Molecular Therapy: Nucleic Acids (Dec 2023)
Progress and harmonization of gene editing to treat human diseases: Proceeding of COST Action CA21113 GenE-HumDi
- Alessia Cavazza,
- Ayal Hendel,
- Rasmus O. Bak,
- Paula Rio,
- Marc Güell,
- Duško Lainšček,
- Virginia Arechavala-Gomeza,
- Ling Peng,
- Fatma Zehra Hapil,
- Joshua Harvey,
- Francisco G. Ortega,
- Coral Gonzalez-Martinez,
- Carsten W. Lederer,
- Kasper Mikkelsen,
- Giedrius Gasiunas,
- Nechama Kalter,
- Manuel A.F.V. Gonçalves,
- Julie Petersen,
- Alejandro Garanto,
- Lluis Montoliu,
- Marcello Maresca,
- Stefan E. Seemann,
- Jan Gorodkin,
- Loubna Mazini,
- Rosario Sanchez,
- Juan R. Rodriguez-Madoz,
- Noelia Maldonado-Pérez,
- Torella Laura,
- Michael Schmueck-Henneresse,
- Cristina Maccalli,
- Julian Grünewald,
- Gloria Carmona,
- Neli Kachamakova-Trojanowska,
- Annarita Miccio,
- Francisco Martin,
- Giandomenico Turchiano,
- Toni Cathomen,
- Yonglun Luo,
- Shengdar Q. Tsai,
- Karim Benabdellah
Affiliations
- Alessia Cavazza
- Infection, Immunity and Inflammation Research and Teaching Department, Great Ormond Street Institute of Child Health, University College London, WC1N 1EH London, UK; Corresponding author: Cavazza Alessia, Infection, Immunity and Inflammation Research and Teaching Department, Great Ormond Street Institute of Child Health, University College London, London, UK.
- Ayal Hendel
- Institute of Nanotechnology and Advanced Materials, The Mina and Everard Goodman Faculty of Life Sciences, Bar-Ilan University, Ramat-Gan 52900, Israel; Corresponding author: Ayal Hendel, Institute of Nanotechnology and Advanced Materials, The Mina and Everard Goodman Faculty of Life Sciences, Bar-Ilan University, Ramat-Gan 52900, Israel.
- Rasmus O. Bak
- Department of Biomedicine, Aarhus University, 8000 Aarhus C, Denmark; Corresponding author: Rasmus O. Bak, Department of Biomedicine, Aarhus University, 8000 Aarhus C, Denmark.
- Paula Rio
- Hematopoietic Innovative Therapies Division, Centro de Investigaciones Energéticas Medioambientales y Tecnológicas and Centro de Investigación Biomédica en Red de Enfermedades Raras (CIEMAT/CIBERER), 28040 Madrid, Spain; Advanced Therapies Unit, Instituto de Investigación Sanitaria Fundación Jiménez Díaz (IIS-FJD, UAM), 28040 Madrid, Spain
- Marc Güell
- Department of Medicine and Life Sciences, Universitat Pompeu Fabra, Barcelona, Spain; Integra Therapeutics S.L., Barcelona, Spain
- Duško Lainšček
- Department of Synthetic Biology and Immunology, National Institute of Chemistry, Hajdrihova 19, 1000 Ljubljana, Slovenia
- Virginia Arechavala-Gomeza
- Nucleic Acid Therapeutics for Rare Disorders (NAT-RD), Biobizkaia Health Research Institute, Barakaldo, Spain; Ikerbasque, Basque Foundation for Science, Bilbao, Spain
- Ling Peng
- Aix Marseille University, CNRS, Centre Interdisciplinaire de Nanoscience de Marseille, UMR 7325, Equipe Labellisée Ligue Contre le Cancer, 13288 Marseille, France
- Fatma Zehra Hapil
- Department of Medical Biology and Genetics, Faculty of Medicine, Akdeniz University, Antalya, Turkey
- Joshua Harvey
- Institute of Ophthalmology, University College London, London, UK
- Francisco G. Ortega
- GENYO, Centre for Genomics and Oncological Research, Pfizer/University of Granada/Andalusian Regional Government, Avenida de la Ilustración 114, 18016 Granada, Spain; IBS Granada, Institute of Biomedical Research, Avenida de Madrid 15, 18012 Granada, Spain
- Coral Gonzalez-Martinez
