Unlocking Precision Gene Therapy

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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

Publicaciones

BAR-Seq clonal tracking of gene-edited cells

Autores: Ferrari, Samuele; Beretta, Stefano; Jacob, Aurelien; Cittaro, Davide; Albano, Luisa; Merelli, Ivan; Naldini, Luigi; Genovese, Pietro
Publicado en: Nature Protocols, Edición 16, 2021, Página(s) 2991-3025, ISSN 1754-2189
Editor: Nature Publishing Group
DOI: 10.1038/s41596-021-00529-x

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

Autores: 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
Publicado en: Nature Cell Biology, Edición 22, 2020, Página(s) 1307-1318, ISSN 1465-7392
Editor: Nature Publishing Group
DOI: 10.1038/s41556-020-00593-7

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

Autores: Felix Lansing, Maciej Paszkowski-Rogacz, Lukas Theo Schmitt, Paul Martin Schneider, Teresa Rojo Romanos, Jan Sonntag, Frank Buchholz
Publicado en: Nucleic Acids Research, Edición 48/1, 2019, Página(s) 472-485, ISSN 0305-1048
Editor: Oxford University Press
DOI: 10.1093/nar/gkz1078

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

Autores: Anjali Soni, Martina Augsburg, Frank Buchholz, M. Teresa Pisabarro
Publicado en: Scientific Reports, Edición 10/1, 2020, ISSN 2045-2322
Editor: Nature Publishing Group
DOI: 10.1038/s41598-020-70867-5

Delivery of Cas9/sgRNA Ribonucleoprotein Complexes via Hydroxystearyl Oligoamino Amides

Autores: Jasmin Kuhn, Yi Lin, Ana Krhac Levacic, Nader Al Danaf, Lun Peng, Miriam Höhn, Don C. Lamb, Ernst Wagner, Ulrich Lächelt
Publicado en: Bioconjugate Chemistry, Edición 31/3, 2020, Página(s) 729-742, ISSN 1043-1802
Editor: American Chemical Society
DOI: 10.1021/acs.bioconjchem.9b00853

Muscle stem cell aging: identifying ways to induce tissue rejuvenation

Autores: Pedro Sousa-Victor, Joana Neves, Pura Muñoz-Cánoves
Publicado en: Mechanisms of Ageing and Development, Edición 188, 2020, Página(s) 111246, ISSN 0047-6374
Editor: Elsevier BV
DOI: 10.1016/j.mad.2020.111246

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

Autores: Maule, Giulia; Arosio, Daniele; Cereseto, Anna
Publicado en: International Journal of Molecular Science, 2020, ISSN 1422-0067
Editor: Multidisciplinary Digital Publishing Institute (MDPI)
DOI: 10.3390/ijms21113903

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

Autores: Uvizl, Alena; Goswami, Ruchi; Gandhi, Shanil Durgeshkumar; Augsburg, Martina; Buchholz, Frank; Guck, Jochen; Mansfeld, Jörg; Girardo, Salvatore
Publicado en: Lab on a chip, Edición 12, 2020, ISSN 1473-0197
Editor: Royal Society of Chemistry
DOI: 10.1039/d0lc01224f

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

Autores: 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
Publicado en: Nature Biotechnology, Edición 38/11, 2020, Página(s) 1298-1308, ISSN 1087-0156
Editor: Nature Publishing Group
DOI: 10.1038/s41587-020-0551-y

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

Autores: Franziska Freitag, Ernst Wagner
Publicado en: Advanced Drug Delivery Reviews, 2020, ISSN 0169-409X
Editor: Elsevier BV
DOI: 10.1016/j.addr.2020.03.005

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

Autores: 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
Publicado en: Journal of Cachexia, Sarcopenia and Muscles, Edición 12, 2021, Página(s) 1879–1896, ISSN 2190-6009
Editor: Wiley and Sons
DOI: 10.1002/jcsm.12792

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

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

Correction of a Factor VIII genomic inversion with designer-recombinases

Autores: 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
Publicado en: Nature Communications, Edición 13, 2022, ISSN 2041-1723
Editor: 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

Autores: 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
Publicado en: Science Advances, Edición 7 (39), 2021, ISSN 2375-2548
Editor: American Association for the Advancement of Science
DOI: 10.1126/sciadv.abi7828

