Unlocking Precision Gene Therapy

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Publications

BAR-Seq clonal tracking of gene-edited cells

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

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

Auteurs: 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
Publié dans: Nature Cell Biology, Numéro 22, 2020, Page(s) 1307-1318, ISSN 1465-7392
Éditeur: Nature Publishing Group
DOI: 10.1038/s41556-020-00593-7

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

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

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

Auteurs: Anjali Soni, Martina Augsburg, Frank Buchholz, M. Teresa Pisabarro
Publié dans: Scientific Reports, Numéro 10/1, 2020, ISSN 2045-2322
Éditeur: Nature Publishing Group
DOI: 10.1038/s41598-020-70867-5

Delivery of Cas9/sgRNA Ribonucleoprotein Complexes via Hydroxystearyl Oligoamino Amides

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

Muscle stem cell aging: identifying ways to induce tissue rejuvenation

Auteurs: Pedro Sousa-Victor, Joana Neves, Pura Muñoz-Cánoves
Publié dans: Mechanisms of Ageing and Development, Numéro 188, 2020, Page(s) 111246, ISSN 0047-6374
Éditeur: Elsevier BV
DOI: 10.1016/j.mad.2020.111246

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

Auteurs: Maule, Giulia; Arosio, Daniele; Cereseto, Anna
Publié dans: International Journal of Molecular Science, 2020, ISSN 1422-0067
Éditeur: Multidisciplinary Digital Publishing Institute (MDPI)
DOI: 10.3390/ijms21113903

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

Auteurs: Uvizl, Alena; Goswami, Ruchi; Gandhi, Shanil Durgeshkumar; Augsburg, Martina; Buchholz, Frank; Guck, Jochen; Mansfeld, Jörg; Girardo, Salvatore
Publié dans: Lab on a chip, Numéro 12, 2020, ISSN 1473-0197
Éditeur: Royal Society of Chemistry
DOI: 10.1039/d0lc01224f

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

Auteurs: 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
Publié dans: Nature Biotechnology, Numéro 38/11, 2020, Page(s) 1298-1308, ISSN 1087-0156
Éditeur: Nature Publishing Group
DOI: 10.1038/s41587-020-0551-y

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

Auteurs: Franziska Freitag, Ernst Wagner
Publié dans: Advanced Drug Delivery Reviews, 2020, ISSN 0169-409X
Éditeur: Elsevier BV
DOI: 10.1016/j.addr.2020.03.005

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

Auteurs: 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
Publié dans: Journal of Cachexia, Sarcopenia and Muscles, Numéro 12, 2021, Page(s) 1879–1896, ISSN 2190-6009
Éditeur: Wiley and Sons
DOI: 10.1002/jcsm.12792

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

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

Correction of a Factor VIII genomic inversion with designer-recombinases

Auteurs: 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
Publié dans: Nature Communications, Numéro 13, 2022, ISSN 2041-1723
Éditeur: 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

Auteurs: 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
Publié dans: Science Advances, Numéro 7 (39), 2021, ISSN 2375-2548
Éditeur: American Association for the Advancement of Science
DOI: 10.1126/sciadv.abi7828

Sox17 Controls Emergence and Remodeling of Nestin-Expressing Coronary Vessels

Auteurs: González-Hernández, Sara; Gómez, Manuel J.; Sánchez-Cabo, Fátima; Méndez-Ferrer, Simón; Muñoz-Cánoves, Pura; Isern, Joan
Publié dans: Circulation Research, Numéro 127, 2020, Page(s) e252-e270, ISSN 0009-7330
Éditeur: 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

Auteurs: 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
Publié dans: Nature Communications, Numéro 12, 2021, ISSN 2041-1723
Éditeur: 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

Auteurs: 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
Publié dans: EMBO Molecular Medicine, Numéro 13(3), 2021, ISSN 1757-4676
Éditeur: John Wiley & Sons Ltd.
DOI: 10.15252/emmm.202013545

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

Auteurs: Varin, Juliette; Morival, Clément; Maillard, Noémien; Adjali, Oumeya; Cronin, Therese
Publié dans: International Journal of Molecular Science, Numéro 22, 2021, ISSN 1422-0067
Éditeur: Multidisciplinary Digital Publishing Institute (MDPI)
DOI: 10.3390/ijms222312818

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

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

Auteurs: Dell'Orso, Stefania; Juan, Aster H.; Moiseeva, Victoria; Garcia-Prat, Laura; Munoz-Cannoves, Pura; Sartorelli, Vittorio
Publié dans: STAR Protocols, Numéro 2, 2021, ISSN 2666-1667
Éditeur: 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

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

CHD4 ensures stem cell lineage fidelity during skeletal muscle regeneration

Auteurs: 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
Publié dans: Stem Cell Reports, Numéro 16, 9, 2021, ISSN 2213-6711
Éditeur: 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

Auteurs: Duran Sürün, Aksana Schneider, Jovan Mircetic, Katrin Neumann, Felix Lansing, Maciej Paszkowski-Rogacz, Vanessa Hänchen, Min Ae Lee-Kirsch, Frank Buchholz
Publié dans: Genes, Numéro 11/5, 2020, Page(s) 511, ISSN 2073-4425
Éditeur: 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

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

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

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

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

Auteurs: 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
Publié dans: Nature Communications, Numéro 11/1, 2020, ISSN 2041-1723
Éditeur: 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

Auteurs: Dastidar, Sumitava; Majumdar, Debanjana; Tipanee, Jaitip; Singh, Kshitiz; Klein, Arnaud F.; Furling, Denis; Chuah, Marinee K.; VandenDriessche, Thierry
Publié dans: Molecular Therapy, Numéro 30 (1), 2022, ISSN 1525-0016
Éditeur: 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

Auteurs: 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
Publié dans: Cell Reports, Numéro 31/7, 2020, Page(s) 107652, ISSN 2211-1247
Éditeur: Cell Press
DOI: 10.1016/j.celrep.2020.107652

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

Auteurs: Ivana Trapani, Alberto Auricchio
Publié dans: Human Molecular Genetics, Numéro 28/R1, 2019, Page(s) R108-R118, ISSN 0964-6906
Éditeur: Oxford University Press
DOI: 10.1093/hmg/ddz130

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

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

A Network of Macrophages Supports Mitochondrial Homeostasis in the Heart

Auteurs: 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
Publié dans: Cell, 2020, ISSN 0092-8674
Éditeur: Cell Press
DOI: 10.1016/j.cell.2020.08.031

Assessing Autophagy in Muscle Stem Cells

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

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

Auteurs: Pura Muñoz‐Cánoves, Joana Neves, Pedro Sousa‐Victor
Publié dans: The FEBS Journal, Numéro 287/3, 2020, Page(s) 406-416, ISSN 1742-464X
Éditeur: Blackwell Publishing Inc.
DOI: 10.1111/febs.15182

In-vivo Transcriptomic Profiling of Systemic Aging using Cell Encapsulation

Auteurs: 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
Publié dans: Numéro 2, 2020
Éditeur: bioRxiv
DOI: 10.1101/2020.03.09.979054

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GENE THERAPY

Numéro de demande/publication: 20 19066915
Date: 2019-06-25
Demandeur(s): FONDAZIONE TELETHON ETS

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