Nanoblades allow high-level genome editing in murine and human organoids

The new frontier of genome engineering with CRISPR-Cas9

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Organoid Models and Applications in Biomedical Research

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Modeling Development and Disease with Organoids

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Functional Repair of CFTR by CRISPR/Cas9 in Intestinal Stem Cell Organoids of Cystic Fibrosis Patients

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Engineering organoids

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Modeling colorectal cancer using CRISPR-Cas9–mediated engineering of human intestinal organoids

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Sequential cancer mutations in cultured human intestinal stem cells

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A functional CFTR assay using primary cystic fibrosis intestinal organoids

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Disease modeling in stem cell-derived 3D organoid systems

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The control of DNA repair by the cell cycle

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Identification of Multipotent Luminal Progenitor Cells in Human Prostate Organoid Cultures

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Organoid culture systems for prostate epithelial and cancer tissue

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Engineered Virus-like Particles for Efficient in Vivo Delivery of Therapeutic Proteins

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Generation of mouse models of myeloid malignancy with combinatorial genetic lesions using CRISPR-Cas9 genome editing

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Efficient ablation of genes in human hematopoietic stem and effector cells using CRISPR/Cas9

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Use of CRISPR-modified human stem cell organoids to study the origin of mutational signatures in cancer

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Glioblastoma Model Using Human Cerebral Organoids

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Efficient genetic engineering of human intestinal organoids using electroporation

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Genome Editing in Primary Cells and in Vivo Using Viral-Derived Nanoblades Loaded with Cas9-SgRNA Ribonucleoproteins

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Fast and efficient generation of knock-in human organoids using homology-independent CRISPR–Cas9 precision genome editing

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Probing the Tumor Suppressor Function of BAP1 in CRISPR-Engineered Human Liver Organoids

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Stable transduction of quiescent T cells without induction of cycle progression by a novel lentiviral vector pseudotyped with measles virus glycoproteins

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Baboon envelope pseudotyped LVs outperform VSV-G-LVs for gene transfer into early-cytokine-stimulated and resting HSCs.

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Establishment of human fetal hepatocyte organoids and CRISPR–Cas9-based gene knockin and knockout in organoid cultures from human liver

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Evaluating CRISPR-based prime editing for cancer modeling and CFTR repair in organoids

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A systematic review and meta-analysis of gene therapy with hematopoietic stem and progenitor cells for monogenic disorders

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Protocol for Application, Standardization and Validation of the Forskolin-Induced Swelling Assay in Cystic Fibrosis Human Colon Organoids

Vonk, A. M., van Mourik, P., Ramalho, A. S., Silva, I. A. L., Statia, M., Kruisselbrink, E., Suen, S. W. F., Dekkers, J. F., Vleggaar, F. P., Houwen, R. H. J., et al. (2020) · STAR Protocols · doi:10.1016/j.xpro.2020.100019 · Cited by 128 · see citing works

Assessing the origin of high-grade serous ovarian cancer using CRISPR-modification of mouse organoids

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Efficient gene transfer into human primary blood lymphocytes by surface-engineered lentiviral vectors that display a T cell–activating polypeptide

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rAAV-CFTRΔR Rescues the Cystic Fibrosis Phenotype in Human Intestinal Organoids and Cystic Fibrosis Mice

Vidović, D., Carlon, M. S., da Cunha, M. F., Dekkers, J. F., Hollenhorst, M. I., Bijvelds, M. J. C., Ramalho, A. S., Van den Haute, C., Ferrante, M., Baekelandt, V., et al. (2016) · American Journal of Respiratory and Critical Care Medicine · doi:10.1164/rccm.201505-0914oc · Cited by 74 · see citing works

A functional genetic toolbox for human tissue-derived organoids

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Strategies for genetic manipulation of adult stem cell-derived organoids

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Baboon envelope pseudotyped lentiviral vectors efficiently transduce human B cells and allow active factor IX B cell secretion in vivo in NOD/SCIDγc‐/‐ mice

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Dissecting cell-type-specific roles of androgen receptor in prostate homeostasis and regeneration through lineage tracing

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Baboon Envelope Pseudotyped “Nanoblades” Carrying Cas9/gRNA Complexes Allow Efficient Genome Editing in Human T, B, and CD34+ Cells and Knock-in of AAV6-Encoded Donor DNA in CD34+ Cells

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FDA-approved drug screening in patient-derived organoids demonstrates potential of drug repurposing for rare cystic fibrosis genotypes

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Baboon envelope LVs efficiently transduced human adult, fetal, and progenitor T cells and corrected SCID-X1 T-cell deficiency

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Tubuloid culture enables long-term expansion of functional human kidney tubule epithelium from iPSC-derived organoids

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Identification of novel human Wnt target genes using adult endodermal tissue-derived organoids

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Phenotyping of Rare CFTR Mutations Reveals Distinct Trafficking and Functional Defects

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Therapeutic correction of hemophilia A using 2D endothelial cells and multicellular 3D organoids derived from CRISPR/Cas9-engineered patient iPSCs

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Generation of Human Embryonic Stem Cell-Derived Lung Organoids for Modeling Infection and Replication Differences between Human Adenovirus Types 3 and 55 and Evaluating Potential Antiviral Drugs

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Light-stimulated insulin secretion from pancreatic islet-like organoids derived from human pluripotent stem cells

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Model of the cAMP activation of chloride transport by CFTR channel and the mechanism of potentiators

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A protocol for efficient CRISPR-Cas9-mediated knock-in in colorectal cancer patient-derived organoids

