Homology-driven genome editing in hematopoietic stem and progenitor cells using ZFN mRNA and AAV6 donors

Fast gapped-read alignment with Bowtie 2

Langmead, B., & Salzberg, S. L. (2012) · Nature Methods · doi:10.1038/nmeth.1923 · Cited by 56,400

Modulating Innate and Adaptive Immunity by (R)-Roscovitine: Potential Therapeutic Opportunity in Cystic Fibrosis

Meijer, L., Nelson, D. J., Riazanski, V., Gabdoulkhakova, A. G., Héry-Arnaud, G., Berre, R. L., Loaëc, N., Oumata, N., Galons, H., Nowak, E., et al. (2016) · Journal of Innate Immunity · doi:10.1159/000444256 · Cited by 5,008

ZFN, TALEN, and CRISPR/Cas-based methods for genome engineering

Gaj, T., Gersbach, C. A., & Barbas, C. F. (2013) · Trends in Biotechnology · doi:10.1016/j.tibtech.2013.04.004 · Cited by 3,820

Double nicking by RNA-guided CRISPR Cas9 for enhanced genome editing specificity

Ran, F. A., Hsu, P. D., Lin, C.-Y., Gootenberg, J. S., Konermann, S., Trevino, A. E., Scott, D. A., Inoue, A., Matoba, S., Zhang, Y., et al. (2013) · Cell · doi:10.1016/j.cell.2013.08.021 · Cited by 3,299

Genome editing with engineered zinc finger nucleases

Urnov, F. D., Rebar, E. J., Holmes, M. C., Zhang, H. S., & Gregory, P. D. (2010) · Nature Reviews Genetics · doi:10.1038/nrg2842 · Cited by 2,285

Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1

Hacein-Bey-Abina, S., Garrigue, A., Wang, G. P., Soulier, J., Lim, A., Morillon, E., Clappier, E., Caccavelli, L., Delabesse, E., Beldjord, K., et al. (2008) · Journal of Clinical Investigation · doi:10.1172/jci35700 · Cited by 1,854

Hematopoietic Stem Cell Gene Therapy with a Lentiviral Vector in X-Linked Adrenoleukodystrophy

Cartier, N., Hacein-Bey-Abina, S., Bartholomae, C. C., Veres, G., Schmidt, M., Kutschera, I., Vidaud, M., Abel, U., Dal-Cortivo, L., Caccavelli, L., et al. (2009) · Science · doi:10.1126/science.1171242 · Cited by 1,508

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

Xiao, X., Li, J., & Samulski, R. J. (1998) · Journal of Virology · doi:10.1128/jvi.72.3.2224-2232.1998 · Cited by 1,381

Gene Editing of CCR5 in Autologous CD4 T Cells of Persons Infected with HIV

Tebas, P., Stein, D., Tang, W. W., Frank, I., Wang, S. Q., Lee, G., Spratt, S. K., Surosky, R. T., Giedlin, M. A., Nichol, G., et al. (2014) · New England Journal of Medicine · doi:10.1056/nejmoa1300662 · Cited by 1,357

Regulation of DNA repair throughout the cell cycle

Branzei, D., & Foiani, M. (2008) · Nature Reviews Molecular Cell Biology · doi:10.1038/nrm2351 · Cited by 1,228

Delivering genes with human immunodeficiency virus-derived vehicles: still state-of-the-art after 25 years

Wolff, J. H., & Mikkelsen, J. G. (2022) · Journal of Biomedical Science · doi:10.1186/s12929-022-00865-4 · Cited by 47

Understanding and overcoming adverse consequences of genome editing on hematopoietic stem and progenitor cells

Lee, B., Lozano, R. J., & Dunbar, C. E. (2021) · Molecular Therapy · doi:10.1016/j.ymthe.2021.09.001 · Cited by 39

Optimization of AAV6 transduction enhances site-specific genome editing of primary human lymphocytes

Rogers, G. L., Huang, C., Clark, R. D. E., Seclén, E., Chen, H.-Y., & Cannon, P. M. (2021) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2021.09.003 · Cited by 41

Genome editing in human hematopoietic stem and progenitor cells via CRISPR-Cas9-mediated homology-independent targeted integration

Bloomer, H., Smith, R. H., Hakami, W., & Larochelle, A. (2020) · Molecular Therapy · doi:10.1016/j.ymthe.2020.12.010 · Cited by 42

High-Efficiency Transduction of Primary Human Hematopoietic Stem/Progenitor Cells by AAV6 Vectors: Strategies for Overcoming Donor-Variation and Implications in Genome Editing

Ling, C., Bhukhai, K., Yin, Z., Tan, M., Yöder, M. C., Leboulch, P., Payen, E., & Srivastava, A. (2016) · Scientific Reports · doi:10.1038/srep35495 · Cited by 41

Adeno-Associated Virus Vectors and Stem Cells: Friends or Foes?

