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

Pubblicazioni

BAR-Seq clonal tracking of gene-edited cells

Autori: Ferrari, Samuele; Beretta, Stefano; Jacob, Aurelien; Cittaro, Davide; Albano, Luisa; Merelli, Ivan; Naldini, Luigi; Genovese, Pietro
Pubblicato in: Nature Protocols, Numero 16, 2021, Pagina/e 2991-3025, ISSN 1754-2189
Editore: Nature Publishing Group
DOI: 10.1038/s41596-021-00529-x

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

Autori: 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
Pubblicato in: Nature Cell Biology, Numero 22, 2020, Pagina/e 1307-1318, ISSN 1465-7392
Editore: Nature Publishing Group
DOI: 10.1038/s41556-020-00593-7

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

Autori: Felix Lansing, Maciej Paszkowski-Rogacz, Lukas Theo Schmitt, Paul Martin Schneider, Teresa Rojo Romanos, Jan Sonntag, Frank Buchholz
Pubblicato in: Nucleic Acids Research, Numero 48/1, 2019, Pagina/e 472-485, ISSN 0305-1048
Editore: Oxford University Press
DOI: 10.1093/nar/gkz1078

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

Autori: Anjali Soni, Martina Augsburg, Frank Buchholz, M. Teresa Pisabarro
Pubblicato in: Scientific Reports, Numero 10/1, 2020, ISSN 2045-2322
Editore: Nature Publishing Group
DOI: 10.1038/s41598-020-70867-5

Delivery of Cas9/sgRNA Ribonucleoprotein Complexes via Hydroxystearyl Oligoamino Amides

Autori: Jasmin Kuhn, Yi Lin, Ana Krhac Levacic, Nader Al Danaf, Lun Peng, Miriam Höhn, Don C. Lamb, Ernst Wagner, Ulrich Lächelt
Pubblicato in: Bioconjugate Chemistry, Numero 31/3, 2020, Pagina/e 729-742, ISSN 1043-1802
Editore: American Chemical Society
DOI: 10.1021/acs.bioconjchem.9b00853

Muscle stem cell aging: identifying ways to induce tissue rejuvenation

Autori: Pedro Sousa-Victor, Joana Neves, Pura Muñoz-Cánoves
Pubblicato in: Mechanisms of Ageing and Development, Numero 188, 2020, Pagina/e 111246, ISSN 0047-6374
Editore: Elsevier BV
DOI: 10.1016/j.mad.2020.111246

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

Autori: Maule, Giulia; Arosio, Daniele; Cereseto, Anna
Pubblicato in: International Journal of Molecular Science, 2020, ISSN 1422-0067
Editore: Multidisciplinary Digital Publishing Institute (MDPI)
DOI: 10.3390/ijms21113903

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

Autori: Uvizl, Alena; Goswami, Ruchi; Gandhi, Shanil Durgeshkumar; Augsburg, Martina; Buchholz, Frank; Guck, Jochen; Mansfeld, Jörg; Girardo, Salvatore
Pubblicato in: Lab on a chip, Numero 12, 2020, ISSN 1473-0197
Editore: Royal Society of Chemistry
DOI: 10.1039/d0lc01224f

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

Autori: 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
Pubblicato in: Nature Biotechnology, Numero 38/11, 2020, Pagina/e 1298-1308, ISSN 1087-0156
Editore: Nature Publishing Group
DOI: 10.1038/s41587-020-0551-y

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

Autori: Franziska Freitag, Ernst Wagner
Pubblicato in: Advanced Drug Delivery Reviews, 2020, ISSN 0169-409X
Editore: Elsevier BV
DOI: 10.1016/j.addr.2020.03.005

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

Autori: 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
Pubblicato in: Journal of Cachexia, Sarcopenia and Muscles, Numero 12, 2021, Pagina/e 1879–1896, ISSN 2190-6009
Editore: Wiley and Sons
DOI: 10.1002/jcsm.12792

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

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

Correction of a Factor VIII genomic inversion with designer-recombinases

Autori: 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
Pubblicato in: Nature Communications, Numero 13, 2022, ISSN 2041-1723
Editore: 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

Autori: 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
Pubblicato in: Science Advances, Numero 7 (39), 2021, ISSN 2375-2548
Editore: American Association for the Advancement of Science
DOI: 10.1126/sciadv.abi7828