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Rapid establishment of human colonic organoid knockout lines

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CRISPR/Cas9 in Gastrointestinal Malignancies

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Organoid Easytag: an efficient workflow for gene targeting in human organoids

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Targeted nonviral delivery of genome editors in vivo

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Basic Guide for Approaching Drug Delivery with Extracellular Vesicles

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Alveolar Epithelial Cell Dysfunction in Acute Respiratory Distress Syndrome: Mechanistic Insights and Targeted Interventions

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Organoids as powerful models of endometrium epithelium in transcriptomic, cellular and functional mimicry

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Organoids: An Emerging Precision Medicine Model for Prostate Cancer Research

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Protocol to create isogenic disease models from adult stem cell-derived organoids using next-generation CRISPR tools

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Organoids in cancer therapies: a comprehensive review

Xin-Yi, J., Yan-Ran, W., Pin-Ru, D., Shi-Yi, Q., & Jiang, H. (2025) · Frontiers in Bioengineering and Biotechnology · doi:10.3389/fbioe.2025.1607488 · Cited by 15 · see citing works

Trojan Horse-Like Vehicles for CRISPR-Cas Delivery: Engineering Extracellular Vesicles and Virus-Like Particles for Precision Gene Editing in Cystic Fibrosis

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Organoid modeling meets cancers of female reproductive tract

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Recent advances and applications of human lung alveolar organoids

Kim, S. K., Sung, E., & Lim, K. (2024) · Molecules and Cells · doi:10.1016/j.mocell.2024.100140 · Cited by 9 · see citing works

Gene Editing by Ferrying of CRISPR/Cas Ribonucleoprotein Complexes in Enveloped Virus-Derived Particles

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DNA-PKcs inhibition improves sequential gene insertion of the full-length CFTR cDNA in airway stem cells

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Ligand-modified rAAV6 vectors with nanoblades allow high-level gene knockin in HSPCs without compromising cell survival

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Gene editing in hematopoietic stem cells by co-delivery of Cas9/sgRNA ribonucleoprotein and templates for homology-directed repair in ‘all-in-one’ lentivirus-derived nanoparticles

Andersen, S., Wolff, J. H., Skov, T. W., Janns, J. H., Davis, L., Haldrup, J., Haslund, D., Revenfeld, A. L., Relkovic, D., Møller, B. K., et al. (2025) · Nucleic Acids Research · doi:10.1093/nar/gkaf767 · Cited by 7 · see citing works

Base and Prime Editing for Inherited Retinal Diseases: Delivery Platforms, Safety, Efficacy, and Translational Perspectives

Zhang, H., Li, Y., Li, J., Li, X., & Li, T. (2025) · Pharmaceutics · doi:10.3390/pharmaceutics17111405 · Cited by 7 · see citing works

Endocrine cancer organoids in basic and translational medical research

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Advances in Engineered Virus-Like Particles for Genome Editing and Therapy

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Organoids and organ‐on‐a‐chip models for investigating the pathophysiology of the human reproductive system

Zhang, H.-B., Deng, S., Jin, F. G., Qu, J., Fan, X., Chen, P., & Fan, X. (2026) · Interdisciplinary medicine · doi:10.1002/inmd.70094 · Cited by 4 · see citing works

Organoids: From Bench to Bedside Applications

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Recent advances of ultrasound-based nano-delivery systems in prostate cancer imaging and treatment

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Ingénierie rétrovirale pour la modification des génomes

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Protein Nanocages as Versatile Vectors for Nucleic Acid Delivery: Main Systems and Their Loading Mechanisms

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CRISPR decodes the RNA regulatory network in prostate cancer: A review from mechanisms to precision therapeutics

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Modeling Alzheimer’s disease with brain organoids: mechanisms, applications, and future directions

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Advances in organoids for personalized medicine: from technological development to clinical application

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Nanoblades allow high-level genome editing in organoidsPreprint

Tirolle, V., Krug, A., Bokobza, E., Bulcaen, M., Ensinck, M. M., Geurts, M. H., Hendriks, D. F. G., Vermeulen, F. L., Larbret, F., Gutiérrez-Guerrero, A., et al. (2022) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/2022.08.04.502859 · Cited by 1 · see citing works

Baboon Envelope Pseudotyped “Nanoblades” Carrying Cas9/gRNA Complexes Allow Efficient Genome Editing in Human T, B, and CD34+ Cells and Knock-in of AAV6-Encoded Donor DNA in CD34+ Cells

Gutiérrez-Guerrero, A., Abrey-Recalde, M. J., Mangeot, P. E., Costa, C., Bernadin, O., Perian, S., Fusil, F., Froment, G., Martinez-Turtos, A., Krug, A., et al. (2021) · Frontiers in Genome Editing · doi:10.3389/fgeed.2021.604371 · Cited by 51 · see citing works

Genome Editing in Primary Cells and in Vivo Using Viral-Derived Nanoblades Loaded with Cas9-SgRNA Ribonucleoproteins

Mangeot, P. E., Risson, V., Fusil, F., Marnef, A., Laurent, E., Blin, J., Mournetas, V., Massouridès, E., Sohier, T. J. M., Corbin, A., et al. (2019) · Nature Communications · doi:10.1038/s41467-018-07845-z · Cited by 346 · see citing works

Site Specific Knock-In Genome Editing in Human HSCs Using Baboon Envelope gp Pseudotypedviral Derived “Nanoblades” Loaded with Cas9/sgRNA Combined with Donor Encoding AAV-6

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