Brown, N. J., Song, L., Rao, K. N., & Hirsch, M. L. (2017) · Human Gene Therapy · doi:10.1089/hum.2017.038 · Cited by 42

CRISPR/Cas9 Genome Engineering in Engraftable Human Brain-Derived Neural Stem Cells

Dever, D. P., Scharenberg, S. G., Camarena, J., Kildebeck, E., Clark, J. T., Martin, R. M., Bak, R. O., Tang, Y., Dohse, M., Birgmeier, J. A., et al. (2019) · iScience · doi:10.1016/j.isci.2019.04.036 · Cited by 40

Myoediting: Toward Prevention of Muscular Dystrophy by Therapeutic Genome Editing

Zhang, Y., Long, C., Bassel‐Duby, R., & Olson, E. N. (2018) · Physiological Reviews · doi:10.1152/physrev.00046.2017 · Cited by 42

Targeted Gene Delivery: Where to Land

Pavani, G., & Amendola, M. (2021) · Frontiers in Genome Editing · doi:10.3389/fgeed.2020.609650 · Cited by 41

Genetic therapies for sickle cell disease

Esrick, E. B., & Bauer, D. E. (2018) · Seminars in Hematology · doi:10.1053/j.seminhematol.2018.04.014 · Cited by 39

Gene therapy and genome editing for primary immunodeficiency diseases

Zhang, Z., Thrasher, A. J., & Zhang, F. (2019) · Genes & Diseases · doi:10.1016/j.gendis.2019.07.007 · Cited by 38

CRISPR/Cas9-modified hematopoietic stem cells—present and future perspectives for stem cell transplantation

Daniel-Moreno, A., Lamsfus‐Calle, A., Raju, J., Antony, J. S., Handgretinger, R., & Mezger, M. (2019) · Bone Marrow Transplantation · doi:10.1038/s41409-019-0510-8 · Cited by 40

Multiplex HDR for disease and correction modeling of SCID by CRISPR genome editing in human HSPCs

Iancu, O., Allen, D. G., Knop, O., Zehavi, Y., Breier, D., Arbiv, A., Lev, A., Lee, Y. N., Beider, K., Nagler, A., et al. (2022) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2022.12.006 · Cited by 37

Functional interrogation of non-coding DNA through CRISPR genome editing

Canver, M. C., Bauer, D. E., & Orkin, S. H. (2017) · Methods · doi:10.1016/j.ymeth.2017.03.008 · Cited by 36

Homology-directed gene-editing approaches for hematopoietic stem and progenitor cell gene therapy

Azhagiri, M. K. K., Babu, P., Venkatesan, V., & Thangavel, S. (2021) · Stem Cell Research & Therapy · doi:10.1186/s13287-021-02565-6 · Cited by 35

Efficient expansion and CRISPR-Cas9-mediated gene correction of patient-derived hepatocytes for treatment of inherited liver diseases

Zhang, K., Wan, P., Wang, L., Wang, Z., Tan, F., Li, J., Ma, X., Cen, J., Yuan, X., Liu, Y., et al. (2024) · Cell stem cell · doi:10.1016/j.stem.2024.04.022 · Cited by 36

A genome editing primer for the hematologist

Hoban, M. D., & Bauer, D. E. (2016) · Blood · doi:10.1182/blood-2016-01-678151 · Cited by 32

T ranslational A dvances in the F ield of P ulmonary H ypertension Molecular Medicine of Pulmonary Arterial Hypertension. From Population Genetics to Precision Medicine and Gene Editing

Austin, E. D., West, J. D., Loyd, J. E., & Hemnes, A. R. (2016) · American Journal of Respiratory and Critical Care Medicine · doi:10.1164/rccm.201605-0905pp · Cited by 33

Stem cell-derived clade F AAVs mediate high-efficiency homologous recombination-based genome editing

Smith, L. J., Wright, J. B., Clark, G., Ul-Hasan, T., Jin, X., Fong, A., Chandra, M., Martin, T. S., Rubin, H. L., Knowlton, D., et al. (2018) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1802343115 · Cited by 33

