Unlocking Precision Gene Therapy

Rezultaty

First report on networking, public engagement and communication activities
Third report on networking, public engagement and communication activities

Third report on networking public engagement and communication activities

Second report on networking, public engagement and communication activities

Second report on networking public engagement and communication activities

Fourth report on networking, public engagement and communication activities

Fourth report on networking public engagement and communication activities

Website and intranet portal
UPGRADE dissemination plan

Release of the Data Management plan

Publikacje

BAR-Seq clonal tracking of gene-edited cells

Autorzy: Ferrari, Samuele; Beretta, Stefano; Jacob, Aurelien; Cittaro, Davide; Albano, Luisa; Merelli, Ivan; Naldini, Luigi; Genovese, Pietro
Opublikowane w: Nature Protocols, Numer 16, 2021, Strona(/y) 2991-3025, ISSN 1754-2189
Wydawca: Nature Publishing Group
DOI: 10.1038/s41596-021-00529-x

FoxO maintains a genuine muscle stem-cell quiescent state until geriatric age.

Autorzy: García-Prat, Laura; Perdiguero, Eusebio; Alonso-Martín, Sonia; Dell'Orso, Stefania; Ravichandran, Srikanth; Brooks, Stephen R.; Juan, Aster H.; Campanario, Silvia; Jiang, Kan; Hong, Xiaotong; Ortet, Laura; Ruiz-Bonilla, Vanessa; Flández, Marta; Moiseeva, Victoria; Rebollo, Elena; Jardí, Mercè; Sun, Hong-Wei; Musarò, Antonio; Sandri, Marco; del Sol, Antonio; Sartorelli, Vittorio; Muñoz-Cáno
Opublikowane w: Nature Cell Biology, Numer 22, 2020, Strona(/y) 1307-1318, ISSN 1465-7392
Wydawca: Nature Publishing Group
DOI: 10.1038/s41556-020-00593-7

A heterodimer of evolved designer-recombinases precisely excises a human genomic DNA locus

Autorzy: Felix Lansing, Maciej Paszkowski-Rogacz, Lukas Theo Schmitt, Paul Martin Schneider, Teresa Rojo Romanos, Jan Sonntag, Frank Buchholz
Opublikowane w: Nucleic Acids Research, Numer 48/1, 2019, Strona(/y) 472-485, ISSN 0305-1048
Wydawca: Oxford University Press
DOI: 10.1093/nar/gkz1078

Nearest-neighbor amino acids of specificity-determining residues influence the activity of engineered Cre-type recombinases

Autorzy: Anjali Soni, Martina Augsburg, Frank Buchholz, M. Teresa Pisabarro
Opublikowane w: Scientific Reports, Numer 10/1, 2020, ISSN 2045-2322
Wydawca: Nature Publishing Group
DOI: 10.1038/s41598-020-70867-5

Delivery of Cas9/sgRNA Ribonucleoprotein Complexes via Hydroxystearyl Oligoamino Amides

Autorzy: Jasmin Kuhn, Yi Lin, Ana Krhac Levacic, Nader Al Danaf, Lun Peng, Miriam Höhn, Don C. Lamb, Ernst Wagner, Ulrich Lächelt
Opublikowane w: Bioconjugate Chemistry, Numer 31/3, 2020, Strona(/y) 729-742, ISSN 1043-1802
Wydawca: American Chemical Society
DOI: 10.1021/acs.bioconjchem.9b00853

Muscle stem cell aging: identifying ways to induce tissue rejuvenation

Autorzy: Pedro Sousa-Victor, Joana Neves, Pura Muñoz-Cánoves
Opublikowane w: Mechanisms of Ageing and Development, Numer 188, 2020, Strona(/y) 111246, ISSN 0047-6374
Wydawca: Elsevier BV
DOI: 10.1016/j.mad.2020.111246

Gene Therapy for Cystic Fibrosis: Progress and Challenges of Genome Editing

Autorzy: Maule, Giulia; Arosio, Daniele; Cereseto, Anna
Opublikowane w: International Journal of Molecular Science, 2020, ISSN 1422-0067
Wydawca: Multidisciplinary Digital Publishing Institute (MDPI)
DOI: 10.3390/ijms21113903

