CRISPR/Cas9 genome editing in human hematopoietic stem cells.

Bak, Rasmus O, Dever, Daniel P, Porteus, Matthew H·2018·Nature Protocols

Cited by (318)

Items 201–300 of 318

  1. Engineered human Diamond-Blackfan anemia disease model confirms therapeutic effects of clinically applicable lentiviral vector at single-cell resolution
    Yang Liu, Ludwig Schmiderer, Martin Hjort, Stefan Lang, Tyra Bremborg, Anna Rydström, Axel Schambach, Jonas Larsson, Stefan Karlsson · 2023 · Haematologica
  2. Comparison of Gene-Editing Approaches for Severe Congenital Neutropenia-Causing Mutations in the ELANE Gene
    Malte Ulrich Ritter, Masoud Nasri, Benjamin Dannenmann, Perihan Mir, Benjamin Secker, Diana Amend, Maksim Klimiankou, Karl Welte, Julia Skokowa · 2024 · The CRISPR Journal
  3. 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
    Sofie Andersen, Jonas Holst Wolff, Thomas Wisbech Skov, Jacob Hørlück Janns, Luther Davis, Jakob Haldrup, Didde Haslund, Anne Louise Revenfeld, Dinko Relkovic, Bjarne Kuno Møller et al. · 2025 · Nucleic Acids Research
  4. Gene regulation in inborn errors of immunity: Implications for gene therapy design and efficacy
    Hana Y. Ghanim, Matthew Hebden Porteus · 2024 · Immunological Reviews
  5. CRISPR-Cas9 Genomic Editing as an Innovation in the Management of Sickle Cell Disease: A Systematic Review
    Aloysius Obinna Ikwuka, Solomon Musa, Francis Chigozie Udeh, Abdullahi Adobanyi Musa, Ugo Collins Chukwuezie · 2023 · American Journal of Medical Science and Innovation
  6. Haploinsufficiency at the CX3CR1 locus of hematopoietic stem cells favors the appearance of microglia-like cells in the central nervous system of transplant recipients
    Annita Montepeloso, Davide Mattioli, Danilo Pellin, Marco Peviani, Pietro Genovese, Alessandra Biffi · 2024 · Nature Communications
  7. Targeted gene editing and near-universal cDNA insertion of CYBA and CYBB as a treatment for chronic granulomatous disease
    Jonas Holst Wolff, Thomas Wisbech Skov, Didde Haslund, Sofie Rahbek Dorset, Anne Louise Revenfeld, Clotilde Aussel, Sofie E. Jørgensen, Mette Holm, Martin Kristian Thomsen, Sandra Ammann et al. · 2025 · Nature Communications
  8. CCR5-targeted allogeneic gamma–delta CD19 chimeric antigen receptor T cells for HIV-associated B cell-malignancy immunotherapy
    Ángel Ramírez-Fernández, Alexander J. Dimitri, Fang Chen, Robert L. Bartoszek, Gregory M. Chen, Laura Córdoba-Espejo, Yuqi Zhou, Yun‐Hsin Tang, Chien‐Ting Lin, Reyes Acosta et al. · 2025 · Nature Biomedical Engineering
  9. Regulatory T cell dysfunction and immunotherapeutic breakthroughs in type 1 diabetes
    Kuang-Ji Zhou, Shan-Jie Rong, Yue-Chen Liu, Fangxia Sun, Ting Wang, Qi-Lin Yu, Cong-Yi Wang · 2026 · Frontiers in Endocrinology
  10. Modeling of human T cell developmentin vitroas a read-out for hematopoietic stem cell multipotency
    Steven Strubbe, Tom Taghon · 2021 · Biochemical Society Transactions
  11. The Wiskott–Aldrich syndrome protein is required for positive selection during T-cell lineage differentiation
    Melissa Pille, John M. Avila, Guillem Sanchez Sanchez, Glenn Goetgeluk, Stijn De Munter, Hanne Jansen, Lore Billiet, Karin Weening, Haipeng Xue, Sarah Bonte et al. · 2023 · Frontiers in Immunology
  12. Gene Therapies for Sickle Cell Disease
    Salome Bwayo Weaver, Divita Singh, Kierra M. Wilson · 2024 · Journal of Pharmacy Technology