Transient inhibition of 53BP1 increases the frequency of targeted integration in human hematopoietic stem and progenitor cells

Baik, R., Cromer, M. K., Glenn, S. E., Vakulskas, C. A., Chmielewski, K. O., Dudek, A. M., Feist, W. N., Klermund, J., Shipp, S., Cathomen, T., et al. (2024) · Nature Communications · doi:10.1038/s41467-023-43413-w · Cited by 31

CRISPR/Cas9 Targeted Gene Editing and Cellular Engineering in Fanconi Anemia

Osborn, M. J., Lonetree, C.-L., Webber, B. R., Patel, D., Dunmire, S. K., DeFeo, A. P., McElroy, A. N., MacMillan, M. L., Wagner, J. E., Blazar, B. R., et al. (2016) · Stem Cells and Development · doi:10.1089/scd.2016.0149 · Cited by 34

Inducible CRISPR genome-editing tool: classifications and future trends

Dai, X., Chen, X., Fang, Q., Li, J., & Bai, Z. (2017) · Critical Reviews in Biotechnology · doi:10.1080/07388551.2017.1378999 · Cited by 32

Inducing CCR5Δ32/Δ32 Homozygotes in the Human Jurkat CD4+ Cell Line and Primary CD4+ Cells by CRISPR-Cas9 Genome-Editing Technology

Qi, C., Li, D., Jiang, X., Jia, X., Lu, L., Wang, Y., Sun, J., Shao, Y., & Wei, M. (2018) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2018.05.012 · Cited by 33

Genome Editing for β-Hemoglobinopathies: Advances and Challenges

Frati, G., & Miccio, A. (2021) · Journal of Clinical Medicine · doi:10.3390/jcm10030482 · Cited by 31

Gene editing and its application for hematological diseases

Osborn, M. J., Belanto, J. J., Tolar, J., & Voytas, D. F. (2016) · International Journal of Hematology · doi:10.1007/s12185-016-2017-z · Cited by 32

Enhanced Transduction of Human Hematopoietic Stem Cells by AAV6 Vectors: Implications in Gene Therapy and Genome Editing

Yang, H., Qing, K., Keeler, G. D., Yin, L., Mietzsch, M., Ling, C., Hoffman, B. E., Agbandje‐McKenna, M., Tan, M., Wang, W., et al. (2020) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2020.03.009 · Cited by 31

Answered and Unanswered Questions in Early-Stage Viral Vector Transduction Biology and Innate Primary Cell Toxicity for Ex-Vivo Gene Editing

Dudek, A. M., & Porteus, M. H. (2021) · Frontiers in Immunology · doi:10.3389/fimmu.2021.660302 · Cited by 30

In Vivo Hematopoietic Stem Cell Transduction

Richter, M., Stone, D., Miao, C. H., Humbert, O., Kiem, H., Papayannopoulou, T., & Lieber, A. (2017) · Hematology/Oncology Clinics of North America · doi:10.1016/j.hoc.2017.06.001 · Cited by 29

Gene Therapy for Beta-Hemoglobinopathies: Milestones, New Therapies and Challenges

Ghiaccio, V., Chappell, M., Rivella, S., & Breda, L. (2019) · Molecular Diagnosis & Therapy · doi:10.1007/s40291-019-00383-4 · Cited by 29

Cell and Gene Therapy for HIV Cure

Peterson, C. W., & Kiem, H. (2017) · Current topics in microbiology and immunology · doi:10.1007/82_2017_71 · Cited by 30

Historical Perspective on the Current Renaissance for Hematopoietic Stem Cell Gene Therapy

Kohn, D. B. (2017) · Hematology/Oncology Clinics of North America · doi:10.1016/j.hoc.2017.06.006 · Cited by 28

Humanized Mouse Model of HIV-1 Latency with Enrichment of Latent Virus in PD-1 + and TIGIT + CD4 T Cells

Llewellyn, G. N., Seclén, E., Wietgrefe, S. W., Liu, S., Chateau, M., Pei, H., Perkey, K., Marsden, M. D., Hinkley, S. J., Paschon, D. E., et al. (2019) · Journal of Virology · doi:10.1128/jvi.02086-18 · Cited by 29

Integration-defective lentiviral vector mediates efficient gene editing through homology-directed repair in human embryonic stem cells

Wang, Y., Wang, Y., Chang, T., Huang, H., & Yee, J.-K. (2017) · Nucleic Acids Research · doi:10.1093/nar/gkw1057 · Cited by 26

