Choice of template delivery mitigates the genotoxic risk and adverse impact of editing in human hematopoietic stem cells

Cited by (133)

Items 101–133 of 133

1. In-depth characterization of stem cell potency and genotoxicity for clinical-scale ex vivo CRISPR-Cas9 gene editing
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2. Unlocking the curative potential of gene transfer and editing for hematopoietic disorders
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3. Single-Stranded DNA with Internal Base Modifications Mediates Highly Efficient Gene Insertion in Primary Cells
   Karen L Kanke, Rachael E. Rayner, Eli Abel, Aparna Venugopalan, Ma Suu, Jacob T. Stack, Reza Nouri, Gongbo Guo, Tatyana A. Vetter, Estelle Cormet‐Boyaka et al. · 2024 · bioRxiv (Cold Spring Harbor Laboratory)
4. Circularization of Single-Stranded DNA Donor Template Unleashes the Power of Non-Viral Gene Delivery for Long-Term HSCs editing
   Gil Letort, Aymeric Duclert, Diane Le Clerre, Isabelle Chion-Sotinel, Roger Salvatori, Emilie Dessez, Margaux Sevin, Marco C. Rotondi, Cosimo Ducani, Philippe N. Duchateau et al. · 2025 · bioRxiv (Cold Spring Harbor Laboratory)
5. ATP2B1 expression identifies human hematopoietic stem cells across ontogeny with superior repopulation and self-renewal capacity
   Angelica Varesi, Murtaza S. Nagree, Isabella Di Biasio, Andy G.X. Zeng, Sayyam Shah, Hyerin Kim, Michael Zhang, Alex Murison, John Edgar Dick, Stephanie Zhi-Juan Xie · 2025 · bioRxiv (Cold Spring Harbor Laboratory)
6. p38 MAPK fuels proliferation stress and DNA damage impairing the functionality of genetically engineered hematopoietic stem and progenitor cells
   Raffaella Di Micco, Lucrezia della Volpe, Roberta Vacca, Federico Midena, Teresa Tavella, Matteo Maria Naldini, Matteo Barcella, Kety Giannetti, Samuele Ferrari, Stefano Beretta et al. · 2023 · Research Square
7. Functional Screening in human HSPCs identifies optimized protein-based enhancers of Homology Directed Repair
   Juan A. Pérez-Bermejo, Oghene Efagene, William M. Matern, Jeffrey K. Holden, Shaheen Kabir, Glen M. Chew, Gaia Andreoletti, Eniola Catton, Craig L. Ennis, Angelica Garcia et al. · 2023 · bioRxiv (Cold Spring Harbor Laboratory)
8. Understanding genetic heterogeneity in gene-edited hematopoietic stem cell products
   H. Becker, Satoshi Yamazaki · 2023 · Experimental Hematology
9. Gene Editing Approaches for Haematological Disorders
   Samuele Ferrari, Daniele Canarutto · 2024 · Elsevier eBooks
10. Improving efficacy of in vivo therapy of sickle cell disease by hijacking natural biology of hematopoietic stem cells
    Jia Yao, Dmitry M. Shayakhmetov · 2024 · Molecular Therapy
11. Protocol for optimizing culture conditions for ex vivo activation during CRISPR-Cas9 gene editing in human hematopoietic stem and progenitor cells
    Lucrezia della Volpe, Roberta Vacca, Raffaella Di Micco · 2025 · STAR Protocols
12. Recent Advances in Gene Editing Cargo Delivery for Hematopoietic Stem and Progenitor Cells
    Yuxiang Tang · 2025 · Theoretical and Natural Science
13. In vivo production of an anti-HIV antibody from primate hematopoietic cells by non-viral knock-in
    Jack M.P. Castelli, Katrina Poljakov, Youngseo Jwa, Molly E. Cassidy, Matthew D. Gray, Jocelyn N. Sanchez Gaytan, Mark Enstrom, Jonathan D. Linton, Anthony Rongvaux, Justin J. Taylor et al. · 2025 · bioRxiv (Cold Spring Harbor Laboratory)
14. REpair of heterozygous Mutations independent of Exogenous Donor template with high efficiency (REMEDY) using allele specific CRISPR targeting and HDR enhancers
    Yasemin Sezgin, Genesis Snyder, Noushin Saljoughian, Colin T. Maguire, Ebru Erzurumluoğlu Gökalp, Devi Jaganathan, Eleonora Silvana D'Ambrosio, Burçak Özeş, Gregory L. Wheeler, Ben Kelly et al. · 2025 · bioRxiv (Cold Spring Harbor Laboratory)
15. Have we really solved rAAV6 toxicity? A closer look at mannose-coupled rAAV6 vectors
    Denise Klatt, Christian Brendel · 2025 · Molecular Therapy — Nucleic Acids