  13. CRISPR/nCas9-Edited CD34+ Cells Rescue Mucopolysaccharidosis IVA Fibroblasts Phenotype
    Angelica María Herreño-Pachón, Andrés Felipe Leal, Shaukat Ali Khan, Carlos Javier Alméciga-Díaz, Shunji Tomatsu · 2025 · International Journal of Molecular Sciences
  14. Genome Engineering of Hematopoietic Stem Cells Using CRISPR/Cas9 System
    Nivedhitha Devaraju, Vignesh Rajendiran, Nithin Sam Ravi, Kumarasamypet Murugesan Mohankumar · 2022 · Methods in molecular biology
  15. Induced Pluripotent Stem Cells and Genome-Editing Tools in Determining Gene Function and Therapy for Inherited Retinal Disorders
    Daniela Benati, Amy Leung, Pedro R.L. Perdigão, Vasileios Toulis, Jacqueline van der Spuy, Alessandra Recchia · 2022 · International Journal of Molecular Sciences
  16. Engineering Oncogenic Heterozygous Gain-of-Function Mutations in Human Hematopoietic Stem and Progenitor Cells
    Tommaso Sconocchia, Johannes Foßelteder, Thomas Köhnke, Ravindra Majeti, Andreas Reinisch · 2023 · Journal of Visualized Experiments
  17. High-efficiency editing in hematopoietic stem cells and the HUDEP-2 cell line based on in vitro mRNA synthesis
    Nikoletta Y. Papaioannou, Petros Patsali, Basma Naiisseh, Panayiota L. Papasavva, Lola Koniali, Ryo Kurita, Yukio Nakamura, Soteroula Christou, Maria Sitarou, Claudio Mussolino et al. · 2023 · Frontiers in Genome Editing
  18. Homeostatic Role of Decorin in Right Ventricular Pressure Overload and Pulmonary Hypertension Induced Remodeling
    Yohei Akazawa, Sriram Vaidyanathan, Michael Bradley Dewar, Kana Yazaki, John D. Dauz, Osami Honjo, Mei Sun, Golam Kabir, Robert P. Jankov, Scott Patrick Heximer et al. · 2024 · JACC Basic to Translational Science
  19. A detection method for the capture of genomic signatures: From disease diagnosis to genome editing
    Orléna Benamozig, Lou Baudrier, Pierre Billon · 2021 · Methods in enzymology on CD-ROM/Methods in enzymology
  20. αDβ2 as a novel target of experimental polymicrobial sepsis
    Sophia Koutsogiannaki, Lifei Hou, Toshiaki Okuno, Miho Shibamura‐Fujiogi, Hongbo Robert Luo, Koichi Yuki · 2022 · Frontiers in Immunology
  21. Engineering Oncogenic Heterozygous Gain-of-Function Mutations in Human Hematopoietic Stem and Progenitor Cells
    Tommaso Sconocchia, Johannes Foßelteder, Thomas Köhnke, Ravindra Majeti, Andreas Reinisch · 2023 · Journal of Visualized Experiments
  22. Using human genetics to develop strategies to increase erythropoietic output from genome-edited hematopoietic stem and progenitor cells
    Sofia E. Luna, Joab Camarena, Jessica P. Hampton, Kiran R. Majeti, Carsten T. Charlesworth, Eric Soupène, Sridhar Selvaraj, Kun Jia, Vivien Sheehan, M. Kyle Cromer et al. · 2023 · bioRxiv (Cold Spring Harbor Laboratory)
  23. Refined DNA repair manipulation enables a universal knock-in strategy in mouse embryos
    Hongyu Chen, Qingtong Tan, Li Li, Lanxin Li, Jiqiang Fu, Wencheng Zhu, Jie Li, Yining Wang, Shiyan Li, Huimin Li et al. · 2025 · Nature Communications
  24. In situ gene editing of hematopoietic stem cells via AAV-delivered CRISPR guide RNAs
    Alborz Karimzadeh, Rebekah Kim, Vivian Garcia, Michael Florea, Bryan L. Peacker, Shio Kobayashi, Drake Watkins, Kathleen A. Messemer, Jing Zeng, Daniel E. Bauer et al. · 2025 · Blood Advances
  25. RNA modification-mediated aberrant alternative splicing: An underlying driver of disease pathogenesis
    Jin Zeng, Li‐Ting Diao, Shu‐Juan Xie, Zhen‐Dong Xiao · 2025 · Cell investigation.