Efficient and gentle delivery of molecules into cells with different elasticity via Progressive Mechanoporation

Autorzy: Uvizl, Alena; Goswami, Ruchi; Gandhi, Shanil Durgeshkumar; Augsburg, Martina; Buchholz, Frank; Guck, Jochen; Mansfeld, Jörg; Girardo, Salvatore
Opublikowane w: Lab on a chip, Numer 12, 2020, ISSN 1473-0197
Wydawca: Royal Society of Chemistry
DOI: 10.1039/d0lc01224f

Efficient gene editing of human long-term hematopoietic stem cells validated by clonal tracking

Autorzy: Samuele Ferrari, Aurelien Jacob, Stefano Beretta, Giulia Unali, Luisa Albano, Valentina Vavassori, Davide Cittaro, Dejan Lazarevic, Chiara Brombin, Federica Cugnata, Anna Kajaste-Rudnitski, Ivan Merelli, Pietro Genovese, Luigi Naldini
Opublikowane w: Nature Biotechnology, Numer 38/11, 2020, Strona(/y) 1298-1308, ISSN 1087-0156
Wydawca: Nature Publishing Group
DOI: 10.1038/s41587-020-0551-y

Optimizing synthetic nucleic acid and protein nanocarriers: The chemical evolution approach

Autorzy: Franziska Freitag, Ernst Wagner
Opublikowane w: Advanced Drug Delivery Reviews, 2020, ISSN 0169-409X
Wydawca: Elsevier BV
DOI: 10.1016/j.addr.2020.03.005

Glucose 6-P dehydrogenase delays the onset of frailty by protecting against muscle damage

Autorzy: Arc-Chagnaud, Coralie; Salvador-Pascual, Andrea; Garcia-Dominguez, Esther; Olaso-Gonzalez, Gloria; Correas, Angela G.; Serna, Eva; Brioche, Thomas; Chopard, Angele; Fernandez-Marcos, Pablo J.; Serrano, Manuel; Serrano, Antonio L.; Muñoz-Cánoves, Pura; Sebastiá, Vicente; Viña, Jose; Gomez-Cabrera, Mari Carmen
Opublikowane w: Journal of Cachexia, Sarcopenia and Muscles, Numer 12, 2021, Strona(/y) 1879–1896, ISSN 2190-6009
Wydawca: Wiley and Sons
DOI: 10.1002/jcsm.12792

Find and cut-and-transfer (FiCAT) mammalian genome engineering

Autorzy: Pallarès-Masmitjà, Maria; Ivančić, Dimitrije; Mir-Pedrol, Júlia; Jaraba-Wallace, Jessica; Tagliani, Tommaso; Oliva, Baldomero; Rahmeh, Amal; Sánchez-Mejías, Avencia; Güell, Marc
Opublikowane w: Nature Communications, 2021, ISSN 2041-1723
Wydawca: Nature Publishing Group
DOI: 10.1038/s41467-021-27183-x

Correction of a Factor VIII genomic inversion with designer-recombinases

Autorzy: Lansing, Felix; Mukhametzyanova, Liliya; Rojo-Romanos, Teresa; Iwasawa, Kentaro; Kimura, Masaki; Paszkowski-Rogacz, Maciej; Karpinski, Janet; Grass, Tobias; Sonntag, Jan; Schneider, Paul Martin; Günes, Ceren; Hoersten, Jenna; Schmitt, Lukas Theo; Rodriguez-Muela, Natalia; Knöfler, Ralf; Takebe, Takanori; Buchholz, Frank
Opublikowane w: Nature Communications, Numer 13, 2022, ISSN 2041-1723
Wydawca: Nature Publishing Group
DOI: 10.1038/s41467-022-28080-7

Integration of feeding behavior by the liver circadian clock reveals network dependency of metabolic rhythms

