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State of the art and perspectives of gene therapy in heart failure: a scientific statement of the Heart Failure Association of the ESC, the ESC Council on Cardiovascular Genomics a... by Linthout, Sophie Van (Author) , Stellos, Konstantinos (Author) , Giacca, Mauro (Author) , Bertero, Edoardo (Author) , Cannata, Antonio (Author) , Carrier, Lucie (Author) , Garcia-Pavia, Pablo (Author) , Ghigo, Alessandra (Author) , González, Arantxa (Author) , Haugaa, Kristina H. (Author) , Imazio, Massimo (Author) , Lopes, Luis R. (Author) , Most, Patrick (Author) , Pollesello, Piero (Author) , Schunkert, Heribert (Author) , Streckfuss-Bömeke, Katrin (Author) , Thum, Thomas (Author) , Tocchetti, Carlo Gabriele (Author) , Tschöpe, Carsten (Author) , van der Meer, Peter (Author) , van Rooij, Eva (Author) , Metra, Marco (Author) , Rosano, Giuseppe M.C. (Author) , Heymans, Stephane (Author) ,

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AAV vector engineering for human aorta transduction: becoming a smooth operator by Rapti, Kleopatra (Author) , Grimm, Dirk (Author) ,

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Treatment of infantile-onset Pompe disease in a rat model with muscle-directed AAV gene therapy by Muñoz, Sergio (Author) , Bertolin, Joan (Author) , Jimenez, Veronica (Author) , Jaén, Maria Luisa (Author) , Garcia, Miquel (Author) , Pujol, Anna (Author) , Vilà, Laia (Author) , Sacristan, Victor (Author) , Barbon, Elena (Author) , Ronzitti, Giuseppe (Author) , El Andari, Jihad (Author) , Tulalamba, Warut (Author) , Pham, Quang Hong (Author) , Ruberte, Jesus (Author) , VandenDriessche, Thierry (Author) , Chuah, Marinee K. (Author) , Grimm, Dirk (Author) , Mingozzi, Federico (Author) , Bosch, Fatima (Author) ,

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Cardiomyocyte-specific RXFP1 overexpression protects against pressure overload-induced cardiac dysfunction independently of relaxin by Wingert, Julia (Author) , Meinhardt, Eric (Author) , Sasipong, Nuttarak (Author) , Pott, Marcel (Author) , Lederer, Christoph (Author) , Torre, Carolina de la (Author) , Sticht, Carsten (Author) , Most, Patrick (Author) , Katus, Hugo (Author) , Frey, Norbert (Author) , Raake, Philip (Author) , Schlegel, Philipp (Author) ,

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2024 update: european consensus statement on gene therapy for spinal muscular atrophy by Kirschner, Janbernd (Author) , Bernert, Günther (Author) , Butoianu, Nina (Author) , De Waele, Liesbeth (Author) , Fattal-Valevski, Aviva (Author) , Haberlova, Jana (Author) , Moreno, Teresa (Author) , Klein, Andrea (Author) , Kostera-Pruszczyk, Anna (Author) , Mercuri, Eugenio (Author) , Quijano-Roy, Susana (Author) , Sejersen, Thomas (Author) , Tizzano, Eduardo F. (Author) , van der Pol, W Ludo (Author) , Wallace, Sean (Author) , Zafeiriou, Dimitrios (Author) , Ziegler, Andreas (Author) , Muntoni, Francesco (Author) , Servais, Laurent (Author) ,

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Efficacy and safety of gene therapy with onasemnogene abeparvovec in children with spinal muscular atrophy in the D-A-CH-region: a population-based observational study by Weiß, Claudia (Author) , Becker, Lena-Luise (Author) , Friese, Johannes (Author) , Blaschek, Astrid (Author) , Hahn, Andreas (Author) , Illsinger, Sabine (Author) , Schwartz, Oliver (Author) , Bernert, Günther (Author) , Hagen, Maja von der (Author) , Husain, Ralf A. (Author) , Goldhahn, Klaus (Author) , Kirschner, Janbernd (Author) , Pechmann, Astrid (Author) , Flotats-Bastardas, Marina (Author) , Schreiber, Gudrun (Author) , Schara, Ulrike (Author) , Plecko, Barbara (Author) , Trollmann, Regina (Author) , Horber, Veronka (Author) , Wilichowski, Ekkehard (Author) , Baumann, Matthias (Author) , Klein, Andrea (Author) , Eisenkölbl, Astrid (Author) , Köhler, Cornelia (Author) , Stettner, Georg M. (Author) , Cirak, Sebahattin (Author) , Hasselmann, Oswald (Author) , Kaindl, Angela M. (Author) , Garbade, Sven (Author) , Johannsen, Jessika (Author) , Ziegler, Andreas (Author) ,

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A framework for N-of-1 trials of individualized gene-targeted therapies for genetic diseases by Kim-McManus, Olivia (Author) , Gleeson, Joseph G. (Author) , Mignon, Laurence (Author) , Smith Fine, Amena (Author) , Yan, Winston (Author) , Nolen, Nicole (Author) , Demarest, Scott (Author) , Berry-Kravis, Elizabeth (Author) , Finkel, Richard (Author) , Leonard, Stefanie (Author) , Finlayson, Samuel (Author) , Augustine, Erika (Author) , Lyon, Gholson J. (Author) , Schüle-Freyer, Rebecca (Author) , Yu, Timothy (Author) ,

