Integrating Prime Editing and Cellular Reprogramming as Novel Strategies for Genetic Cardiac Disease Modeling and Treatment.

Autor: Yao B; Experimental Cardiology Laboratory, Department of Cardiology, Division of Heart and Lungs, University Medical Center Utrecht, Utrecht, The Netherlands.; Regenerative Medicine Center Utrecht, Circulatory Health Research Center, University Medical Center Utrecht, University Utrecht, Utrecht, The Netherlands., Lei Z; Experimental Cardiology Laboratory, Department of Cardiology, Division of Heart and Lungs, University Medical Center Utrecht, Utrecht, The Netherlands.; Regenerative Medicine Center Utrecht, Circulatory Health Research Center, University Medical Center Utrecht, University Utrecht, Utrecht, The Netherlands.; CDL Research, University Medical Center Utrecht, Utrecht, The Netherlands., Gonçalves MAFV; Department of Cell and Chemical Biology, Leiden University Medical Centre, Leiden, The Netherlands., Sluijter JPG; Experimental Cardiology Laboratory, Department of Cardiology, Division of Heart and Lungs, University Medical Center Utrecht, Utrecht, The Netherlands. j.sluijter@umcutrecht.nl.; Regenerative Medicine Center Utrecht, Circulatory Health Research Center, University Medical Center Utrecht, University Utrecht, Utrecht, The Netherlands. j.sluijter@umcutrecht.nl.

Publikováno v: Current cardiology reports [Curr Cardiol Rep] 2024 Nov; Vol. 26 (11), pp. 1197-1208. Date of Electronic Publication: 2024 Sep 11.

Progress and harmonization of gene editing to treat human diseases: Proceeding of COST Action CA21113 GenE-HumDi.

