doi:10.1002/advs.75548 · Cited by 5

Teaching an old vector new tricks: the surprising versatility of AAV vaccines

Winston, S. M., Wiggins, K., Schultz‐Cherry, S., & Davidoff, A. M. (2025) · Journal of Virology · doi:10.1128/jvi.00730-25 · Cited by 4

Overview of gene editing strategies against HBV

Janetzki, Z. T., McCoullough, L. C., Revill, P. A., & Littlejohn, M. (2026) · Antiviral Research · doi:10.1016/j.antiviral.2026.106355 · Cited by 4

CRISPR–Cas Systems in Human Disease Therapy: Advances, Clinical Applications, Limitations, and Future Directions

Mengistu, G. (2026) · The Journal of Gene Medicine · doi:10.1002/jgm.70091 · Cited by 4

CRISPR in clinical oncology: translational advances from molecular diagnostics to therapeutics

Grigg, S. E., Shembrey, C., Fareh, M., Blombery, P. A., Corn, J. E., Seymour, J. F., & Casan, J. M. L. (2026) · Nature Reviews Clinical Oncology · doi:10.1038/s41571-026-01179-2 · Cited by 4

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

CD4+ T Cells Mediate MHC-Deficient Tumor Rejection and Endothelial Cell Reprogramming

Kim, S. I., Haerr, M. E., Al-Ghezi, M., Chen, C., Zhang, Y., Phipps, J. L., Arora, C., Gribbin, K. P., Kim, G. T., Markosyan, N., et al. (2025) · Cancer Immunology Research · doi:10.1158/2326-6066.cir-24-1342 · Cited by 3

Tailoring Cardiac Synthetic Transcriptional Modulation Towards Precision Medicine

Schoger, E., Lelek, S., Panáková, D., & Zelarayan, L. C. (2022) · Frontiers in Cardiovascular Medicine · doi:10.3389/fcvm.2021.783072 · Cited by 3

Magnetic Forces Enable Control of Biological Processes In Vivo

Bao, G. (2020) · Journal of Applied Mechanics · doi:10.1115/1.4049331 · Cited by 3

CRISPR editing as a therapeutic strategy for Duchenne muscular dystrophy—anti-Cas9 immune response casts its shadow over safety and efficacy

Dowling, J. J. (2022) · Gene Therapy · doi:10.1038/s41434-022-00323-8 · Cited by 3

Transgenic mice for in vivo epigenome editing with CRISPR-based systemsPreprint

Gemberling, M. P., Siklenka, K., Rodriguez, E., Eisinger, K. R. T., Barrera, A., Liu, F., Kantor, A., Li, L., Cigliola, V., Hazlett, M. F., et al. (2021) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/2021.03.08.434430 · Cited by 3

Efficient and durable gene activation by Cas9-mediated epigenome editing in vivoPreprint

Qin, Y., Akbulut, T., Mandraju, R., Connolly, K., Bechill, J., Assadian, F., Shottek, A., Levy, S., Benoit, J. D., & Yamagata, T. (2024) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/2024.05.03.592438 · Cited by 3

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

A Translational Roadmap for Neurological Nonsense Mutation Disorders

Li, J., Zhu, Z., & Xu, S. (2026) · International Journal of Molecular Sciences · doi:10.3390/ijms27031418 · Cited by 2

Anti-Cas9 immunity: A formidable challenge for muscle genome editing

Lagor, W. R. (2021) · Molecular Therapy · doi:10.1016/j.ymthe.2021.12.006 · Cited by 2

Genome editing pig models with elements for controllable gene expression

Jin, Q., Shi, H., Liu, X., Ju, J., Wang, K., & Lai, L. (2023) · Animal Research and One Health · doi:10.1002/aro2.20 · Cited by 2

Genome-Editing Technologies in Crop Improvement

Das, R., Kumar, P., Agrawal, S., Singh, K., Singh, N., Singh, S., Singh, A. K., Rajput, V. D., Shukla, P. K., Minkina, T., et al. (2024) · Interdisciplinary biotechnological advances · doi:10.1007/978-981-99-9338-3_4 · Cited by 2