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Split intein-mediated protein trans-splicing to express large dystrophins by Tasfaout, Hichem (Author) , Halbert, Christine L. (Author) , McMillen, Timothy S. (Author) , Allen, James M. (Author) , Reyes, Theodore R. (Author) , Flint, Galina V. (Author) , Grimm, Dirk (Author) , Hauschka, Stephen D. (Author) , Regnier, Michael (Author) , Chamberlain, Jeffrey S. (Author) ,

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Devising a framework of optogenetic coding in the auditory pathway: insights from auditory midbrain recordings by Michael, Maria (Author) , Wolf, Bettina Julia (Author) , Klinge-Strahl, Astrid (Author) , Jeschke, Marcus (Author) , Moser, Tobias (Author) , Dieter, Alexander (Author) ,

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Development of an AAV9-RNAi-mediated silencing strategy to abrogate TRPM4 expression in the adult heart by Medert, Rebekka (Author) , Jungmann, Andreas (Author) , Hildebrand, Staffan (Author) , Busch, Martin (Author) , Grimm, Dirk (Author) , Flockerzi, Veit (Author) , Müller, Oliver J. (Author) , Most, Patrick (Author) , Schumacher, Dagmar (Author) , Freichel, Marc (Author) ,

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Arzneimittel für neuartige Therapien - Perspektiven, Chancen, Herausforderungen by Rascher, Wolfgang (Author) , Klingebiel, Thomas (Author) , Herting, Egbert (Author) , Hoffmann, Georg F. (Author) , Berner, Reinhard (Author) , Krägeloh-Mann, Ingeborg (Author) , Gärtner, Jutta (Author) , Zepp, Fred (Author) , Schara, Ulrike (Author) ,

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Arzneimittel für neuartige Therapien: Kurzfassung der Stellungnahme der Deutschen Gesellschaft für Kinder- und Jugendmedizin e.V. (DGKJ) = Advanced therapy medicinal products - s... by Rascher, Wolfgang (Author) , Klingebiel, Thomas (Author) , Herting, Egbert (Author) , Hoffmann, Georg F. (Author) , Berner, Reinhard (Author) , Krägeloh-Mann, Ingeborg (Author) , Gärtner, Jutta (Author) , Zepp, Fred (Author) , Schara, Ulrike (Author) ,

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Rechtliche Aspekte der Genom-Editierung an der menschlichen Keimbahn: a comparative legal study

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European ad-hoc consensus statement on gene replacement therapy for spinal muscular atrophy by Kirschner, Janbernd (Author) , Butoianu, Nina (Author) , Goemans, Nathalie (Author) , Haberlova, Jana (Author) , Kostera-Pruszczyk, Anna (Author) , Mercuri, Eugenio (Author) , van der Pol, W. Ludo (Author) , Quijano-Roy, Susana (Author) , Sejersen, Thomas (Author) , Tizzano, Eduardo F. (Author) , Ziegler, Andreas (Author) , Servais, Laurent (Author) , Muntoni, Francesco (Author) ,

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Gene transfer to the vascular system: novel translational perspectives for vascular diseases by Remes, Anca (Author) , Basha, D. I. (Author) , Frey, N. (Author) , Wagner, Andreas H. (Author) , Müller, Oliver J. (Author) ,

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Various effects of AAV9-mediated βARKct gene therapy on the heart in dystrophin-deficient (mdx) mice and δ-sarcoglycan-deficient (Sgcd-/-) mice by Bauer, Ralf (Author) , Enns, Helene (Author) , Jungmann, Andreas (Author) , Volz, Hans Christian (Author) , Schinkel, Stefanie (Author) , Raake, Philip (Author) , Most, Patrick (Author) , Katus, Hugo (Author) , Müller, Oliver J. (Author) ,

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Gentherapien für neuromuskuläre Erkrankungen by Saffari, Afshin (Author) , Weiler, Markus (Author) , Hoffmann, Georg F. (Author) , Ziegler, Andreas (Author) ,

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Exosome-mediated therapeutic delivery: a new horizon for human neurodegenerative disorders’ treatment (with a focus on siRNA delivery improvement) by Seyfizadeh, Nayer (Author) , Seyfizadeh, Narges (Author) ,

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Next-generation AAV vectors for clinical use: an ever-accelerating race by Weinmann, Jonas (Author) , Grimm, Dirk (Author) ,

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Viral gene transfer of APPsα rescues synaptic failure in an Alzheimer’s disease mouse model by Fol, Romain (Author) , Braudeau, Jerome (Author) , Ludewig, Susann (Author) , Abel, Tobias (Author) , Weyer, Sascha W. (Author) , Röderer, Jan-Peter (Author) , Brod, Florian (Author) , Audrain, Mickael (Author) , Bemelmans, Alexis-Pierre (Author) , Buchholz, Christian J. (Author) , Korte, Martin (Author) , Cartier, Nathalie (Author) , Müller, Ulrike C. (Author) ,

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