- GENYO, Centre for Genomics and Oncological Research, Pfizer/University of Granada/Andalusian Regional Government, Avenida de la Ilustración 114, 18016 Granada, Spain; IBS Granada, Institute of Biomedical Research, Avenida de Madrid 15, 18012 Granada, Spain
- Carsten W. Lederer
- Department of Molecular Genetics Thalassaemia, The Cyprus Institute of Neurology and Genetics, Nicosia, Cyprus
- Kasper Mikkelsen
- Department of Biomedicine, Aarhus University, 8000 Aarhus C, Denmark
- Giedrius Gasiunas
- CasZyme, 10224 Vilnius, Lithuania
- Nechama Kalter
- Institute of Nanotechnology and Advanced Materials, The Mina and Everard Goodman Faculty of Life Sciences, Bar-Ilan University, Ramat-Gan 52900, Israel
- Manuel A.F.V. Gonçalves
- Department of Cell and Chemical Biology, Leiden University Medical Center, Einthovenweg 20, 2333 ZC Leiden, the Netherlands
- Julie Petersen
- Steno Diabetes Center Aarhus, Aarhus University Hospital, 8200 Aarhus N, Denmark
- Alejandro Garanto
- Department of Pediatrics and Department of Human Genetics, Amalia Children’s Hospital, Radboud University Medical Center, Nijmegen, the Netherlands
- Lluis Montoliu
- Department of Molecular and Cellular Biology, National Centre for Biotechnology (CNB-CSIC) and Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER-ISCIII), Madrid, Spain
- Marcello Maresca
- Genome Engineering, Discovery Sciences, BioPharmaceuticals R&D Unit, AstraZeneca, Gothenburg, Sweden
- Stefan E. Seemann
- Center for Non-coding RNA in Technology and Health, Department of Veterinary and Animal Sciences, University of Copenhagen, Copenhagen, Denmark
- Jan Gorodkin
- Center for Non-coding RNA in Technology and Health, Department of Veterinary and Animal Sciences, University of Copenhagen, Copenhagen, Denmark
- Loubna Mazini
- Laboratory of Genetic Engineering, Technologic, Medical and Academic Park (TMAP), Marrakech, Morocco
- Rosario Sanchez
- GENYO, Centre for Genomics and Oncological Research, Pfizer/University of Granada/Andalusian Regional Government, PTS Granada, Granada, Spain; Department of Medicinal & Organic Chemistry and Excellence Research Unit of ''Chemistry Applied to Biomedicine and the Environment,'' Faculty of Pharmacy, University of Granada, Campus de Cartuja s/n, Granada, Spain; Instituto de Investigación Biosanitaria de Granada (ibs.GRANADA), Universidad de Granada, Granada, Spain
- Juan R. Rodriguez-Madoz
- Cancer Center Clinica Universidad de Navarra (CCUN), Pamplona, Spain; Instituto de Investigación Sanitaria de Navarra (IdiSNA), Pamplona, Spain; Centro de Investigación Biomédica en Red de Cancer (CIBERONC), Madrid, Spain
- Noelia Maldonado-Pérez
- Steno Diabetes Center Aarhus, Aarhus University Hospital, 8200 Aarhus N, Denmark
- Torella Laura
- DNA & RNA Medicine Division, Center for Applied Medical Research (CIMA) Universidad de Navarra, 31008 Pamplona, Spain
- Michael Schmueck-Henneresse
- Berlin Institute for Health (BIH) at Charité - Universitätsmedizin Berlin, BIH Center for Regenerative Therapies (BCRT), Charitéplatz 1, 10117 Berlin, Germany
- Cristina Maccalli
- Laboratory of Immune Biological Therapy, Research Branch, Sidra Medicine, PO Box 26999, Doha, Qatar
- Julian Grünewald
- Department of Medicine, Cardiology, Angiology, Pneumology, Klinikum rechts der Isar, Technical University of Munich, TUM School of Medicine and Health, TranslaTUM, MIBE, Munich, Germany; Center for Organoid Systems, Munich, Germany
- Gloria Carmona
