Advanced delivery systems for gene editing: A comprehensive review from the GenE-HumDi COST Action Working Group
In the past decade, precise targeting through genome editing has emerged as a promising alternative to traditional therapeutic approaches. Genome editing can be performed using various platforms, where programmable DNA nucleases create permanent genetic changes at specific genomic locations due to t...
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Elsevier
2025-03-01
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| author | Alessia Cavazza Francisco J. Molina-Estévez Álvaro Plaza Reyes Victor Ronco Asma Naseem Špela Malenšek Peter Pečan Annalisa Santini Paula Heredia Araceli Aguilar-González Houria Boulaiz Qianqian Ni Marina Cortijo-Gutierrez Kristina Pavlovic Inmaculada Herrera Berta de la Cerda Emilio M. Garcia-Tenorio Eva Richard Sergio Granados-Principal Arístides López-Márquez Mariana Köber Marijana Stojanovic Melita Vidaković Irene Santos-Garcia Lorea Blázquez Emily Haughton Dongnan Yan Rosario María Sánchez-Martín Loubna Mazini Gloria Gonzalez Aseguinolaza Annarita Miccio Paula Rio Lourdes R. Desviat Manuel A.F.V. Gonçalves Ling Peng Cecilia Jiménez-Mallebrera Francisco Martin Molina Dhanu Gupta Duško Lainšček Yonglun Luo Karim Benabdellah |
| author_facet | Alessia Cavazza Francisco J. Molina-Estévez Álvaro Plaza Reyes Victor Ronco Asma Naseem Špela Malenšek Peter Pečan Annalisa Santini Paula Heredia Araceli Aguilar-González Houria Boulaiz Qianqian Ni Marina Cortijo-Gutierrez Kristina Pavlovic Inmaculada Herrera Berta de la Cerda Emilio M. Garcia-Tenorio Eva Richard Sergio Granados-Principal Arístides López-Márquez Mariana Köber Marijana Stojanovic Melita Vidaković Irene Santos-Garcia Lorea Blázquez Emily Haughton Dongnan Yan Rosario María Sánchez-Martín Loubna Mazini Gloria Gonzalez Aseguinolaza Annarita Miccio Paula Rio Lourdes R. Desviat Manuel A.F.V. Gonçalves Ling Peng Cecilia Jiménez-Mallebrera Francisco Martin Molina Dhanu Gupta Duško Lainšček Yonglun Luo Karim Benabdellah |
| author_sort | Alessia Cavazza |
| collection | DOAJ |
| description | In the past decade, precise targeting through genome editing has emerged as a promising alternative to traditional therapeutic approaches. Genome editing can be performed using various platforms, where programmable DNA nucleases create permanent genetic changes at specific genomic locations due to their ability to recognize precise DNA sequences. Clinical application of this technology requires the delivery of the editing reagents to transplantable cells ex vivo or to tissues and organs for in vivo approaches, often representing a barrier to achieving the desired editing efficiency and safety. In this review, authored by members of the GenE-HumDi European Cooperation in Science and Technology (COST) Action, we described the plethora of delivery systems available for genome-editing components, including viral and non-viral systems, highlighting their advantages, limitations, and potential application in a clinical setting. |
| format | Article |
| id | doaj-art-5fcf4f6d779c4efe9cd13b251b9cd391 |
| institution | Kabale University |
| issn | 2162-2531 |
| language | English |
| publishDate | 2025-03-01 |
| publisher | Elsevier |
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| series | Molecular Therapy: Nucleic Acids |