  26. Tools and delivery technologies for mitochondrial gene editing
    Nei Li, Bowen Wu, Yi Xiao, Yijia Hao, Fan Wu, Yinghui Wei, Ying Xu, Xuexiang Han · 2025 · Cell Biomaterials
  27. MiR-29b inhibits COC expansion and oocyte in vitro maturation via induction of ROS by targeting CYCS
    Xiaoshu Zhan, Nanshan Qi, Derek Toms, Renee Freiburger, Lauren Fletcher, Bingyun Wang, Julang Li · 2024 · Animal Reproduction Science
  28. Failure of metabolic checkpoint control during late-stage granulopoiesis drives neutropenia in reticular dysgenesis
    Wenqing Wang, Martin Arreola Villanueva, Thomas P. Mathews, Andrew DeVilbiss, Zhiyu Zhao, Misty S. Martin-Sandoval, Abdulvasey Mohammed, Giorgia Benegiamo, Avni Awani, Ludger J.E. Goeminne et al. · 2024 · Blood
  29. Genes as Medicine
    Joseph D. Long, Edward C. Trope, Jennifer Yang, Kristen Rector, Caroline Y. Kuo · 2022 · Hematology/Oncology Clinics of North America
  30. The Tetraspanin Protein CD9 Modulates Infection with Human Herpesvirus 6A and 6B in a CD46-Dependent Manner
    Vivien Rodacker Schack, Litten Sørensen Rossen, Clara Christina Ekebjærg, Katrine Kyd Holstein Thuesen, Bettina B. Bundgaard, Per Höllsberg · 2021 · Journal of Virology
  31. The CRISPR Revolution in the Drug Discovery Workflow: An Industry Perspective
    Annalisa Lattanzi, Danilo Maddalo · 2022 · The CRISPR Journal
  32. Highly Efficient Repair of the ΔF508 Mutation in Airway Stem Cells of Cystic Fibrosis Patients with Functional Rescue of the Differentiated Epithelia
    Sriram Vaidyanathan, Ameen Abdulla Salahudeen, Zachary M. Sellers, Dawn T. Bravo, S. S. Choi, Arpit Batish, Wei Le, Sean de la O, Milan P. Kaushik, Noah Galper et al. · 2019 · bioRxiv (Cold Spring Harbor Laboratory)
  33. ZNF410 uniquely activates the NuRD component CHD4 to silence fetal hemoglobin expression
    Xianjiang Lan, Ren Ren, Ruopeng Feng, Lana C. Ly, Yemin Lan, Zhe Zhang, Nicholas G. Aboreden, Kunhua Qin, John R. Horton, Jeremy D. Grevet et al. · 2020 · bioRxiv (Cold Spring Harbor Laboratory)
  34. Gene therapy for inborn errors of immunity: past progress, current status and future directions
    R. W. Torrance, Katharine Orf, Thomas Andrew Fox · 2025 · Rare Disease and Orphan Drugs Journal
  35. Genetically heterozygous - transcriptionally homozygous IRF7 deficiency underlies herpesvirus CNS infections in humans
    Marvin Werner, Jonas Holst Wolff, Justin D. Taft, Thomas Zillinger, Daniëla Maria Hinke, Toke Seierøe Barfod, Kerstin De Keukeleere, Franziska Winzig, Bettina B. Bundgaard, Ryo Narita et al. · 2025 · The Journal of Experimental Medicine
  36. Assessing mRNA and sgRNA Quality for Cell and Gene Therapy Applications Using Nanopore Direct RNA Sequencing
    Kamalakar Chatla, Brian Roper, Luladey Ayalew, Peggy Ko, Steffen Lippold, Meenakshi K. Doma, Julien Camperi · 2026 · Analytical Chemistry
  37. Targeted gene therapy into a safe harbor site in human hematopoietic progenitor cells
    Fátima Rodríguez-Fornés, Oscar Quintana Bustamante, M. Luz Lozano, Jose Carlos Segovia, Juan Antonio Bueren, Guillermo Güenechea · 2020 · Gene Therapy
  38. Near-perfect precise on-target editing of human hematopoietic stem and progenitor cells
    Fanny-Meï Cloarec-Ung, Jamie Beaulieu, Arunan Suthananthan, Bernhard Lehnertz, Guy Sauvageau, Hilary M. Sheppard, David J. H. F. Knapp · 2023 · eLife