- Red Andaluza de diseño y traslación de Terapias Avanzadas-RAdytTA, Fundación Pública Andaluza Progreso y Salud-FPS, Sevilla, España
- Neli Kachamakova-Trojanowska
- Malopolska Centre of Biotechnology, Jagiellonian University, 30-387 Krakow, Poland
- Annarita Miccio
- Imagine Institute, Laboratory of Chromatin and Gene Regulation During Development, Université de Paris Cité, INSERM UMR 1163, 75015 Paris, France
- Francisco Martin
- Bioquímica y Biología Molecular III e Immunology Department, Facultad de Medicina, Universidad de Granada and Department of Genomic Medicine, Pfizer-University of Granada-Andalusian Regional Government Centre for Genomics and Oncological Research (GENYO), PTS, Av. de la Ilustración 114, 18016 Granada, Spain
- Giandomenico Turchiano
- Infection, Immunity and Inflammation Research and Teaching Department, Great Ormond Street Institute of Child Health, University College London, WC1N 1EH London, UK
- Toni Cathomen
- Institute for Transfusion Medicine and Gene Therapy, Medical Center-University of Freiburg, Freiburg, Germany; Medical Faculty, University of Freiburg, 79106 Freiburg, Germany
- Yonglun Luo
- Department 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, Denmark.
- Shengdar Q. Tsai
- Department of Hematology, St. Jude Children’s Research Hospital, Memphis, TN, USA; Corresponding author: Shengdar Q. Tsai, Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA.
- Karim Benabdellah
- Department of Genomic Medicine, Pfizer-University of Granada-Andalusian Regional Government Centre for Genomics and Oncological Research (GENYO), PTS, Av. de la Ilustración 114, 18016 Granada, Spain; Corresponding author: Karim Benabdellah, GENYO, Centre for Genomics and Oncological Research, Pfizer/University of Granada/Andalusian Regional Government, Avenida de la Ilustración 114, 18016 Granada, Spain.
- Journal volume & issue
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Vol. 34
p. 102066
Abstract
The European Cooperation in Science and Technology (COST) is an intergovernmental organization dedicated to funding and coordinating scientific and technological research in Europe, fostering collaboration among researchers and institutions across countries. Recently, COST Action funded the ''Genome Editing to treat Human Diseases'' (GenE-HumDi) network, uniting various stakeholders such as pharmaceutical companies, academic institutions, regulatory agencies, biotech firms, and patient advocacy groups. GenE-HumDi’s primary objective is to expedite the application of genome editing for therapeutic purposes in treating human diseases. To achieve this goal, GenE-HumDi is organized in several working groups, each focusing on specific aspects. These groups aim to enhance genome editing technologies, assess delivery systems, address safety concerns, promote clinical translation, and develop regulatory guidelines. The network seeks to establish standard procedures and guidelines for these areas to standardize scientific practices and facilitate knowledge sharing. Furthermore, GenE-HumDi aims to communicate its findings to the public in accessible yet rigorous language, emphasizing genome editing’s potential to revolutionize the treatment of many human diseases. The inaugural GenE-HumDi meeting, held in Granada, Spain, in March 2023, featured presentations from experts in the field, discussing recent breakthroughs in delivery methods, safety measures, clinical translation, and regulatory aspects related to gene editing.