Hematopoietic stem cell gene therapy: The optimal use of lentivirus and gene editing approaches

Lamsfus‐Calle, A., Daniel-Moreno, A., Ureña-Bailén, G., Raju, J., Antony, J. S., Handgretinger, R., & Mezger, M. (2019) · Blood Reviews · doi:10.1016/j.blre.2019.100641 · Cited by 29

Identifying and retargeting transcriptional hot spots in the human genome

Cheng, J. K., Lewis, A. M., Kim, D. S., Dyess, T. J., & Alper, H. S. (2016) · Biotechnology Journal · doi:10.1002/biot.201600015 · Cited by 26

CRISPR Editing in Biological and Biomedical Investigation

Ju, X., Xu, J., & Sun, Z. S. (2017) · Journal of Cellular Biochemistry · doi:10.1002/jcb.26154 · Cited by 27

Precise integration of inducible transcriptional elements (PrIITE) enables absolute control of gene expression

Pinto, R., Hansen, L. H., Hintze, J., Almeida, R., Larsen, S., Coskun, M., Davidsen, J., Mitchelmore, C., David, L., Troelsen, J. T., et al. (2017) · Nucleic Acids Research · doi:10.1093/nar/gkx371 · Cited by 25

Delivery methods for site-specific nucleases: Achieving the full potential of therapeutic gene editing

Liu, J., & Shui, S. (2016) · Journal of Controlled Release · doi:10.1016/j.jconrel.2016.11.014 · Cited by 24

Genome edited B cells: a new frontier in immune cell therapies

Rogers, G. L., & Cannon, P. M. (2021) · Molecular Therapy · doi:10.1016/j.ymthe.2021.09.019 · Cited by 27

Gene Therapy and Genome Editing

Boulad, F., Mansilla‐Soto, J., Cabriolu, A., Rivière, I., & Sadelain, M. (2018) · Hematology/Oncology Clinics of North America · doi:10.1016/j.hoc.2017.11.007 · Cited by 24

Homologous Recombination-Based Genome Editing by Clade F AAVs Is Inefficient in the Absence of a Targeted DNA Break

Rogers, G. L., Chen, H.-Y., Morales, H., & Cannon, P. M. (2019) · Molecular Therapy · doi:10.1016/j.ymthe.2019.08.019 · Cited by 24

CRISPR-Cas9 engineering of the RAG2 locus via complete coding sequence replacement for therapeutic applications

Allen, D. G., Knop, O., Itkowitz, B., Kalter, N., Rosenberg, M., Iancu, O., Beider, K., Lee, Y. N., Nagler, A., Somech, R., et al. (2023) · Nature Communications · doi:10.1038/s41467-023-42036-5 · Cited by 25

Current landscape of gene‐editing technology in biomedicine: Applications, advantages, challenges, and perspectives

Zhou, W., Yang, J., Zhang, Y., Hu, X., & Wang, W. (2022) · MedComm · doi:10.1002/mco2.155 · Cited by 23

Nanoparticles targeting hematopoietic stem and progenitor cells: Multimodal carriers for the treatment of hematological diseases

Cruz, L. J., Rezaei, S., Grosveld, F. G., Philipsen, S., & Eich, C. (2022) · Frontiers in Genome Editing · doi:10.3389/fgeed.2022.1030285 · Cited by 23

Targeted modulation of E3 ligases using engineered ubiquitin variants

LeBlanc, N., Mallette, E., & Zhang, W. (2020) · FEBS Journal · doi:10.1111/febs.15536 · Cited by 22

Human gene-engineered calreticulin mutant stem cells recapitulate MPN hallmarks and identify targetable vulnerabilities

Foßelteder, J., Pabst, G., Sconocchia, T., Schlacher, A., Auinger, L., Kashofer, K., Beham‐Schmid, C., Trajanoski, S., Waskow, C., Schöll, W., et al. (2023) · Leukemia · doi:10.1038/s41375-023-01848-6 · Cited by 21

Mouse models in hematopoietic stem cell gene therapy and genome editing

Radtke, S., Humbert, O., & Kiem, H. (2019) · Biochemical Pharmacology · doi:10.1016/j.bcp.2019.113692 · Cited by 22

Therapeutic gene editing strategies using CRISPR-Cas9 for the β-hemoglobinopathies

Papizan, J. B., Porter, S. N., Sharma, A., & Pruett‐Miller, S. M. (2020) · Journal of Biomedical Research · doi:10.7555/jbr.34.20200096 · Cited by 21