  39. Gene-addition/editing therapy in sickle cell disease
    Galia Pollock, Olivier Nègre, Jean‐Antoine Ribeil · 2023 · La Presse Médicale
  40. Advances in pharmacotherapy for sickle cell disease: what is the current state of play?
    Sheinei Alan, Julie Kanter · 2024 · Expert Opinion on Pharmacotherapy
  41. Innovative Platforms in Regenerative Medicine: Bridging Research and Clinical Solutions
    Seyedeh Neda Jalali, Zahra Fathi, Sohameh Mohebbi · 2025 · Jentashapir Journal of Cellular and Molecular Biology
  42. Donor insertion into CX3CR1 allows epigenetic modulation of a constitutive promoter on hematopoietic stem cells and its activation upon myeloid differentiation
    Iris Ramos-Hernández, Carla Fuster‐García, Araceli Aguilar-González, María L Lozano-Vinagre, Guillermo Guenechea-Amurrio, Francisco Jose Sanchez-Luque, Manuel A.F.V. Gonçalves, Toni Cathomen, Pilar Muñoz, Francisco J. Molina-Estévez et al. · 2025 · Nucleic Acids Research
  43. Genome engineering with Cas9 and AAV repair templates, successes and pitfalls
    Marie‐Christine Birling, Yann Hérault, Guillaume Pavlovic · 2025 · Mammalian Genome
  44. Evolution mechanism of scientific topics using inheritance and variation features of knowledge genes: A case study in the hematopoietic stem-cell field
    Haiyun Xu, Ming Ma, Liang Chen, Chao Wang, LO Mallasiy Muhayil Asir · 2025 · Journal of Information Science
  45. Red blood cell–derived transglutaminase 2 influences thrombus formation
    Naoual Ouazzani Chahdi, Judith J. de Vries, Hande Eyisoylu, Hanke L. Matlung, Chi M. Hau, Rienk Nieuwland, Moniek P.M. de Maat, Joost C.M. Meijers, Robin van Bruggen · 2025 · Research and Practice in Thrombosis and Haemostasis
  46. Non-genotoxic transplantation and in vivo selection through epitope editing
    Gabriele Casirati, Andrea Cosentino, Marta Freschi, Jing Zeng, Adele Mucci, Sébastien Levesque, Nola R. Neri, Enrico Drago, Viola Carzaniga, Francesco Romano et al. · 2026 · Nature
  47. Comprehensive UHPLC- and CE-Based Methods for Engineered Cas9 Characterization
    Julien Camperi, Gary Console, Laura Y. Zheng, Nicole M. Stephens, Mary Montti, Brian Roper, Minhua Zheng, Maryam Moshref, Yavuz S. Dagdas, Patrick G. Holder et al. · 2022 · Talanta
  48. CRISPR Gene Editing of Hematopoietic Stem and Progenitor Cells
    Reza Shahbazi, Patricia Lipson, Karthikeya S.V. Gottimukkala, Daniel D. Lane, Jennifer E. Adair · 2022 · Methods in molecular biology
  49. CRISPR: the Janus god of modern science
    Priyanka Khare, Sayanti Mandal, Anil Kumar Verma, Deepak Parashar, Kapilesh Jadhav, Saurabh K. Gupta · 2024 · Elsevier eBooks
  50. A novel study on CXXC5: unraveling its regulatory mechanisms in hematopoietic stem cell biology through proteomics and gene editing
    Shanshan Liu, Yan Gao, Xianqi Feng, Yujie Xu, Minghui Hu, Hairong Fei, Hongying Zheng, Junxia Huang, Tianlan Li, Chunting Zhao et al. · 2024 · Genes & Genomics
  51. Novel humanized loss-of-function NF1 mouse model of juvenile myelomonocytic leukemia
    Roshani Sinha, Rachana Vinay Patil, Rosa Romano, Devesh Sharma, Esmond Lee, Rhonda Perriman, Saori Takeda, Benjamin J. Lesch, Zhenyu Yao, Y. Lucy Liu et al. · 2025 · Blood Advances
  52. Development of Cellular Models to Study Efficiency and Safety of Gene Edition by Homologous Directed Recombination Using the CRISPR/Cas9 System
    Sabina Sánchez-Hernández, Araceli Aguilar-González, Beatriz Guijarro-Albaladejo, Noelia Maldonado‐Pérez, Iris Ramos-Hernández, Marina Cortijo-Gutierréz, Rosario Maria Sanchez-Martin, Karim Benabdellah, Francisco Martı́n · 2020 · Cells
  53. A Review of CRISPR Cas9 for SCA: Treatment Strategies and Could Target β-globin Gene and BCL11A Gene using CRISPR Cas9 Prevent the Patient from Sickle Cell Anemia?
