Unlocking Precision Gene Therapy

Deliverables

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

Publications

BAR-Seq clonal tracking of gene-edited cells

Author(s): Ferrari, Samuele; Beretta, Stefano; Jacob, Aurelien; Cittaro, Davide; Albano, Luisa; Merelli, Ivan; Naldini, Luigi; Genovese, Pietro
Published in: Nature Protocols, Issue 16, 2021, Page(s) 2991-3025, ISSN 1754-2189
Publisher: Nature Publishing Group
DOI: 10.1038/s41596-021-00529-x

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

Author(s): 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
Published in: Nature Cell Biology, Issue 22, 2020, Page(s) 1307-1318, ISSN 1465-7392
Publisher: Nature Publishing Group
DOI: 10.1038/s41556-020-00593-7

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

Author(s): Felix Lansing, Maciej Paszkowski-Rogacz, Lukas Theo Schmitt, Paul Martin Schneider, Teresa Rojo Romanos, Jan Sonntag, Frank Buchholz
Published in: Nucleic Acids Research, Issue 48/1, 2019, Page(s) 472-485, ISSN 0305-1048
Publisher: Oxford University Press
DOI: 10.1093/nar/gkz1078

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

Author(s): Anjali Soni, Martina Augsburg, Frank Buchholz, M. Teresa Pisabarro
Published in: Scientific Reports, Issue 10/1, 2020, ISSN 2045-2322
Publisher: Nature Publishing Group
DOI: 10.1038/s41598-020-70867-5

Delivery of Cas9/sgRNA Ribonucleoprotein Complexes via Hydroxystearyl Oligoamino Amides

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

Muscle stem cell aging: identifying ways to induce tissue rejuvenation

Author(s): Pedro Sousa-Victor, Joana Neves, Pura Muñoz-Cánoves
Published in: Mechanisms of Ageing and Development, Issue 188, 2020, Page(s) 111246, ISSN 0047-6374
Publisher: Elsevier BV
DOI: 10.1016/j.mad.2020.111246

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

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

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

Author(s): Uvizl, Alena; Goswami, Ruchi; Gandhi, Shanil Durgeshkumar; Augsburg, Martina; Buchholz, Frank; Guck, Jochen; Mansfeld, Jörg; Girardo, Salvatore
Published in: Lab on a chip, Issue 12, 2020, ISSN 1473-0197
Publisher: Royal Society of Chemistry
DOI: 10.1039/d0lc01224f

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

Author(s): 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
Published in: Nature Biotechnology, Issue 38/11, 2020, Page(s) 1298-1308, ISSN 1087-0156
Publisher: Nature Publishing Group
DOI: 10.1038/s41587-020-0551-y

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

Author(s): Franziska Freitag, Ernst Wagner
Published in: Advanced Drug Delivery Reviews, 2020, ISSN 0169-409X
Publisher: Elsevier BV
DOI: 10.1016/j.addr.2020.03.005

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

Author(s): 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
Published in: Journal of Cachexia, Sarcopenia and Muscles, Issue 12, 2021, Page(s) 1879–1896, ISSN 2190-6009
Publisher: Wiley and Sons
DOI: 10.1002/jcsm.12792

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

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

Correction of a Factor VIII genomic inversion with designer-recombinases

Author(s): 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
Published in: Nature Communications, Issue 13, 2022, ISSN 2041-1723
Publisher: 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

Author(s): 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
Published in: Science Advances, Issue 7 (39), 2021, ISSN 2375-2548
Publisher: American Association for the Advancement of Science
DOI: 10.1126/sciadv.abi7828

Sox17 Controls Emergence and Remodeling of Nestin-Expressing Coronary Vessels

Author(s): González-Hernández, Sara; Gómez, Manuel J.; Sánchez-Cabo, Fátima; Méndez-Ferrer, Simón; Muñoz-Cánoves, Pura; Isern, Joan
Published in: Circulation Research, Issue 127, 2020, Page(s) e252-e270, ISSN 0009-7330
Publisher: 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

Author(s): 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
Published in: Nature Communications, Issue 12, 2021, ISSN 2041-1723
Publisher: 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

Author(s): 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
Published in: EMBO Molecular Medicine, Issue 13(3), 2021, ISSN 1757-4676
Publisher: John Wiley & Sons Ltd.
DOI: 10.15252/emmm.202013545

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

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

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

Author(s): Ferrari, Samuele; Vavassori, Valentina; Canarutto, Daniele; Jacob, Aurelien; Castiello, Maria Carmina; Javed, Attya Omer; Genovese, Pietro
Published in: Frontiers in Genome Editing, 2021, ISSN 2673-3439
Publisher: 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