Autorzy: Greco, Carolina M.; Koronowski, Kevin B.; Smith, Jacob G.; Shi, Jiejun; Kunderfranco, Paolo; Carriero, Roberta; Chen, Siwei; Samad, Muntaha; Welz, Patrick-Simon; Zinna, Valentina M.; Mortimer, Thomas; Chun, Sung Kook; Shimaji, Kohei; Sato, Tomoki; Petrus, Paul; Kumar, Arun; Vaca-Dempere, Mireia; Deryagin, Oleg; Van, Cassandra; Monroy Kuhn, José Manuel; Lutter, Dominik; Sel
Opublikowane w: Science Advances, Numer 7 (39), 2021, ISSN 2375-2548
Wydawca: American Association for the Advancement of Science
DOI: 10.1126/sciadv.abi7828

Sox17 Controls Emergence and Remodeling of Nestin-Expressing Coronary Vessels

Autorzy: González-Hernández, Sara; Gómez, Manuel J.; Sánchez-Cabo, Fátima; Méndez-Ferrer, Simón; Muñoz-Cánoves, Pura; Isern, Joan
Opublikowane w: Circulation Research, Numer 127, 2020, Strona(/y) e252-e270, ISSN 0009-7330
Wydawca: Lippincott Williams & Wilkins Ltd.
DOI: 10.1161/circresaha.120.317121

Translational control by DHX36 binding to 5′UTR G-quadruplex is essential for muscle stem-cell regenerative functions

Autorzy: Xiaona Chen, Jie Yuan, Guang Xue, Silvia Campanario, Di Wang, Wen Wang, Xi Mou, Shiau Wei Liew, Mubarak Ishaq Umar, Joan Isern, Yu Zhao, Liangqiang He, Yuying Li, Christopher J. Mann, Xiaohua Yu, Lei Wang, Eusebio Perdiguero, Wei Chen, Yuanchao Xue, Yoshikuni Nagamine, Chun Kit Kwok, Hao Sun, Pura Muñoz-Cánoves & Huating Wang
Opublikowane w: Nature Communications, Numer 12, 2021, ISSN 2041-1723
Wydawca: Nature Publishing Group
DOI: 10.1038/s41467-021-25170-w

Modeling, optimization, and comparable efficacy of T cell and hematopoietic stem cell gene editing for treating hyper-IgM syndrome

Autorzy: Vavassori, Valentina; Mercuri, Elisabetta; Marcovecchio, Genni E.; Castiello, Maria C.; Schiroli, Giulia; Albano, Luisa; Margulies, Carrie; Buquicchio, Frank; Fontana, Elena; Beretta, Stefano; Merelli, Ivan; Cappelleri, Andrea; Rancoita, Paola M.V.; Lougaris, Vassilios; Plebani, Alessandro; Kanariou, Maria; Lankester, Arjan; Ferrua, Francesca; Scanziani, Eugenio; Cotta-Ramusino, Cecilia; Villa, An
Opublikowane w: EMBO Molecular Medicine, Numer 13(3), 2021, ISSN 1757-4676
Wydawca: John Wiley & Sons Ltd.
DOI: 10.15252/emmm.202013545

Risk Mitigation of Immunogenicity: A Key to Personalized Retinal Gene Therapy

Autorzy: Varin, Juliette; Morival, Clément; Maillard, Noémien; Adjali, Oumeya; Cronin, Therese
Opublikowane w: International Journal of Molecular Science, Numer 22, 2021, ISSN 1422-0067
Wydawca: Multidisciplinary Digital Publishing Institute (MDPI)
DOI: 10.3390/ijms222312818

Gene Editing of Hematopoietic Stem Cells: Hopes and Hurdles Toward Clinical Translation

Autorzy: Ferrari, Samuele; Vavassori, Valentina; Canarutto, Daniele; Jacob, Aurelien; Castiello, Maria Carmina; Javed, Attya Omer; Genovese, Pietro
Opublikowane w: Frontiers in Genome Editing, 2021, ISSN 2673-3439
Wydawca: Frontiers Media S.A.
DOI: 10.3389/fgeed.2021.618378

Protocol for RNA-seq library preparation starting from a rare muscle stem cell population or a limited number of mouse embryonic stem cells

Autorzy: Dell'Orso, Stefania; Juan, Aster H.; Moiseeva, Victoria; Garcia-Prat, Laura; Munoz-Cannoves, Pura; Sartorelli, Vittorio
Opublikowane w: STAR Protocols, Numer 2, 2021, ISSN 2666-1667
Wydawca: Elsevier
DOI: 10.1016/j.xpro.2021.100451