Autor: Cavazza A; Infection, Immunity and Inflammation Research and Teaching Department, Great Ormond Street Institute of Child Health, University College London, WC1N 1EH London, UK., Hendel A; Institute of Nanotechnology and Advanced Materials, The Mina and Everard Goodman Faculty of Life Sciences, Bar-Ilan University, Ramat-Gan 52900, Israel., Bak RO; Department of Biomedicine, Aarhus University, 8000 Aarhus C, Denmark., Rio P; 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., Güell M; Department of Medicine and Life Sciences, Universitat Pompeu Fabra, Barcelona, Spain.; Integra Therapeutics S.L., Barcelona, Spain., Lainšček D; Department of Synthetic Biology and Immunology, National Institute of Chemistry, Hajdrihova 19, 1000 Ljubljana, Slovenia., Arechavala-Gomeza V; Nucleic Acid Therapeutics for Rare Disorders (NAT-RD), Biobizkaia Health Research Institute, Barakaldo, Spain.; Ikerbasque, Basque Foundation for Science, Bilbao, Spain., Peng L; Aix Marseille University, CNRS, Centre Interdisciplinaire de Nanoscience de Marseille, UMR 7325, Equipe Labellisée Ligue Contre le Cancer, 13288 Marseille, France., Hapil FZ; Department of Medical Biology and Genetics, Faculty of Medicine, Akdeniz University, Antalya, Turkey., Harvey J; Institute of Ophthalmology, University College London, London, UK., Ortega FG; 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., Gonzalez-Martinez C; 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., Lederer CW; Department of Molecular Genetics Thalassaemia, The Cyprus Institute of Neurology and Genetics, Nicosia, Cyprus., Mikkelsen K; Department of Biomedicine, Aarhus University, 8000 Aarhus C, Denmark., Gasiunas G; CasZyme, 10224 Vilnius, Lithuania., Kalter N; Institute of Nanotechnology and Advanced Materials, The Mina and Everard Goodman Faculty of Life Sciences, Bar-Ilan University, Ramat-Gan 52900, Israel., Gonçalves MAFV; Department of Cell and Chemical Biology, Leiden University Medical Center, Einthovenweg 20, 2333 ZC Leiden, the Netherlands., Petersen J; Steno Diabetes Center Aarhus, Aarhus University Hospital, 8200 Aarhus N, Denmark., Garanto A; Department of Pediatrics and Department of Human Genetics, Amalia Children's Hospital, Radboud University Medical Center, Nijmegen, the Netherlands., Montoliu L; 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., Maresca M; Genome Engineering, Discovery Sciences, BioPharmaceuticals R&D Unit, AstraZeneca, Gothenburg, Sweden., Seemann SE; Center for Non-coding RNA in Technology and Health, Department of Veterinary and Animal Sciences, University of Copenhagen, Copenhagen, Denmark., Gorodkin J; Center for Non-coding RNA in Technology and Health, Department of Veterinary and Animal Sciences, University of Copenhagen, Copenhagen, Denmark., Mazini L; Laboratory of Genetic Engineering, Technologic, Medical and Academic Park (TMAP), Marrakech, Morocco., Sanchez R; 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., Rodriguez-Madoz JR; 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., Maldonado-Pérez N; Steno Diabetes Center Aarhus, Aarhus University Hospital, 8200 Aarhus N, Denmark., Laura T; DNA & RNA Medicine Division, Center for Applied Medical Research (CIMA) Universidad de Navarra, 31008 Pamplona, Spain., Schmueck-Henneresse M; Berlin Institute for Health (BIH) at Charité - Universitätsmedizin Berlin, BIH Center for Regenerative Therapies (BCRT), Charitéplatz 1, 10117 Berlin, Germany., Maccalli C; Laboratory of Immune Biological Therapy, Research Branch, Sidra Medicine, PO Box 26999, Doha, Qatar., Grünewald J; 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., Carmona G; Red Andaluza de diseño y traslación de Terapias Avanzadas-RAdytTA, Fundación Pública Andaluza Progreso y Salud-FPS, Sevilla, España., Kachamakova-Trojanowska N; Malopolska Centre of Biotechnology, Jagiellonian University, 30-387 Krakow, Poland., Miccio A; Imagine Institute, Laboratory of Chromatin and Gene Regulation During Development, Université de Paris Cité, INSERM UMR 1163, 75015 Paris, France., Martin F; 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., Turchiano G; Infection, Immunity and Inflammation Research and Teaching Department, Great Ormond Street Institute of Child Health, University College London, WC1N 1EH London, UK., Cathomen T; Institute for Transfusion Medicine and Gene Therapy, Medical Center-University of Freiburg, Freiburg, Germany.; Medical Faculty, University of Freiburg, 79106 Freiburg, Germany., Luo Y; Department of Biomedicine, Aarhus University, 8000 Aarhus C, Denmark.; Steno Diabetes Center Aarhus, Aarhus University Hospital, 8200 Aarhus N, Denmark., Tsai SQ; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA., Benabdellah K; 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.

Publikováno v: Molecular therapy. Nucleic acids [Mol Ther Nucleic Acids] 2023 Oct 29; Vol. 34, pp. 102066. Date of Electronic Publication: 2023 Oct 29 (Print Publication: 2023).

Efficient and scalable generation of primordial germ cells in 2D culture using basement membrane extract overlay.

Autor: Overeem AW; Department of Anatomy and Embryology, Leiden University Medical Center, 2333 ZC Leiden, the Netherlands., Chang YW; Department of Anatomy and Embryology, Leiden University Medical Center, 2333 ZC Leiden, the Netherlands., Moustakas I; Department of Anatomy and Embryology, Leiden University Medical Center, 2333 ZC Leiden, the Netherlands.; Sequencing Analysis Support Core, Leiden University Medical Center, 2333 ZC Leiden, the Netherlands., Roelse CM; Department of Anatomy and Embryology, Leiden University Medical Center, 2333 ZC Leiden, the Netherlands., Hillenius S; Department of Anatomy and Embryology, Leiden University Medical Center, 2333 ZC Leiden, the Netherlands., Helm TV; Department of Anatomy and Embryology, Leiden University Medical Center, 2333 ZC Leiden, the Netherlands., Schrier VFV; Department of Anatomy and Embryology, Leiden University Medical Center, 2333 ZC Leiden, the Netherlands., Gonçalves MAFV; Department of Cell and Chemical Biology, Leiden University Medical Center, 2333 ZC Leiden, the Netherlands., Mei H; Sequencing Analysis Support Core, Leiden University Medical Center, 2333 ZC Leiden, the Netherlands., Freund C; Department of Anatomy and Embryology, Leiden University Medical Center, 2333 ZC Leiden, the Netherlands.; Leiden University Medical Center hiPSC Hotel, Leiden University Medical Centre, 2333 ZC Leiden, the Netherlands., Chuva de Sousa Lopes SM; Department of Anatomy and Embryology, Leiden University Medical Center, 2333 ZC Leiden, the Netherlands.; Department for Reproductive Medicine, Ghent University Hospital, 9000 Ghent, Belgium.