Author(s): Dell'Orso, Stefania; Juan, Aster H.; Moiseeva, Victoria; Garcia-Prat, Laura; Munoz-Cannoves, Pura; Sartorelli, Vittorio
Published in: STAR Protocols, Issue 2, 2021, ISSN 2666-1667
Publisher: 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

Author(s): Potapov, Ilya; García-Prat, Laura; Ravichandran, Srikanth; Muñoz-Cánoves, Pura; del Sol, Antonio
Published in: The FEBS Journal, 2021, ISSN 1742-4658
Publisher: John Wiley & Sons
DOI: 10.1111/febs.15832

CHD4 ensures stem cell lineage fidelity during skeletal muscle regeneration

Author(s): 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
Published in: Stem Cell Reports, Issue 16, 9, 2021, ISSN 2213-6711
Publisher: 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

Author(s): Duran Sürün, Aksana Schneider, Jovan Mircetic, Katrin Neumann, Felix Lansing, Maciej Paszkowski-Rogacz, Vanessa Hänchen, Min Ae Lee-Kirsch, Frank Buchholz
Published in: Genes, Issue 11/5, 2020, Page(s) 511, ISSN 2073-4425
Publisher: 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

Author(s): Céline Vandamme, Rebecca Xicluna, Leslie Hesnard, Marie Devaux, Nicolas Jaulin, Mickaël Guilbaud, Johanne Le Duff, Célia Couzinié, Philippe Moullier, Xavier Saulquin, Oumeya Adjali
Published in: Frontiers in Immunology, Issue 10, 2020, ISSN 1664-3224
Publisher: Frontiers
DOI: 10.3389/fimmu.2019.03110

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

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

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

Author(s): 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
Published in: Nature Communications, Issue 11/1, 2020, ISSN 2041-1723
Publisher: 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

Author(s): Dastidar, Sumitava; Majumdar, Debanjana; Tipanee, Jaitip; Singh, Kshitiz; Klein, Arnaud F.; Furling, Denis; Chuah, Marinee K.; VandenDriessche, Thierry
Published in: Molecular Therapy, Issue 30 (1), 2022, ISSN 1525-0016
Publisher: 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

Author(s): 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
Published in: Cell Reports, Issue 31/7, 2020, Page(s) 107652, ISSN 2211-1247
Publisher: Cell Press
DOI: 10.1016/j.celrep.2020.107652

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

Author(s): Ivana Trapani, Alberto Auricchio
Published in: Human Molecular Genetics, Issue 28/R1, 2019, Page(s) R108-R118, ISSN 0964-6906
Publisher: Oxford University Press
DOI: 10.1093/hmg/ddz130

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

Author(s): Hoersten, Jenna; Ruiz-Gómez, Gloria; Lansing, Felix; Rojo-Romanos, Teresa; Schmitt, Lukas Theo; Sonntag, Jan; Pisabarro, M Teresa; Buchholz, Frank
Published in: Nucleic Acids Research, Issue 50(2), 2022, Page(s) 1174–1186, ISSN 0305-1048
Publisher: Oxford University Press
DOI: 10.1093/nar/gkab1240

A Network of Macrophages Supports Mitochondrial Homeostasis in the Heart

Author(s): 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
Published in: Cell, 2020, ISSN 0092-8674
Publisher: Cell Press
DOI: 10.1016/j.cell.2020.08.031

Assessing Autophagy in Muscle Stem Cells

Author(s): Campanario, Silvia; Ramírez-Pardo, Ignacio; Hong, Xiaotong; Isern, Joan; Muñoz-Cánoves, Pura
Published in: Frontiers in Cell and Developmental Biology, 2021, ISSN 2296-634X
Publisher: Frontiers
DOI: 10.3389/fcell.2020.620409

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

Author(s): Pura Muñoz‐Cánoves, Joana Neves, Pedro Sousa‐Victor
Published in: The FEBS Journal, Issue 287/3, 2020, Page(s) 406-416, ISSN 1742-464X
Publisher: Blackwell Publishing Inc.
DOI: 10.1111/febs.15182

In-vivo Transcriptomic Profiling of Systemic Aging using Cell Encapsulation

Author(s): 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
Published in: Issue 2, 2020
Publisher: bioRxiv
DOI: 10.1101/2020.03.09.979054

Intellectual Property Rights

GENE THERAPY

Application/Publication number: 20 19066915
Date: 2019-06-25
Applicant(s): FONDAZIONE TELETHON ETS

Deliverables

Publications

Intellectual Property Rights

Share this page

Download