Computational modelling of stem cell–niche interactions facilitates discovery of strategies to enhance tissue regeneration and counteract ageing

Autorzy: Potapov, Ilya; García-Prat, Laura; Ravichandran, Srikanth; Muñoz-Cánoves, Pura; del Sol, Antonio
Opublikowane w: The FEBS Journal, 2021, ISSN 1742-4658
Wydawca: John Wiley & Sons
DOI: 10.1111/febs.15832

CHD4 ensures stem cell lineage fidelity during skeletal muscle regeneration

Autorzy: Sreenivasan, Krishnamoorthy; Rodríguez-delaRosa, Alejandra; Johnny, Kim; Mesquita, Diana; Segalés, Jessica; Gomez-del Arco, Pablo; Espejo, Isabel; Ianni, Alessandro; Di Croce, Luciano; Relaix, Frederic; Redondo, Juan Miguel; Braun, Thomas; Serrano, Antonio L; Perdiguero, Eusebio; Muñoz-Cánoves, Pura
Opublikowane w: Stem Cell Reports, Numer 16, 9, 2021, ISSN 2213-6711
Wydawca: Cell Press
DOI: 10.1016/j.stemcr.2021.07.022

Efficient Generation and Correction of Mutations in Human iPS Cells Utilizing mRNAs of CRISPR Base Editors and Prime Editors

Autorzy: Duran Sürün, Aksana Schneider, Jovan Mircetic, Katrin Neumann, Felix Lansing, Maciej Paszkowski-Rogacz, Vanessa Hänchen, Min Ae Lee-Kirsch, Frank Buchholz
Opublikowane w: Genes, Numer 11/5, 2020, Strona(/y) 511, ISSN 2073-4425
Wydawca: Multidisciplinary Digital Publishing Institute (MDPI)
DOI: 10.3390/genes11050511

Tetramer-Based Enrichment of Preexisting Anti-AAV8 CD8+ T Cells in Human Donors Allows the Detection of a TEMRA Subpopulation

Autorzy: Céline Vandamme, Rebecca Xicluna, Leslie Hesnard, Marie Devaux, Nicolas Jaulin, Mickaël Guilbaud, Johanne Le Duff, Célia Couzinié, Philippe Moullier, Xavier Saulquin, Oumeya Adjali
Opublikowane w: Frontiers in Immunology, Numer 10, 2020, ISSN 1664-3224
Wydawca: Frontiers
DOI: 10.3389/fimmu.2019.03110

Cripto shapes macrophage plasticity and restricts EndMT in injured and diseased skeletal muscle

Autorzy: Francescopaolo Iavarone, Ombretta Guardiola, Alessandra Scagliola, Gennaro Andolfi, Federica Esposito, Antonio Serrano, Eusebio Perdiguero, Silvia Brunelli, Pura Muñoz‐Cánoves, Gabriella Minchiotti
Opublikowane w: EMBO reports, Numer 21/4, 2020, ISSN 1469-221X
Wydawca: Nature Publishing Group
DOI: 10.15252/embr.201949075

Sestrin prevents atrophy of disused and aging muscles by integrating anabolic and catabolic signals

Autorzy: Jessica Segalés, Eusebio Perdiguero, Antonio L. Serrano, Pedro Sousa-Victor, Laura Ortet, Mercè Jardí, Andrei V. Budanov, Laura Garcia-Prat, Marco Sandri, David M. Thomson, Michael Karin, Jun Hee Lee, Pura Muñoz-Cánoves
Opublikowane w: Nature Communications, Numer 11/1, 2020, ISSN 2041-1723
Wydawca: Nature Publishing Group
DOI: 10.1038/s41467-019-13832-9

Comprehensive transcriptome-wide analysis of spliceopathy correction of myotonic dystrophy using CRISPR-Cas9 in iPSCs-derived cardiomyocytes

Autorzy: Dastidar, Sumitava; Majumdar, Debanjana; Tipanee, Jaitip; Singh, Kshitiz; Klein, Arnaud F.; Furling, Denis; Chuah, Marinee K.; VandenDriessche, Thierry
Opublikowane w: Molecular Therapy, Numer 30 (1), 2022, ISSN 1525-0016
Wydawca: Nature Publishing Group
DOI: 10.1016/j.ymthe.2021.08.004