Publikováno v: Cell reports methods [Cell Rep Methods] 2023 May 23; Vol. 3 (6), pp. 100488. Date of Electronic Publication: 2023 May 23 (Print Publication: 2023).

High-capacity adenovector delivery of forced CRISPR-Cas9 heterodimers fosters precise chromosomal deletions in human cells.

Autor: Tasca F; Department of Cell and Chemical Biology, Leiden University Medical Center, Einthovenweg 20, 2333 ZC Leiden, the Netherlands., Brescia M; Department of Cell and Chemical Biology, Leiden University Medical Center, Einthovenweg 20, 2333 ZC Leiden, the Netherlands., Liu J; Department of Cell and Chemical Biology, Leiden University Medical Center, Einthovenweg 20, 2333 ZC Leiden, the Netherlands., Janssen JM; Department of Cell and Chemical Biology, Leiden University Medical Center, Einthovenweg 20, 2333 ZC Leiden, the Netherlands., Mamchaoui K; Sorbonne Université, Inserm, Institut de Myologie, Centre de Recherche en Myologie, Paris, France., Gonçalves MAFV; Department of Cell and Chemical Biology, Leiden University Medical Center, Einthovenweg 20, 2333 ZC Leiden, the Netherlands.

Publikováno v: Molecular therapy. Nucleic acids [Mol Ther Nucleic Acids] 2023 Feb 22; Vol. 31, pp. 746-762. Date of Electronic Publication: 2023 Feb 22 (Print Publication: 2023).

Integrating gene delivery and gene-editing technologies by adenoviral vector transfer of optimized CRISPR-Cas9 components.

Autor: Maggio I; Department of Pediatrics/Willem-Alexander Kinderziekenhuis (WAKZ), Leiden University Medical Center, Albinusdreef 2, 2300 RC, Leiden, The Netherlands.; Department of Cell and Chemical Biology (CCB), Leiden University Medical Center, Einthovenweg 20, 2333 ZC, Leiden, The Netherlands., Zittersteijn HA; Department of Cell and Chemical Biology (CCB), Leiden University Medical Center, Einthovenweg 20, 2333 ZC, Leiden, The Netherlands., Wang Q; Department of Cell and Chemical Biology (CCB), Leiden University Medical Center, Einthovenweg 20, 2333 ZC, Leiden, The Netherlands., Liu J; Department of Cell and Chemical Biology (CCB), Leiden University Medical Center, Einthovenweg 20, 2333 ZC, Leiden, The Netherlands., Janssen JM; Department of Cell and Chemical Biology (CCB), Leiden University Medical Center, Einthovenweg 20, 2333 ZC, Leiden, The Netherlands., Ojeda IT; Department of Cell and Chemical Biology (CCB), Leiden University Medical Center, Einthovenweg 20, 2333 ZC, Leiden, The Netherlands., van der Maarel SM; Department of Human Genetics, Leiden University Medical Centre, Einthovenweg 20, 2333 ZC, Leiden, The Netherlands., Lankester AC; Department of Pediatrics/Willem-Alexander Kinderziekenhuis (WAKZ), Leiden University Medical Center, Albinusdreef 2, 2300 RC, Leiden, The Netherlands., Hoeben RC; Department of Cell and Chemical Biology (CCB), Leiden University Medical Center, Einthovenweg 20, 2333 ZC, Leiden, The Netherlands., Gonçalves MAFV; Department of Cell and Chemical Biology (CCB), Leiden University Medical Center, Einthovenweg 20, 2333 ZC, Leiden, The Netherlands. M.F.V.Goncalves@lumc.nl.

Publikováno v: Gene therapy [Gene Ther] 2020 May; Vol. 27 (5), pp. 209-225. Date of Electronic Publication: 2020 Jan 03.