Sox17 Controls Emergence and Remodeling of Nestin-Expressing Coronary Vessels

Autores: González-Hernández, Sara; Gómez, Manuel J.; Sánchez-Cabo, Fátima; Méndez-Ferrer, Simón; Muñoz-Cánoves, Pura; Isern, Joan
Publicado en: Circulation Research, Edición 127, 2020, Página(s) e252-e270, ISSN 0009-7330
Editor: 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

Autores: 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
Publicado en: Nature Communications, Edición 12, 2021, ISSN 2041-1723
Editor: 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

Autores: 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
Publicado en: EMBO Molecular Medicine, Edición 13(3), 2021, ISSN 1757-4676
Editor: John Wiley & Sons Ltd.
DOI: 10.15252/emmm.202013545

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

Autores: Varin, Juliette; Morival, Clément; Maillard, Noémien; Adjali, Oumeya; Cronin, Therese
Publicado en: International Journal of Molecular Science, Edición 22, 2021, ISSN 1422-0067
Editor: Multidisciplinary Digital Publishing Institute (MDPI)
DOI: 10.3390/ijms222312818

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

Autores: Ferrari, Samuele; Vavassori, Valentina; Canarutto, Daniele; Jacob, Aurelien; Castiello, Maria Carmina; Javed, Attya Omer; Genovese, Pietro
Publicado en: Frontiers in Genome Editing, 2021, ISSN 2673-3439
Editor: 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

Autores: Dell'Orso, Stefania; Juan, Aster H.; Moiseeva, Victoria; Garcia-Prat, Laura; Munoz-Cannoves, Pura; Sartorelli, Vittorio
Publicado en: STAR Protocols, Edición 2, 2021, ISSN 2666-1667
Editor: 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

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

CHD4 ensures stem cell lineage fidelity during skeletal muscle regeneration

Autores: 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
Publicado en: Stem Cell Reports, Edición 16, 9, 2021, ISSN 2213-6711
Editor: 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

Autores: Duran Sürün, Aksana Schneider, Jovan Mircetic, Katrin Neumann, Felix Lansing, Maciej Paszkowski-Rogacz, Vanessa Hänchen, Min Ae Lee-Kirsch, Frank Buchholz
Publicado en: Genes, Edición 11/5, 2020, Página(s) 511, ISSN 2073-4425
Editor: 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

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

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

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

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

Autores: 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
Publicado en: Nature Communications, Edición 11/1, 2020, ISSN 2041-1723
Editor: 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

Autores: Dastidar, Sumitava; Majumdar, Debanjana; Tipanee, Jaitip; Singh, Kshitiz; Klein, Arnaud F.; Furling, Denis; Chuah, Marinee K.; VandenDriessche, Thierry
Publicado en: Molecular Therapy, Edición 30 (1), 2022, ISSN 1525-0016
Editor: 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

Autores: 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
Publicado en: Cell Reports, Edición 31/7, 2020, Página(s) 107652, ISSN 2211-1247
Editor: Cell Press
DOI: 10.1016/j.celrep.2020.107652

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

Autores: Ivana Trapani, Alberto Auricchio
Publicado en: Human Molecular Genetics, Edición 28/R1, 2019, Página(s) R108-R118, ISSN 0964-6906
Editor: Oxford University Press
DOI: 10.1093/hmg/ddz130

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

Autores: Hoersten, Jenna; Ruiz-Gómez, Gloria; Lansing, Felix; Rojo-Romanos, Teresa; Schmitt, Lukas Theo; Sonntag, Jan; Pisabarro, M Teresa; Buchholz, Frank
Publicado en: Nucleic Acids Research, Edición 50(2), 2022, Página(s) 1174–1186, ISSN 0305-1048
Editor: Oxford University Press
DOI: 10.1093/nar/gkab1240

A Network of Macrophages Supports Mitochondrial Homeostasis in the Heart

Autores: 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
Publicado en: Cell, 2020, ISSN 0092-8674
Editor: Cell Press
DOI: 10.1016/j.cell.2020.08.031

Assessing Autophagy in Muscle Stem Cells

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

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

Autores: Pura Muñoz‐Cánoves, Joana Neves, Pedro Sousa‐Victor
Publicado en: The FEBS Journal, Edición 287/3, 2020, Página(s) 406-416, ISSN 1742-464X
Editor: Blackwell Publishing Inc.
DOI: 10.1111/febs.15182

In-vivo Transcriptomic Profiling of Systemic Aging using Cell Encapsulation

Autores: 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
Publicado en: Edición 2, 2020
Editor: bioRxiv
DOI: 10.1101/2020.03.09.979054

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