Mesenchymal stromal cells improve the transplantation outcome of CRISPR-Cas9 gene-edited human HSPCs

Crippa, S., Conti, A., Vavassori, V., Ferrari, S., Beretta, S., Rivis, S., Bosotti, R., Scala, S., Pirroni, S., Hernández, R. J., et al. (2022) · Molecular Therapy · doi:10.1016/j.ymthe.2022.08.011 · Cited by 19

Combining Engineered Nucleases with Adeno-associated Viral Vectors for Therapeutic Gene Editing

Epstein, B. E., & Schaffer, D. V. (2017) · Advances in experimental medicine and biology · doi:10.1007/978-3-319-63904-8_2 · Cited by 20

TraFo-CRISPR: Enhanced Genome Engineering by Transient Foamy Virus Vector-Mediated Delivery of CRISPR/Cas9 Components

Lindel, F., Dodt, C., Weidner, N., Noll, M., Bergemann, F., Behrendt, R., Fischer, S., Dietrich, J., Cartellieri, M., Hamann, M. V., et al. (2019) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2019.10.006 · Cited by 22

Genome-edited adult stem cells: Next-generation advanced therapy medicinal products

Benabdellah, K., Sánchez-Hernández, S., Aguilar-González, A., Maldonado‐Pérez, N., Gutiérrez-Guerrero, A., Cortijo-Gutierréz, M., Ramos-Hernández, I., Tristán‐Manzano, M., Galindo‐Moreno, P., Herrera, C., et al. (2020) · Stem Cells Translational Medicine · doi:10.1002/sctm.19-0338 · Cited by 21

Senescence and inflammation are unintended adverse consequences of CRISPR-Cas9/AAV6-mediated gene editing in hematopoietic stem cells

Conti, A., Giannetti, K., Midena, F., Beretta, S., Gualandi, N., Marco, R. D., Carsana, E., Varesi, A., Tavella, T., Alessandrini, L., et al. (2025) · Cell Reports Medicine · doi:10.1016/j.xcrm.2025.102157 · Cited by 24

In Vivo Hematopoietic Stem Cell Genome Editing: Perspectives and Limitations

Psatha, N., Paschoudi, K., Παπαδοπούλου, Α., & Yannaki, E. (2022) · Genes · doi:10.3390/genes13122222 · Cited by 24

CRISPR/Cas9-Based Cellular Engineering for Targeted Gene Overexpression

Osborn, M. J., Lees, C. J., McElroy, A. N., Merkel, S. C., Eide, C. R., Mathews, W. E., Feser, C. J., Tschann, M. M., McElmury, R., Webber, B. R., et al. (2018) · International Journal of Molecular Sciences · doi:10.3390/ijms19040946 · Cited by 21

In vitro transcribed mRNA for expression of designer nucleases: Advantages as a novel therapeutic for the management of chronic HBV infection

Ely, A., Singh, P., Smith, T. S., & Arbuthnot, P. B. (2020) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2020.05.010 · Cited by 19

Intrathymic adeno-associated virus gene transfer rapidly restores thymic function and long-term persistence of gene-corrected T cells

Pouzolles, M., Machado, A., Guilbaud, M., Irla, M., Gailhac, S., Barennes, P., Cesana, D., Calabria, A., Benedicenti, F., Sergé, A., et al. (2019) · Journal of Allergy and Clinical Immunology · doi:10.1016/j.jaci.2019.08.029 · Cited by 17

Removal of innate immune barriers allows efficient transduction of quiescent human hematopoietic stem cells

Valeri, E., Unali, G., Piras, F., Alezz, M. A., Pais, G., Benedicenti, F., Lidonnici, M. R., Cuccovillo, I., Castiglioni, I., Arévalo, S., et al. (2023) · Molecular Therapy · doi:10.1016/j.ymthe.2023.11.020 · Cited by 17

Gene Therapy: The View from NCATS

Brooks, P. J., Yang, N. N., & Austin, C. P. (2016) · Human Gene Therapy · doi:10.1089/hum.2016.29018.pjb · Cited by 16

Stacking up CRISPR against RNA i for therapeutic gene inhibition

Haussecker, D. (2016) · FEBS Journal · doi:10.1111/febs.13742 · Cited by 16

Therapeutic genome editing with engineered nucleases

Haas, S. A., Dettmer‐Monaco, V., & Cathomen, T. (2017) · Hämostaseologie · doi:10.5482/hamo-16-09-0035 · Cited by 15