    Bambang Edi Suwito, Arga Setyo Adji, Jordan Steven Widjaja, Syalomitha Claudia Stefanie Angel, Aufar Zimamuz Zaman Al Hajiri, Nanda Fadhilah Witris Salamy, Choirotussanijjah Choirotussanijjah · 2023 · Open Access Macedonian Journal of Medical Sciences
  54. TRACE-Seq Reveals Clonal Reconstitution Dynamics of Gene Targeted Human Hematopoietic Stem Cells
    Rajiv Sharma, Daniel P. Dever, Ciaran M. Lee, Armon Azizi, Yidan Pan, Joab Camarena, Thomas Köhnke, Gang Bao, Matthew Hebden Porteus, Ravindra Majeti · 2020 · bioRxiv (Cold Spring Harbor Laboratory)
  55. Reticular Dysgenesis-associated Adenylate Kinase 2 deficiency causes failure of myelopoiesis through disordered purine metabolism
    Wenqing Wang, Andrew DeVilbiss, Martin Arreola Villanueva, Thomas P. Mathews, Zhiyu Zhao, Misty S. Martin-Sandoval, Giorgia Benegiamo, Avni Awani, Ludger J.E. Goeminne, Daniel P. Dever et al. · 2021 · bioRxiv (Cold Spring Harbor Laboratory)
  56. Near-perfect precise on-target editing of human hematopoietic stem and progenitor cells
    Fanny-Meï Cloarec-Ung, Jamie Beaulieu, Arunan Suthananthan, Bernhard Lehnertz, Guy Sauvageau, Hilary M. Sheppard, David J. H. F. Knapp · 2023 · bioRxiv (Cold Spring Harbor Laboratory)
  57. Knockout studies using CD34+ hematopoietic cells suggest that CD44 is a physiological human neutrophil E-selectin ligand
    Yuqi Zhu, Sriram Neelamegham · 2023 · bioRxiv (Cold Spring Harbor Laboratory)
  58. Engineering hematopoietic stem and progenitor cells to generate red blood cells as viral traps against HIV-1
    Sofia E. Luna, William N. Feist, Kaya Ben-Efraim, Michael A. Pendergast, Freja K. Ekman, Britney N. Hernandez, Megan E. Merritt-Garza, Kathleen Romero, Zak Kostamo, Nelson A. Amorin et al. · 2026 · Molecular Therapy Advances
  59. Knowledge mapping and bibliometric insights into gene therapy for rare inherited hematologic pathologies: focus on sickle cell disease, hemophilia, and thalassemia
    Moutaz W. Sweileh · 2025 · Orphanet Journal of Rare Diseases
  60. Revisiting hemoglobin constant spring: molecular insights, pathophysiological mechanisms, and clinical perspectives
    Narawich Wongkhammul, Pinyaphat Khamphikham, Supawadee Maneekesorn, Pimlak Charoenkwan · 2025 · Orphanet Journal of Rare Diseases
  61. Functional classification of platelet gene variants using CRISPR HDR in CD34+ cell-derived megakaryocytes
    Yasuhiro Kosaka, Brandon Lopez, Nina Y. Kishimoto, Shancy Petsel Jacob, Emilie Montenont, Rodrigo Huallanca, Graeson Coughenour, Jorge Di Paola, Justyne E. Ross, Kristy Lee et al. · 2025 · The American Journal of Human Genetics
  62. Stem cell-based therapies for silicosis: mechanisms, sources, clinical translation, and emerging strategies
    Xinru Feng, Bo Xiao, Lixia Hou, Bingxi Zhang, Li Tian, Biwen Mo, Dong Yao · 2026 · Frontiers in Immunology