Expression of Cas9 in a Syngeneic Model of Primary Central Nervous System Lymphoma Induces Intracerebral NK and CD8 T Cell-Mediated Lymphoma Cell Lysis Via Perforin

Montesinos‐Rongen, M., Sánchez-Ruiz, M., Siebert, S., Winter, C., Siebert, R., Brunn, A., & Deckert, M. (2022) · The CRISPR Journal · doi:10.1089/crispr.2022.0049 · Cited by 2

The effect of emerging molecular and genetic therapies on cardiopulmonary disease in Duchenne muscular dystrophy

Mckim, D. A., Cripe, T. P., & Cripe, L. H. (2020) · Pediatric Pulmonology · doi:10.1002/ppul.25079 · Cited by 2

Resurrecting a miniature Cas9 ancestor for genome and epigenome editing

Butterfield, G. L., & Gersbach, C. A. (2025) · Nature Biotechnology · doi:10.1038/s41587-025-02707-8 · Cited by 1

CRISPR-based gene therapy for huntington’s disease: current advances and future prospects

Zaheer, A., AboQuella, N. M., Wadan, A. S., Saad, H. A., Kumar, D., Panjwani, S., Rath, S., & Ahmed, S. I. (2025) · Neurogenetics · doi:10.1007/s10048-025-00858-z · Cited by 2

High-fidelity genome and prime editing enabled by the AI-designed openCRISPR-1

Hwang, H.-Y., Yi, H., Gwon, Y., Jeon, E. J., & Kim, D. (2026) · Genome Medicine · doi:10.1186/s13073-026-01682-2 · Cited by 2

Tissue-specific silencing of synthetic mRNAs by de-targeting elements maps vaccination-competent tissues and allows Cas9 de-immunization

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On-target and off-target activities of CRISPR therapeutics across scales

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‘Hit and run’ therapy averts macular degeneration

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Genome editing technologies

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Cell-Based Delivery Approaches for DNA-Binding Domains to the Central Nervous System

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Immune Responses to Adeno-Associated Virus-Mediated CRISPR Therapy

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Deleting in vivo β-catenin degradation domain in mouse hepatocytes drives hepatocellular carcinoma or hepatoblastoma-like tumorsPreprint

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The effect of emerging therapies on cardiopulmonary disease in Duchenne muscular dystrophyPreprint

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Safety Aspects of Genome Editing

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Design of efficacious somatic cell genome editing strategies for recessive and polygenic diseasesPreprint

Carlson-Stevermer, J., Das, A., Abdeen, A. A., Fiflis, D. N., Grindel, B. I., Akcan, T., Alam, T., Kletzien, H., Kohlenberg, L. K., Goedland, M., et al. (2020) · doi:10.21203/rs.3.rs-90150/v1

Synthetic mRNA Gene Therapies and Hepatotropic Non-viral Vectors for the Treatment of Chronic HBV Infections

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In vivo application of CRISPR/Cas9 mediated gene re-expression in the failing mouse and human myocardium

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Bioethical Decision-Making About Somatic Cell Genome Editing: Sickle-Cell Disease as a Case Study

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Comparative Analysis of Lipid Nanoparticle-Mediated Delivery of CRISPR-Cas9 RNP Versus mRNA/sgRNA for Gene Editing in vitro and in vivoPreprint

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Lipid Nanoparticle-Mediated Delivery of CRISPR-Cas9 for Therapeutic Gene Correction

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Development and Characterization of Viral-Based Gene Editing In Vivo

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Adenovirus-Mediated Somatic Genome Editing of Pten by CRISPR/Cas9 in Mouse Liver in Spite of Cas9-Specific Immune Responses

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A Multifunctional AAV-CRISPR-Cas9 and Its Host Response

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A Self-Deleting AAV-CRISPR System for In Vivo Genome Editing

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