BAR-Seq clonal tracking of gene-edited cells

Ferrari, S., Beretta, S., Jacob, A., Cittaro, D., Albano, L., Merelli, I., Naldini, L., & Genovese, P. (2021) · Nature Protocols · doi:10.1038/s41596-021-00529-x · Cited by 15

A Curative DNA Code for Hematopoietic Defects

Porteus, M. H., Pavel-Dinu, M., & Pai, S. (2022) · Hematology/Oncology Clinics of North America · doi:10.1016/j.hoc.2022.05.002 · Cited by 15

Nuclease-free Adeno-Associated Virus-Mediated Il2rg Gene Editing in X-SCID Mice

Hiramoto, T., Li, L. B., Funk, S. E., Hirata, R. K., & Russell, D. W. (2018) · Molecular Therapy · doi:10.1016/j.ymthe.2018.02.028 · Cited by 16

Advances and Obstacles in Homology-Mediated Gene Editing of Hematopoietic Stem Cells

Salisbury-Ruf, C. T., & Larochelle, A. (2021) · Journal of Clinical Medicine · doi:10.3390/jcm10030513 · Cited by 15

Genetic Manipulation Strategies for β-Thalassemia: A Review

Zakaria, N. A., Bahar, R., Abdullah, W. Z., Yusoff, A. A. M., Shamsuddin, S., Wahab, R. A., & Johan, M. F. (2022) · Frontiers in Pediatrics · doi:10.3389/fped.2022.901605 · Cited by 15

Time-Restricted PiggyBac DNA Transposition by Transposase Protein Delivery Using Lentivirus-Derived Nanoparticles

Skipper, K. A., Nielsen, M. G., Andersen, S., Ryø, L. B., Bak, R. O., & Mikkelsen, J. G. (2018) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2018.02.006 · Cited by 16

Lentiviral Vectors: From Wild-Type Viruses to Efficient Multi-Functional Delivery Vectors

Arrasate, A., Lopez-Robles, C., Zuazo, M., Baños-Mateos, S., Martín, C., Lamsfus‐Calle, A., & Fertin, M. J. (2025) · International Journal of Molecular Sciences · doi:10.3390/ijms26178497 · Cited by 14

Curative approaches for sickle cell disease: A review of allogeneic and autologous strategies

Bauer, D. E., Brendel, C., & Fitzhugh, C. D. (2017) · Blood Cells Molecules and Diseases · doi:10.1016/j.bcmd.2017.08.014 · Cited by 15

Treating Sickle Cell Disease: Gene Therapy Approaches

Cavazzana, M., Corsia, A., Brusson, M., Miccio, A., & Semeraro, M. (2024) · The Annual Review of Pharmacology and Toxicology · doi:10.1146/annurev-pharmtox-022124-022000 · Cited by 15

Genome editing: a robust technology for human stem cells

Chandrasekaran, A. P., Song, M., & Ramakrishna, S. (2017) · Cellular and Molecular Life Sciences · doi:10.1007/s00018-017-2522-0 · Cited by 14

Efficient Enrichment of Gene-Modified Primary T Cells via CCR5-Targeted Integration of Mutant Dihydrofolate Reductase

Paul, B., Ibarra, G. S. R., Hubbard, N., Einhaus, T., Astrakhan, A., Rawlings, D. J., Kiem, H., & Peterson, C. W. (2018) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2018.04.002 · Cited by 14

AAV6 Is Superior to Clade F AAVs in Stimulating Homologous Recombination-Based Genome Editing in Human HSPCs

Dudek, A. M., & Porteus, M. H. (2019) · Molecular Therapy · doi:10.1016/j.ymthe.2019.09.005 · Cited by 14

Gene therapy's out-of-body experience

Scott, C. T., & DeFrancesco, L. (2016) · Nature Biotechnology · doi:10.1038/nbt.3592 · Cited by 14

Gene Therapy Approaches to Human Immunodeficiency Virus and Other Infectious Diseases

Rogers, G. L., & Cannon, P. M. (2017) · Hematology/Oncology Clinics of North America · doi:10.1016/j.hoc.2017.06.008 · Cited by 13

Tissue and cell-type-specific transduction using rAAV vectors in lung diseases

Kochergin-Nikitsky, K. S., Belova, L., Лавров, А. В., & Smirnikhina, S. (2021) · Journal of Molecular Medicine · doi:10.1007/s00109-021-02086-y · Cited by 14