  63. Mitochondrial genome editing tools: prospects in animal breeding
    Yan Xu, MingYue Chen, Shunkai Yang, Yuyang Guo, Yichao Dai, Yutong Chen, Haijiang Zhong, Taisen Ma, Dingrui Zha, Yutao He et al. · 2026 · Journal of genetics and genomics/Journal of Genetics and Genomics
  64. A dihydrouracil CRBN ligand mitigates IMiD associated safety liabilities in heterobifunctional targeted protein degrader
    Monica C. Rodrigo-Brenni, Jasper C. Komen, Ghaith M. Hamza, Natacha Bohin, Tomas Adomavicius, Angelo Andres, Stefan Blaho, Ulf Börjesson, Gavin W. Collie, Gian Marco De Donatis et al. · 2026 · Nature Communications
  65. A Novel Strategy to Produce CAR‐γδ T Cells via Site‐Directed Gene Integration by a Combination of CRISPR/Cas9 and AAV
    Ruoyu Dong, Yi-Xi Zhang, Guangxun Yuan, Xiao-Yan Yue, Yingying Ding, Xun Zeng, Hao-Wen Xiao · 2026 · Cancer Medicine
  66. Novel SERPINC1 variants in hereditary antithrombin deficiency: first pathogenic deep-intronic variant, revealed by multiple genomic and transcriptomic approaches
    P Christian Remmelzwaal, René Mulder, Jelkje J. de Boer-Bergsma, Albertus Vinke, Hilde Kooistra, Nic J.G.M. Veeger, Albertus T.J. Wierenga, Jan Jacob Schuringa, Michaël V. Lukens, Karina Meijer · 2026 · Journal of Thrombosis and Haemostasis
  67. Tailoring cells for clinical needs: Meeting report from the Advanced Therapy in Healthcare symposium (October 28–29 2017, Doha, Qatar)
    Sara Deola, Bella Samia Guerrouahen, Heba Sidahmed, Anjud Al-Mohannadi, Muhammad Elnaggar, Ramaz Elsadig, Essam Mohamed Abdelalim, Goran Petrovski, Massimo Gadina, Adrian James Thrasher et al. · 2018 · Journal of Translational Medicine
  68. Plasmid‐Based Donor Templates for Nonviral CRISPR/Cas9‐Mediated Gene Knock‐In in Human T Cells
    Kate Senger, Ilseyar Akhmetzyanova, Benjamin Haley, Sascha Rutz, Soyoung A. Oh · 2022 · Current Protocols
  69. Random Integration Analysis of Recombinant Adeno-Associated Virus 6 Packaged in Sf9 Insect Cells
    Mingyu Zhang, Xinyuan Liu, Cheng Zhang · 2023 · Molecular Biology
  70. Random Integration Analysis of Recombinant Adeno-Associated Virus 6 Packaged in Sf9 Insect Cells
    Mancheng Zhang, Xiaoming Liu, Changsheng Zhang · 2023 · Molecular Biology
  71. Engineered cytokine-expressing MSCs support ex vivo culture of human HSPCs and AML cells
    Johannes Foßelteder, Thomas Brauchart, Angelika Schlacher, Tommaso Sconocchia, Erdem Özkaya, Lisa Auinger, Peter Schlenke, Heinz Sill, Armin Zebisch, Andreas Reinisch · 2025 · Experimental Hematology
  72. Homology mediated end joining enables efficient non-viral targeted integration of large DNA templates in primary human T cells
    Beau R. Webber, Matthew J. Johnson, Nicholas J. Slipek, Walker S. Lahr, Anthony P. DeFeo, Joseph G. Skeate, Xiaohong Qiu, Blaine Rathmann, Miechaleen D. Diers, Bryce J. Wick et al. · 2021 · bioRxiv (Cold Spring Harbor Laboratory)