Attenuated Epigenetic Suppression of Muscle Stem Cell Necroptosis Is Required for Efficient Regeneration of Dystrophic Muscles

Autorzy: Krishnamoorthy Sreenivasan, Alessandro Ianni, Carsten Künne, Boris Strilic, Stefan Günther, Eusebio Perdiguero, Marcus Krüger, Simone Spuler, Stefan Offermanns, Pablo Gómez-del Arco, Juan Miguel Redondo, Pura Munoz-Canoves, Johnny Kim, Thomas Braun
Opublikowane w: Cell Reports, Numer 31/7, 2020, Strona(/y) 107652, ISSN 2211-1247
Wydawca: Cell Press
DOI: 10.1016/j.celrep.2020.107652

Has retinal gene therapy come of age? From bench to bedside and back to bench

Autorzy: Ivana Trapani, Alberto Auricchio
Opublikowane w: Human Molecular Genetics, Numer 28/R1, 2019, Strona(/y) R108-R118, ISSN 0964-6906
Wydawca: Oxford University Press
DOI: 10.1093/hmg/ddz130

Pairing of single mutations yields obligate Cre-type site-specific recombinases

Autorzy: Hoersten, Jenna; Ruiz-Gómez, Gloria; Lansing, Felix; Rojo-Romanos, Teresa; Schmitt, Lukas Theo; Sonntag, Jan; Pisabarro, M Teresa; Buchholz, Frank
Opublikowane w: Nucleic Acids Research, Numer 50(2), 2022, Strona(/y) 1174–1186, ISSN 0305-1048
Wydawca: Oxford University Press
DOI: 10.1093/nar/gkab1240

A Network of Macrophages Supports Mitochondrial Homeostasis in the Heart

Autorzy: Nicolás-Ávila, José Ángel; Lechuga-Vieco, Ana Victoria; Esteban-Martínez, Lorena; Sánchez-Díaz, María; Díaz-García, Elena; Santiago, Demetrio J.; Rubio-Ponce, Andrea; LiangYao Li, Jackson; Balachander, Akhila; Quintana, Juan A.; Martínez-de-Mena, Raquel; Castejón-Vega, Beatriz; Pun-García, Andrés; Través, Paqui G; Bonzón-Kulichenko, Elena; García-Marqués, Fernando; Cussó, Lorena
Opublikowane w: Cell, 2020, ISSN 0092-8674
Wydawca: Cell Press
DOI: 10.1016/j.cell.2020.08.031

Assessing Autophagy in Muscle Stem Cells

Autorzy: Campanario, Silvia; Ramírez-Pardo, Ignacio; Hong, Xiaotong; Isern, Joan; Muñoz-Cánoves, Pura
Opublikowane w: Frontiers in Cell and Developmental Biology, 2021, ISSN 2296-634X
Wydawca: Frontiers
DOI: 10.3389/fcell.2020.620409

Understanding muscle regenerative decline with aging: new approaches to bring back youthfulness to aged stem cells

Autorzy: Pura Muñoz‐Cánoves, Joana Neves, Pedro Sousa‐Victor
Opublikowane w: The FEBS Journal, Numer 287/3, 2020, Strona(/y) 406-416, ISSN 1742-464X
Wydawca: Blackwell Publishing Inc.
DOI: 10.1111/febs.15182

In-vivo Transcriptomic Profiling of Systemic Aging using Cell Encapsulation

Autorzy: Mashinchian, Omid; Hong, Xiaotong; Migliavacca, Eugenia; Boss, Christophe; De Franceschi, Filippo; Michaud, Joris; Le Moal, Emmeran; Collerette-Tremblay, Jasmin; Isern, Joan; Metairon, Sylviane; Raymond, Frederic; Descombes, Patrick; Bouche, Nicolas; Muñoz-Cánoves, Pura; Feige, Jerome N.; Bentzinger, C. Florian
Opublikowane w: Numer 2, 2020
Wydawca: bioRxiv
DOI: 10.1101/2020.03.09.979054

Rezultaty

Publikacje

Udostępnij tę stronę

Pobierz