Chemically modified in-vitro-transcribed mRNA encoding thrombopoietin stimulates thrombopoiesis in mice

Zhang, Y., Xi, X., Yu, H., Yang, L., Lin, J., Yang, W., Liu, J., Fan, X., & Xu, Y. (2022) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2022.08.017 · Cited by 13

20 years of Nature Biotechnology biomedical research

Azvolinsky, A., Schmidt, C. W., Waltz, E., & Webb, S. (2016) · Nature Biotechnology · doi:10.1038/nbt.3509 · Cited by 13

Genome Editing in Stem Cells for Disease Therapeutics

Song, M., & Ramakrishna, S. (2018) · Molecular Biotechnology · doi:10.1007/s12033-018-0072-9 · Cited by 13

Engineering hematopoietic stem cells toward a functional cure of human immunodeficiency virus infection

Wang, J., & Holmes, M. C. (2016) · Cytotherapy · doi:10.1016/j.jcyt.2016.07.007 · Cited by 12

Gene editing-based targeted integration for correction of Wiskott-Aldrich syndrome

Pille, M., Avila, J. M., Park, S. H., Le, C. Q., Xue, H., Haerynck, F., Saxena, L., Lee, C. M., Shpall, E. J., Bao, G., et al. (2024) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2024.101208 · Cited by 11

Prime editing in hematopoietic stem cells—From ex vivo to in vivo CRISPR-based treatment of blood disorders

Wolff, J. H., & Mikkelsen, J. G. (2023) · Frontiers in Genome Editing · doi:10.3389/fgeed.2023.1148650 · Cited by 12

Clinical Applications of Genome Editing to HIV Cure

Wang, C. X., & Cannon, P. M. (2016) · AIDS Patient Care and STDs · doi:10.1089/apc.2016.0233 · Cited by 11

Efficient Correction of the Sickle Mutation in Human Hematopoietic Stem Cells Using a Cas9 Ribonucleoprotein ComplexPreprint

DeWitt, M. A., Magis, W., Bray, N., Wang, T., Berman, J. R., Urbinati, F., Muñoz, D. P., Kohn, D. B., Walters, M. C., Carroll, D., et al. (2016) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/036236 · Cited by 12

CRISPR/Cas-Based Gene Editing Strategies for DOCK8 Immunodeficiency Syndrome

Ravendran, S., Hernández, S. S., König, S., & Bak, R. O. (2022) · Frontiers in Genome Editing · doi:10.3389/fgeed.2022.793010 · Cited by 11

HEK293T Cells Are Heterozygous for CCR5 Delta 32 Mutation

Qi, C., Jia, X., Lu, L., Ma, P., & Wei, M. (2016) · PLoS ONE · doi:10.1371/journal.pone.0152975 · Cited by 10

Challenges and opportunities in gene editing of B cells

Edelstein, J., Fritz, M., & Lai, S. K. (2022) · Biochemical Pharmacology · doi:10.1016/j.bcp.2022.115285 · Cited by 9

CRISPR/Cas9-mediated generic protein tagging in mammalian cells

Thöne, F. M. B., Kurrle, N., von Melchner, H., & Schnütgen, F. (2019) · Methods · doi:10.1016/j.ymeth.2019.02.018 · Cited by 9

Gene Editing-Based Technologies for Beta-hemoglobinopathies Treatment

Rahimmanesh, I., Boshtam, M., Kouhpayeh, S., Khanahmad, H., Dabiri, A., Ahangarzadeh, S., Esmaeili, Y., Bidram, E., Vaseghi, G., Javanmard, S. H., et al. (2022) · Biology · doi:10.3390/biology11060862 · Cited by 11

Use of mariner transposases for one-step delivery and integration of DNA in prokaryotes and eukaryotes by transfection

Trubitsyna, M., Michlewski, G., Finnegan, D., Elfick, A. P. D., Rosser, S. J., Richardson, J. M., & French, C. E. (2017) · Nucleic Acids Research · doi:10.1093/nar/gkx113 · Cited by 9

Basic and Clinical Application of Adeno-Associated Virus–Mediated Genome Editing

He, X., Xie, H., Liu, X., & Gu, F. (2018) · Human Gene Therapy · doi:10.1089/hum.2018.190 · Cited by 7

A comparison of DNA repair pathways to achieve a site-specific gene modification of the Bruton's tyrosine kinase gene

Gray, D., Santos, J., Keir, A., Villegas, I. J., Maddock, S., Trope, E. C., Long, J. D., & Kuo, C. Y. (2021) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2021.12.014 · Cited by 11

Hematopoietic stem and progenitors cells gene editing: Beyond blood disorders

Buffa, V., Vargas, J. R. A., Galy, A. H. M., Spinozzi, S., & Rocca, C. J. (2023) · Frontiers in Genome Editing · doi:10.3389/fgeed.2022.997142 · Cited by 10

Genome editing for sickle cell disease: still time to correct?