| spelling | doaj-art-5fcf4f6d779c4efe9cd13b251b9cd3912025-02-05T04:31:44ZengElsevierMolecular Therapy: Nucleic Acids2162-25312025-03-01361102457Advanced delivery systems for gene editing: A comprehensive review from the GenE-HumDi COST Action Working GroupAlessia Cavazza0Francisco J. Molina-Estévez1Álvaro Plaza Reyes2Victor Ronco3Asma Naseem4Špela Malenšek5Peter Pečan6Annalisa Santini7Paula Heredia8Araceli Aguilar-González9Houria Boulaiz10Qianqian Ni11Marina Cortijo-Gutierrez12Kristina Pavlovic13Inmaculada Herrera14Berta de la Cerda15Emilio M. Garcia-Tenorio16Eva Richard17Sergio Granados-Principal18Arístides López-Márquez19Mariana Köber20Marijana Stojanovic21Melita Vidaković22Irene Santos-Garcia23Lorea Blázquez24Emily Haughton25Dongnan Yan26Rosario María Sánchez-Martín27Loubna Mazini28Gloria Gonzalez Aseguinolaza29Annarita Miccio30Paula Rio31Lourdes R. Desviat32Manuel A.F.V. Gonçalves33Ling Peng34Cecilia Jiménez-Mallebrera35Francisco Martin Molina36Dhanu Gupta37Duško Lainšček38Yonglun Luo39Karim Benabdellah40Molecular and Cellular Immunology Section, Department of Infection, Immunity & Inflammation, UCL Great Ormond Street Institute of Child Health, University College London, 20 Guilford Street, London WC1N 1DZ, UK; Department of Medical and Surgical Sciences for Children and Adults, University of Modena and Reggio Emilia School of Medicine, Via del Pozzo 71, 41125 Modena, Italy; Corresponding author: Alessia Cavazza, Molecular and Cellular Immunology Section, Department of Infection, Immunity & Inflammation, UCL Great Ormond Street Institute of Child Health, University College London, 20 Guilford Street, London WC1N 1DZ, UK.Department of Genomic Medicine, Pfizer-University of Granada-Andalusian Regional Government Centre for Genomics and Oncological Research (GENYO), Av. de la Ilustración, 114, 18016 Granada, Spain; Fundación para la Investigación Biosanitaria de Andalucía Oriental, Alejandro Otero (FIBAO), Avda. de Madrid 15, 18012 Granada, Spain; Biosanitary Research Institute of Granada (ibs. GRANADA), University of Granada, Av. de Madrid, 15, Beiro, 18012 Granada, SpainDepartment of Regeneration and Cell Therapy, Andalusian Molecular Biology and Regenerative Medicine Centre (CABIMER), Avda. Americo Vespucio, 24, 41092 Seville, SpainDepartment of Genomic Medicine, Pfizer-University of Granada-Andalusian Regional Government Centre for Genomics and Oncological Research (GENYO), Av. de la Ilustración, 114, 18016 Granada, SpainMolecular and Cellular Immunology Section, Department of Infection, Immunity & Inflammation, UCL Great Ormond Street Institute of Child Health, University College London, 20 Guilford Street, London WC1N 1DZ, UKDepartment of Synthetic Biology and Immunology, National Institute of Chemistry, Hajdrihova 19, 1000 Ljubljana, Slovenia; Graduate School of Biomedicine, University of Ljubljana, Kongresni trg, 1000 Ljubljana, SloveniaDepartment of Synthetic Biology and Immunology, National Institute of Chemistry, Hajdrihova 19, 1000 Ljubljana, Slovenia; Graduate School of Biomedicine, University of Ljubljana, Kongresni trg, 1000 Ljubljana, SloveniaImagine Institute, UMR 163 INSERM, 24 Bd du Montparnasse, 75015 Paris, France; Paris City University, 45 Rue des Saints-Pères, 75006 Paris, FranceDepartment of Genomic Medicine, Pfizer-University of Granada-Andalusian Regional Government Centre for Genomics and Oncological Research (GENYO), Av. de la Ilustración, 114, 18016 Granada, Spain; Department of Anatomy and Human Embryology, Faculty of Medicine, University of Granada, Avenida de la Investigación 11, 18016 Granada, SpainDepartment of Genomic Medicine, Pfizer-University of Granada-Andalusian Regional Government Centre for Genomics and Oncological Research (GENYO), Av. de la Ilustración, 114, 18016 Granada, Spain; Biosanitary Research Institute of Granada (ibs. GRANADA), University of Granada, Av. de Madrid, 15, Beiro, 18012 Granada, Spain; Department of Medicinal & Organic Chemistry and Excellence Research Unit of “Chemistry applied to Bio-medicine and the