Sox17 Controls Emergence and Remodeling of Nestin-Expressing Coronary Vessels

Autori: González-Hernández, Sara; Gómez, Manuel J.; Sánchez-Cabo, Fátima; Méndez-Ferrer, Simón; Muñoz-Cánoves, Pura; Isern, Joan
Pubblicato in: Circulation Research, Numero 127, 2020, Pagina/e e252-e270, ISSN 0009-7330
Editore: 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

Autori: 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
Pubblicato in: Nature Communications, Numero 12, 2021, ISSN 2041-1723
Editore: 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

Autori: 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
Pubblicato in: EMBO Molecular Medicine, Numero 13(3), 2021, ISSN 1757-4676
Editore: John Wiley & Sons Ltd.
DOI: 10.15252/emmm.202013545

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

Autori: Varin, Juliette; Morival, Clément; Maillard, Noémien; Adjali, Oumeya; Cronin, Therese
Pubblicato in: International Journal of Molecular Science, Numero 22, 2021, ISSN 1422-0067
Editore: Multidisciplinary Digital Publishing Institute (MDPI)
DOI: 10.3390/ijms222312818

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

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

Autori: Dell'Orso, Stefania; Juan, Aster H.; Moiseeva, Victoria; Garcia-Prat, Laura; Munoz-Cannoves, Pura; Sartorelli, Vittorio
Pubblicato in: STAR Protocols, Numero 2, 2021, ISSN 2666-1667
Editore: 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

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

CHD4 ensures stem cell lineage fidelity during skeletal muscle regeneration

Autori: 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
Pubblicato in: Stem Cell Reports, Numero 16, 9, 2021, ISSN 2213-6711
Editore: 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

Autori: Duran Sürün, Aksana Schneider, Jovan Mircetic, Katrin Neumann, Felix Lansing, Maciej Paszkowski-Rogacz, Vanessa Hänchen, Min Ae Lee-Kirsch, Frank Buchholz
Pubblicato in: Genes, Numero 11/5, 2020, Pagina/e 511, ISSN 2073-4425
Editore: 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

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

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

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

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

Autori: 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
Pubblicato in: Nature Communications, Numero 11/1, 2020, ISSN 2041-1723
Editore: 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

Autori: Dastidar, Sumitava; Majumdar, Debanjana; Tipanee, Jaitip; Singh, Kshitiz; Klein, Arnaud F.; Furling, Denis; Chuah, Marinee K.; VandenDriessche, Thierry
Pubblicato in: Molecular Therapy, Numero 30 (1), 2022, ISSN 1525-0016
Editore: 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

Autori: 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
Pubblicato in: Cell Reports, Numero 31/7, 2020, Pagina/e 107652, ISSN 2211-1247
Editore: Cell Press
DOI: 10.1016/j.celrep.2020.107652

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

Autori: Ivana Trapani, Alberto Auricchio
Pubblicato in: Human Molecular Genetics, Numero 28/R1, 2019, Pagina/e R108-R118, ISSN 0964-6906
Editore: Oxford University Press
DOI: 10.1093/hmg/ddz130

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

Autori: Hoersten, Jenna; Ruiz-Gómez, Gloria; Lansing, Felix; Rojo-Romanos, Teresa; Schmitt, Lukas Theo; Sonntag, Jan; Pisabarro, M Teresa; Buchholz, Frank
Pubblicato in: Nucleic Acids Research, Numero 50(2), 2022, Pagina/e 1174–1186, ISSN 0305-1048
Editore: Oxford University Press
DOI: 10.1093/nar/gkab1240

A Network of Macrophages Supports Mitochondrial Homeostasis in the Heart

Autori: 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
Pubblicato in: Cell, 2020, ISSN 0092-8674
Editore: Cell Press
DOI: 10.1016/j.cell.2020.08.031

Assessing Autophagy in Muscle Stem Cells

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

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

Autori: Pura Muñoz‐Cánoves, Joana Neves, Pedro Sousa‐Victor
Pubblicato in: The FEBS Journal, Numero 287/3, 2020, Pagina/e 406-416, ISSN 1742-464X
Editore: Blackwell Publishing Inc.
DOI: 10.1111/febs.15182

In-vivo Transcriptomic Profiling of Systemic Aging using Cell Encapsulation

Autori: 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
Pubblicato in: Numero 2, 2020
Editore: bioRxiv
DOI: 10.1101/2020.03.09.979054

Diritti di proprietà intellettuale

GENE THERAPY

Numero candidatura/pubblicazione: 20 19066915
Data: 2019-06-25
Candidato/i: FONDAZIONE TELETHON ETS

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Pubblicazioni

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