16. Base editing of β 0 thalassemia mutations as a therapeutic strategy for β-hemoglobinopathies: efficacy and genotoxicity studies
    Giulia Hardouin, Pierre Martinucci, Samantha Scaramuzza, Panagiotis Antoniou, Federico Corradi, Alexandra Tachtsidi, Guillaume Corre, Margaux Mombled, Sandra Manceau, Cécile Rouillon et al. · 2025 · bioRxiv (Cold Spring Harbor Laboratory)
17. A modified cyclosporine enhances lentivector transduction ex vivo and in vivo by degrading IFITM3
    Dara Annett, Bethan J. Critchley, Kate L. Morling, Ben Graham, Valeria Pingitore, Thomas E. Whittaker, Nada Kurdi, J.M. Warne, Shefta E. Moula, Diego León‐Rico et al. · 2026 · Molecular Therapy
18. Gene Therapy for Inborn Errors of Immunity
    Soraia P. Caetano-Silva, Claire Booth · 2025 · Wiley eBooks
19. In-Depth Characterization of Stem Cell Potency and Genotoxicity for Clinical-Scale Ex Vivo CRISPR/Cas9 Gene Editing
    Asma Naseem, Winston Vetharoy, Thomas E. Whittaker, Federica Zinghirino, R Ghosh, Eduardo Gómez‐Castañeda, Hyeon-Ju Ali, Carlo Cipriani, Eugenio Montini, Adrian James Thrasher et al. · 2026 · bioRxiv (Cold Spring Harbor Laboratory)
20. Therapeutic Genome Engineering for the Treatment of Sickle Cell Disease
    Alexis Kristine Leonard, Akshay Sharma, Fang-Yun Chao, Jonathan S. Yen · 2026 · Springer eBooks
21. Selector adeno-associated viral vectors facilitate on-target precise genome editing and purge off-target chromosomal insertions
    Zhen Li, Xiaoling Wang, Jin Liu, Josephine M. Janssen, Rob C. Hoeben, Manuel A.F.V. Gonçalves · 2026 · Trends in biotechnology
22. Ex vivo gene therapy for β-hemoglobinpathies and metabolic disorders
    Marine Laurent · 2023 · theses.fr (ABES)
23. CRISPR-Cas9-based strategies for enhanced targeted integration
    Laurie Lacombe · 2024 · theses.fr (ABES)
24. Synthetic Biology for Diagnosis and Therapy of Genetic Disorders
    Devangi Zala, Shuvomoy Banerjee, Ansh Mehta, Anand K. Tiwari · 2026 · Springer eBooks
25. Research progress and future perspectives on in vivo CAR-T vector delivery technology
    Kuo Yu, Zhen-Kun WANG · 2026 · Shengming kexue
26. Gene editing of hematopoietic stem cells: applications and advances
    Qi Li, Hongxia Wang, Y He, Genyue Hong, Guoxiu Bao · 2026 · International Journal of Hematology
27. Targeted lipid nanoparticles unlock in vivo human haematopoietic stem cell gene editing
    Zhangyi Luo, Audrey T. Zhu, Michael J. Mitchell · 2026 · Nature Biomedical Engineering
28. Targeted genomic integration and rearrangement using prime assembly
    Sébastien Levesque, Nozomu Kawashima, Gue‐Ho Hwang, Jing Zeng, Vasil Toskov, Timothy Barry, William P. Mannherz, Luke Homfeldt, Basheer Becerra, Vivien A. C. Schoonenberg et al. · 2026 · Nature
29. Targeting white matter astrocytes with a Gfap-disruptive Cas9 nuclease benefits a murine model of Alexander disease
    Anne E.J. Hillen, Irene Capuano, Barbara Gega, Rossana Norata, Margaux Mombled, Ilaria Laface, Giada Zambonini, Andrea Lomagno, Marine Rouillon, Serena Gea Giannelli et al. · 2026 · Molecular Therapy
30. Sickle cell disease-specific barriers to in vivo hematopoietic stem cell gene therapy
    Rachel Choi, Vivien Sheehan · 2026 · Seminars in Hematology
31. Prime editing of human hematopoietic stem cells for correction of GATA2 deficiency
    Jonas Holst Wolff, Thomas Wisbech Skov, Sofie Rahbek Dorset, Janni Sjelborg, Sujan Ravendran, Anne Louise Revenfeld, Didde Haslund, Anaïs Marie Julie Møller, Mette Holm, Martin Kristian Thomsen et al. · 2026 · Science Translational Medicine
32. Targeted insertion of partial CFTR cDNA replicates native CFTR expression with restored channel function in CF airway epithelium
    C. Barillà, Shingo Suzuki, So Hyun Park, D. Nalluri, Hui Wen, Mohamed A. Hanafy, Daniel Betancourth, Ayushi Umrigar, Shih‐Hsing Leir, Katreya Lovrenert et al. · 2026 · Molecular Therapy — Nucleic Acids
33. Outcomes and challenges of stem cell transplantation in patients with ribosomopathies
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