  73. Les diasporas comme ressources d'intégration dans l'économie mondiale.
    Natalia Buga · 2011
  74. Combining Cell-Intrinsic and -Extrinsic Resistance to HIV-1 By Engineering Hematopoietic Stem Cells for CCR5 Knockout and B Cell Secretion of Therapeutic Antibodies
    William N. Feist, Sofia E. Luna, Kaya Ben-Efraim, Maria V. Filsinger Interrante, Nelson A. Amorin, Nicole M. Johnston, Theodora U. J. Bruun, Hana Y. Ghanim, Benjamin J. Lesch, Amanda M. Dudek et al. · 2024 · bioRxiv (Cold Spring Harbor Laboratory)
  75. Highly efficient and scarless genome editing via essential-gene-coupled homology-directed repair
    Joo Hye Yeo, Hyongbum Henry Kim, Sang Ho Oh, Jinu Lee · 2026 · Genome Research
  76. Recurrent pre-leukemic deletions in myeloid malignancies are the result of DNA double-strand breaks followed by microhomology-mediated end joining
    Tzah Feldman, Akhiad Bercovich, Yoni Moskovitz, Noa Chapal-Ilani, Amanda Mitchell, Jessie J.F. Medeiros, Nathali Kaushansky, Tamir Biezuner, Mark David Minden, Vikas K Gupta et al. · 2020 · bioRxiv (Cold Spring Harbor Laboratory)
  77. [Research advances in transplantation for thalassemia major].
    Chu-Wen Huang, Hua Jiang · 2020 · PubMed Central
  78. SIRPAssing other xenograft murine models?
    Rachel E. Rau · 2020 · Blood
  79. Optimized guide RNA selection recommendations for using sp Cas9 gene editing in human hematopoietic stem and progenitor cells
    Han J. M. P. Verhagen, Carlijn Kuijk, Anne M. Kokke, Laurens Rutgers, Santhe A. van der Meulen, Gerard J. van Mierlo, Carlijn Voermans, Emile van den Akker · 2020 · bioRxiv (Cold Spring Harbor Laboratory)
  80. The “Editors” Take to RAG: Promise of CRISPR/Cas9/rAAV6-Based Gene Therapy for RAG2 Deficiency
    Vivien Béziat, Isabelle Meyts · 2021 · Journal of Clinical Immunology
  81. NHEJ-Mediated Gene Editing: An Efficient Approach to Correct Mutations in Hematopoietic Stem and Progenitor Cells from Patients with Fanconi Anemia
    Francisco J Roman‐Rodriguez, Laura Ugalde, Lara A. Alvarez, Begoña Díez, Cristina Risueño, Marta Cortón, Massimo Bogliolo, Carmen Ayuso, Marı́a José Ramı́rez, Sandra Rodríguez et al. · 2018 · SSRN Electronic Journal
  82. WITHDRAWN: A Universal Approach to Target Various HBB Gene Mutations in Hematopoietic Stem/Progenitor Cells for Beta-Thalassemia Gene Therapy by CRISPR/Cas9 and the rAAV6 Vector
    Li-Na He, Yi Yang, Yi Cheng, Han Wu, Shouheng Lin, Bing Song, Neng-Qing Liu, Diyu Chen, Dian Lu, Ying-Hong Yang et al. · 2020 · Research Square
  83. Gene Therapy in Cellular Immunodeficiencies
    Tom Fox, Claire Booth, Emma C. Morris · 2021 · Rare diseases of the immune system
  84. Ultra-deep sequencing reveals no evidence of oncogenic mutations or enrichment by ex vivo CRISPR/Cas9 genome editing in human hematopoietic stem and progenitor cells
    M. Kyle Cromer, Valentin V. Barsan, Erich B. Jaeger, Mengchi Wang, Jessica P. Hampton, Feng Chen, Drew Kennedy, Irina Khrebtukova, Ana Granat, Tiffany Truong et al. · 2021 · bioRxiv (Cold Spring Harbor Laboratory)
  85. A Universal Approach to Target Various HBB Gene Mutations in Hematopoietic Stem/Progenitor Cells for Beta-Thalassemia Gene Therapy by CRISPR/Cas9 and the rAAV6 Vector