Environment, ” Faculty of Pharmacy, University of Granada, Campus de Cartuja s/n, 18071 Granada, SpainBiosanitary Research Institute of Granada (ibs. GRANADA), University of Granada, Av. de Madrid, 15, Beiro, 18012 Granada, Spain; Department of Anatomy and Human Embryology, Faculty of Medicine, University of Granada, Avenida de la Investigación 11, 18016 Granada, SpainDepartment of Diagnostic Radiology, Yong Loo Lin School of Medicine, National University of Singapore, Singapore 117597, SingaporeDepartment of Genomic Medicine, Pfizer-University of Granada-Andalusian Regional Government Centre for Genomics and Oncological Research (GENYO), Av. de la Ilustración, 114, 18016 Granada, SpainDepartment of Genomic Medicine, Pfizer-University of Granada-Andalusian Regional Government Centre for Genomics and Oncological Research (GENYO), Av. de la Ilustración, 114, 18016 Granada, SpainDepartment of Hematology, Reina Sofía University Hospital, Av. Menéndez Pidal, Poniente Sur, 14004 Córdoba, Spain; Maimonides Institute of Biomedical Research in Cordoba (IMIBIC), Cell Therapy, Av. Menéndez Pidal, Poniente Sur, 14004 Córdoba, SpainDepartment of Regeneration and Cell Therapy, Andalusian Molecular Biology and Regenerative Medicine Centre (CABIMER), Avda. Americo Vespucio, 24, 41092 Seville, SpainCentro de Biología Molecular Severo Ochoa UAM-CSIC, IUBM, CIBERER, IDIPAZ, Universidad Autónoma de Madrid, C. de Pedro Rico, 6, Fuencarral-El Pardo, 28029 Madrid, SpainCentro de Biología Molecular Severo Ochoa UAM-CSIC, IUBM, CIBERER, IDIPAZ, Universidad Autónoma de Madrid, C. de Pedro Rico, 6, Fuencarral-El Pardo, 28029 Madrid, SpainDepartment of Genomic Medicine, Pfizer-University of Granada-Andalusian Regional Government Centre for Genomics and Oncological Research (GENYO), Av. de la Ilustración, 114, 18016 Granada, Spain; Biosanitary Research Institute of Granada (ibs. GRANADA), University of Granada, Av. de Madrid, 15, Beiro, 18012 Granada, Spain; Department of Biochemistry and Molecular Biology 2, Faculty of Pharmacy, University of Granada, Campus de Cartuja s/n, 18071 Granada, SpainNeuromuscular Unit, Institut de Recerca Sant Joan de Déu, Hospital Sant Joan de Déu, C. de Sta. Rosa, 39, 08950 Barcelona, Spain; Biomedical Research Network on Rare Diseases (CIBERER), C. de Melchor Fernández Almagro, 3, Fuencarral-El Pardo, 28029 Madrid, Spain; Department of Genetics, Microbiology and Statistics, University of Barcelona, Gran Via de les Corts Catalanes, 585, L'Eixample, 08007 Barcelona, SpainBiomedical Research Network on Bioengineering, Biomaterials and Nanomedicine (CIBER-BBN), C/ Monforte de Lemos 3-5, Pabellón 11, Planta 0, 28029 Madrid, Spain; Institut de Ciència de Materials de Barcelona (ICMAB-CSIC), Campus UAB, Universitat Autònoma de Barcelona, 08193 Bellaterra, Barcelona, SpainInstitute for Biological Research “Siniša Stanković”, University of Belgrade, Bulevar despota Stefana 142, 10060 Belgrade, SerbiaInstitute for Biological Research “Siniša Stanković”, University of Belgrade, Bulevar despota Stefana 142, 10060 Belgrade, SerbiaDepartment of Neurosciences, Biogipuzkoa Health Research Institute, Paseo Dr. Begiristain, s/n, 20014 San Sebastián, Gipuzkoa, SpainDepartment of Neurosciences, Biogipuzkoa Health Research Institute, Paseo Dr. Begiristain, s/n, 20014 San Sebastián, Gipuzkoa, Spain; CIBERNED, ISCIII CIBER, Carlos III Institute, Spanish Ministry of Sciences and Innovation), Av. de Monforte de Lemos, 5, Fuencarral-El Pardo, 28029 Madrid, Spain; Ikerbasque, Basque Foundation for Science, Euskadi Pl., 5, Abando, 48009 Bilbao, Biscay, SpainInstitute of Developmental & Regenerative Medicine, University of Oxford, Campus, Old Rd, Roosevelt Dr, Headington, Oxford