Ceglie, G., Lecis, M., Canciani, G., Algeri, M., & Frati, G. (2023) · Frontiers in Pediatrics · doi:10.3389/fped.2023.1249275 · Cited by 14

Enhancement of adeno‐associated virus serotype 6 transduction into T cells with cell‐penetrating peptides

Moço, P. D., Dash, S., & Kamen, A. (2023) · The Journal of Gene Medicine · doi:10.1002/jgm.3627 · Cited by 9

CD34+ cells from dental pulp stem cells with a ZFN-mediated and homology-driven repair-mediated locus-specific knock-in of an artificial β-globin gene

Chattong, S., Ruangwattanasuk, O., Yindeedej, W., Setpakdee, A., & Manotham, K. (2017) · Gene Therapy · doi:10.1038/gt.2017.42 · Cited by 10

A directed evolution approach to select for novel Adeno-associated virus capsids on an HIV-1 producer T cell line

Wooley, D. P., Sharma, P., Weinstein, J. R., Narayan, P. K. L., Schaffer, D. V., & Excoffon, K. J. D. A. (2017) · Journal of Virological Methods · doi:10.1016/j.jviromet.2017.09.008 · Cited by 10

In Utero Gene Therapy and Genome Editing

Hartman, H. A., Rossidis, A. C., & Peranteau, W. H. (2018) · Current Stem Cell Reports · doi:10.1007/s40778-018-0117-9 · Cited by 9

Production of therapeutic levels of human FIX-R338L by engineered B cells using GMP-compatible medium

David, M., Monteferrario, D., Saviane, G., Jeanneau, C., Marchetti, I., Dupont, C. F., Dumont, C., Fontenot, J. D., de la Rosa, M., & Fenard, D. (2023) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2023.101111 · Cited by 8

Toward In Vivo Gene Therapy Using CRISPR

Skipper, K. A., & Mikkelsen, J. G. (2019) · Methods in molecular biology · doi:10.1007/978-1-4939-9170-9_18 · Cited by 8

478. Highly Efficient Targeted Gene Addition in CD34+ Hematopoietic Stem/Progenitor Cells by Combining ZFN mRNA and AAV6 Donor Delivery

Wang, J., Exline, C. M., DeClercq, J., Li, P. W. F., Gregory, P. D., Cannon, P. M., & Holmes, M. C. (2015) · Molecular Therapy · doi:10.1016/s1525-0016(16)34087-4

Delivery of Genome Editing Reagents to Hematopoietic Stem/Progenitor Cells

Hoban, M. D., Romero, Z. G., Cost, G. J., Mendel, M. C., Holmes, M. C., & Kohn, D. B. (2016) · Current Protocols in Stem Cell Biology · doi:10.1002/9780470151808.sc05b04s36 · Cited by 9

330. Using CRISPR/Cas9 to Edit Hematopoietic Stem and Progenitor Cells

Seclén, E., Chen, H.-Y., Llewellyn, G. N., Yang, S. J., Chateau, M., Wang, C. X., & Cannon, P. M. (2016) · Molecular Therapy · doi:10.1016/s1525-0016(16)33139-2

Editing the Sickle Cell Disease Mutation in Human Hematopoietic Stem Cells: Comparison of Endonucleases and Homologous Donor Templates

Romero, Z. G., Lomova, A., Said, S., Hoyt-Miggelbrink, A. M., Kuo, C. Y., Campo-Fernández, B., Hoban, M. D., Masiuk, K. E., Clark, D. N., Long, J. D., et al. (2019) · Molecular Therapy · doi:10.1016/j.ymthe.2019.05.014 · Cited by 123

Long-term multilineage engraftment of autologous genome-edited hematopoietic stem cells in nonhuman primates

Peterson, C. W., Wang, J., Norman, K. K., Norgaard, Z. K., Humbert, O., Tse, C. K., Yan, J. J., Trimble, R. G., Shivak, D. A., Rebar, E. J., et al. (2016) · Blood · doi:10.1182/blood-2015-09-672337 · Cited by 68