    Lina He, Yi Yang, Yi Cheng, Han Wu, Shouheng Lin, Bing Song, Neng-Qing Liu, Diyu Chen, Dian Lu, Yinghong Yang et al. · 2020 · Research Square
  86. Regulation of vesicular trafficking in presence of mutations in contractile proteins
    Yuanyuan Dai · 2022
  87. Testing of Antibiotic Resistance Genes (Args) in Environmental Samples Using the Crispr-Cas12a Biosensing Assay
    Sheldon Shervon Hilaire, Juhong Chen, Kang Xia · 2022 · SSRN Electronic Journal
  88. CRISPR/Cas9 system in hematopoietic stem cells: Basic research and clinical applications
    Esra Albayrak · 2023 · Journal of Experimental and Clinical Medicine
  89. Nghiên cứu chỉnh sửa gen trên tế bào gốc tạo máu bằng hệ thống crispr/cas9
    Vũ Thị Hà, Đoàn Thị Kim Phượng, Lương Thị Lan Anh, Nguyễn Văn Long, Bạch Huy Anh, Đào Ngọc Bắc, Trần Thị Huyền Trang, Nguyễn Việt Trung, Trần Đức Phấn · 2023 · Tạp chí Nghiên cứu Y học
  90. Targeted knock-ins with pseudovirus for the stable expression of large transgenes
    2023 · Nature Biomedical Engineering
  91. Integrational modelling for molecular analysis in a CRISPR/Cas edited iPSC-cardiomyocyte model of Dilated Cardiomyopathy
    Xu, Hang · 2023
  92. Near-perfect precise on-target editing of human hematopoietic stem and progenitor cells
    Fanny-Meï Cloarec-Ung, Jamie Beaulieu, Arunan Suthananthan, Bernhard Lehnertz, Guy Sauvageau, Hilary M. Sheppard, David J. H. F. Knapp · 2023 · eLife
  93. Near-perfect precise on-target editing of human hematopoietic stem and progenitor cells
    Fanny-Meï Cloarec-Ung, Jamie Beaulieu, Arunan Suthananthan, Bernhard Lehnertz, Guy Sauvageau, Hilary M. Sheppard, David J. H. F. Knapp · 2024 · eLife
  94. Genome editing in K562 cells suggests a functional role for the XmnI Gγ polymorphism: a widely used genetic marker in β-thalassemia and sickle cell disease patients
    Azadeh Ahmadifard, Nahal Maroofi, Maryam Maleki Tehrani, Tahere Dabestani, Masoumeh Sadat Mousavi Maleki, Sepideh Bayrami, Mehdi Banan · 2024 · Cellular and Molecular Biology
  95. PROTAC molecule-mediated SpCas9 protein degradation for precise genome editing
    Shengnan Sun, Renhong Sun, Minkang Tan, Maarten Kip, Xiaobao Yang, Baoxu Pang · 2025 · bioRxiv (Cold Spring Harbor Laboratory)
  96. The future of genome studies of complex human and plant disease
    Sharav A. Desai, Vipul Pralhadbhai Patel, Sandip D. Nagare, Nishant B. Pagare, Madhulika J. More · 2025 · Elsevier eBooks
  97. 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
  98. RNA Editors Sculpt the Transcriptome During Terminal Erythropoiesis
    Areum Han, Alena V. Yermalovich, Mohamad Ali Toufic Najia, Daniel S. Pearson, Yuko Fujiwara, Michael Bolgov, Caroline Kubaczka, Trista E. North, Vanessa Lundin, Stuart H. Orkin et al. · 2025 · bioRxiv (Cold Spring Harbor Laboratory)
  99. Development of Novel Low-DMSO Cryoprotectant for Peripheral Blood Stem Cell Preservation
    Zhi Rui Guo, Mingxin He, Yi-Huizhi Zhang, Qiang Jiang, Qiang Wang · 2025 · Journal of Visualized Experiments
  100. Editing of ADA2 Point Mutation in Human Hematopoietic Stem Cells
    Pavel Kopcil, Carolina Wieczorek Ervik, Ganna Reint, Katariina Mamia, Monika Szymańska, Shiva Dahal‐Koirala, Jacob Conradi, Sigrid Fu Skjelbostad, Oda A. Dønåsen, Xiaojun Jiang et al. · 2025 · bioRxiv (Cold Spring Harbor Laboratory)