OX3 7TY, UKInstitute of Developmental & Regenerative Medicine, University of Oxford, Campus, Old Rd, Roosevelt Dr, Headington, Oxford OX3 7TY, UK; Nuffield Department of Women’s and Reproductive Health, University of Oxford, John Radcliffe Hospital, Oxford OX3 9DU, UKDepartment of Genomic Medicine, Pfizer-University of Granada-Andalusian Regional Government Centre for Genomics and Oncological Research (GENYO), Av. de la Ilustración, 114, 18016 Granada, Spain; Biosanitary Research Institute of Granada (ibs. GRANADA), University of Granada, Av. de Madrid, 15, Beiro, 18012 Granada, Spain; Department of Medicinal & Organic Chemistry and Excellence Research Unit of “Chemistry applied to Bio-medicine and the Environment, ” Faculty of Pharmacy, University of Granada, Campus de Cartuja s/n, 18071 Granada, SpainTechnological, Medical and Academic Park (TMAP), N°109, Abdelkrim Elkhatabi, Bd Abdelkrim Al Khattabi, Marrakech 40000, MoroccoDNA & RNA Medicine Division, Gene Therapy for Rare Diseases Department, Center for Applied Medical Research (CIMA), University of Navarra, IdisNA, Av. de Pío XII, 55, 31008 Pamplona, Navarra, Spain; Vivet Therapeutics, Av. de Pío XII 31, 31008 Pamplona, Navarra, SpainImagine Institute, UMR 163 INSERM, 24 Bd du Montparnasse, 75015 Paris, France; Paris City University, 45 Rue des Saints-Pères, 75006 Paris, FranceBiomedical Research Network on Rare Diseases (CIBERER), C. de Melchor Fernández Almagro, 3, Fuencarral-El Pardo, 28029 Madrid, Spain; Division of Hematopoietic Innovative Therapies, CIEMAT, Av. Complutense, 40, Moncloa - Aravaca, 28040 Madrid, Spain; Advanced Therapies Unit, IIS-Fundación Jimenez Diaz (IIS-FJD, UAM), Av. de los Reyes Católicos, 2, Moncloa - Aravaca, 28040 Madrid, SpainCentro de Biología Molecular Severo Ochoa UAM-CSIC, IUBM, CIBERER, IDIPAZ, Universidad Autónoma de Madrid, C. de Pedro Rico, 6, Fuencarral-El Pardo, 28029 Madrid, SpainLeiden University Medical Center, Department of Cell and Chemical Biology, Einthovenweg 20, 2333 ZC Leiden, the NetherlandsAix-Marseille Universite, CNRS, Centre Interdisciplinaire de Nanoscience de Marseille, UMR 7325, “Equipe Labellisee Ligue Ćontre le Cancer”, Campus de Luminy, case 913, 13009 Marseille, FranceNeuromuscular Unit, Institut de Recerca Sant Joan de Déu, Hospital Sant Joan de Déu, C. de Sta. Rosa, 39, 08950 Barcelona, Spain; Biomedical Research Network on Rare Diseases (CIBERER), C. de Melchor Fernández Almagro, 3, Fuencarral-El Pardo, 28029 Madrid, SpainDepartment of Genomic Medicine, Pfizer-University of Granada-Andalusian Regional Government Centre for Genomics and Oncological Research (GENYO), Av. de la Ilustración, 114, 18016 Granada, Spain; Biosanitary Research Institute of Granada (ibs. GRANADA), University of Granada, Av. de Madrid, 15, Beiro, 18012 Granada, Spain; Department of Biochemistry and Molecular Biology III and Immunology, Faculty of Medicine, University of Granada, Avenida de la Investigación 11, 18016 Granada, SpainInstitute of Developmental & Regenerative Medicine, University of Oxford, Campus, Old Rd, Roosevelt Dr, Headington, Oxford OX3 7TY, UK; Department of Laboratory Medicine, Karolinska Institutet, Alfred Nobels allé 8, 141 52 Huddinge, SwedenDepartment of Synthetic Biology and Immunology, National Institute of Chemistry, Hajdrihova 19, 1000 Ljubljana, Slovenia; Centre for Technologies of Gene and Cell Therapy, National Institute of Chemistry, Hajdrihova 19, 1000 Ljubljana, Slovenia; EN-FIST Centre of Excellence, Trg Osvobodilne fronte 13, 1000 Ljubljana, SloveniaDepartment of Biomedicine, Aarhus University, 8000 Aarhus C, Denmark; Steno Diabetes Center Aarhus, Aarhus University Hospital, 8200 Aarhus N, Denmark; Corresponding author: Yonglun Luo, Department of Biomedicine, Aarhus University, 8000 Aarhus C, Denmark.Department of Genomic Medicine, Pfizer-University of Granada-Andalusian Regional Government Centre for Genomics and Oncological Research (GENYO), Av. de la Ilustración, 114, 18016 Granada, Spain; Corresponding author: Karim Benabdellah, Department of Genomic Medicine, Pfizer-University of Granada-Andalusian Regional Government Centre for Genomics and Oncological Research (GENYO), Av. de la Ilustración, 114, 18016 Granada, Spain.In the past decade, precise targeting through genome editing has emerged as a promising alternative to traditional therapeutic approaches. Genome editing can be performed using various platforms, where programmable DNA nucleases create permanent genetic changes at specific genomic locations due to their ability to recognize precise DNA sequences. Clinical application of this technology requires the delivery of the editing reagents to transplantable cells ex vivo or to tissues and organs for in vivo approaches, often representing a barrier to achieving the desired editing efficiency and safety. In this review, authored by members of the GenE-HumDi European Cooperation in Science and Technology (COST) Action, we described the plethora of delivery systems available for genome-editing components, including viral and non-viral systems, highlighting their advantages, limitations, and potential application in a clinical setting.http://www.sciencedirect.com/science/article/pii/S2162253125000113MT: Delivery StrategiesEuropean Cooperation in Science and TechnologyCOSTGenE-HumDigenome editingbase editors |
| spellingShingle | Alessia Cavazza Francisco J. Molina-Estévez Álvaro Plaza Reyes Victor Ronco Asma Naseem Špela Malenšek Peter Pečan Annalisa Santini Paula Heredia Araceli Aguilar-González Houria Boulaiz Qianqian Ni Marina Cortijo-Gutierrez Kristina Pavlovic Inmaculada Herrera Berta de la Cerda Emilio M. Garcia-Tenorio Eva Richard Sergio Granados-Principal Arístides López-Márquez Mariana Köber Marijana Stojanovic Melita Vidaković Irene Santos-Garcia Lorea Blázquez Emily Haughton Dongnan Yan Rosario María Sánchez-Martín Loubna Mazini Gloria Gonzalez Aseguinolaza Annarita Miccio Paula Rio Lourdes R. Desviat Manuel A.F.V. Gonçalves Ling Peng Cecilia Jiménez-Mallebrera Francisco Martin Molina Dhanu Gupta Duško Lainšček Yonglun Luo Karim Benabdellah Advanced delivery systems for gene editing: A comprehensive review from the GenE-HumDi COST Action Working Group Molecular Therapy: Nucleic Acids MT: Delivery Strategies European Cooperation in Science and Technology COST GenE-HumDi genome editing base editors |
| title | Advanced delivery systems for gene editing: A comprehensive review from the GenE-HumDi COST Action Working Group |
| title_full | Advanced delivery systems for gene editing: A comprehensive review from the GenE-HumDi COST Action Working Group |
| title_fullStr | Advanced delivery systems for gene editing: A comprehensive review from the GenE-HumDi COST Action Working Group |
| title_full_unstemmed | Advanced delivery systems for gene editing: A comprehensive review from the GenE-HumDi COST Action Working Group |
| title_short | Advanced delivery systems for gene editing: A comprehensive review from the GenE-HumDi COST Action Working Group |
| title_sort | advanced delivery systems for gene editing a comprehensive review from the gene humdi cost action working group |
| topic | MT: Delivery Strategies European Cooperation in Science and Technology COST GenE-HumDi genome editing base editors |
| url | http://www.sciencedirect.com/science/article/pii/S2162253125000113 |
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