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Therapeutic AASS inhibition by AAV-miRNA rescues glutaric aciduria type I severe phenotype in mice.

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Title: Therapeutic AASS inhibition by AAV-miRNA rescues glutaric aciduria type I severe phenotype in mice.
Authors: Segur-Bailach E; Institut d'Investigacions Biomèdiques August Pi i Sunyer (IDIBAPS), 08036 Barcelona, Spain; Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER), 08036 Barcelona, Spain., Mateu-Bosch A; Institut d'Investigacions Biomèdiques August Pi i Sunyer (IDIBAPS), 08036 Barcelona, Spain., Bofill-De Ros X; Department of Molecular Biology and Genetics, Aarhus University, Aarhus, Denmark., Parés M; Institut d'Investigacions Biomèdiques August Pi i Sunyer (IDIBAPS), 08036 Barcelona, Spain., da Silva Buttkus P; Institute of Experimental Genetics, German Mouse Clinic, Helmholtz Zentrum München, 85764 Neuherberg, Germany., Rathkolb B; Institute of Experimental Genetics, German Mouse Clinic, Helmholtz Zentrum München, 85764 Neuherberg, Germany; German Center for Diabetes Research (DZD), 85764 Neuherberg, Germany; Institute of Molecular Animal Breeding and Biotechnology, Genecenter, Ludwig-Maximilians-Universität München, 81377 Munich, Germany., Gailus-Durner V; Institute of Experimental Genetics, German Mouse Clinic, Helmholtz Zentrum München, 85764 Neuherberg, Germany., Hrabě de Angelis M; Institute of Experimental Genetics, German Mouse Clinic, Helmholtz Zentrum München, 85764 Neuherberg, Germany; Experimental Genetics, TUM School of Life Sciences, Technische Universität München, 85354 Freising, Germany; German Center for Diabetes Research (DZD), 85764 Neuherberg, Germany., Moeini P; DNA & RNA Medicine Division, Gene Therapy for Rare Diseases Department, Center for Applied Medical Research (CIMA), University of Navarra, IdisNA, Pamplona, Spain., Gonzalez-Aseguinolaza G; DNA & RNA Medicine Division, Gene Therapy for Rare Diseases Department, Center for Applied Medical Research (CIMA), University of Navarra, IdisNA, Pamplona, Spain., Tort F; Institut d'Investigacions Biomèdiques August Pi i Sunyer (IDIBAPS), 08036 Barcelona, Spain; Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER), 08036 Barcelona, Spain; Secció d'Errors Congènits del Metabolisme-IBC, Servei de Bioquímica i Genètica Molecular, Hospital Clínic de Barcelona, 08028 Barcelona, Spain., Ribes A; Institut d'Investigacions Biomèdiques August Pi i Sunyer (IDIBAPS), 08036 Barcelona, Spain; Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER), 08036 Barcelona, Spain; Secció d'Errors Congènits del Metabolisme-IBC, Servei de Bioquímica i Genètica Molecular, Hospital Clínic de Barcelona, 08028 Barcelona, Spain., van Karnebeek CDM; Departments of Pediatrics and Human Genetics, Emma Center for Personalized Medicine, Amsterdam Gastro-Enterology Endocrinology Metabolism, Amsterdam University Medical Centers, Amsterdam, the Netherlands., García-Villoria J; Institut d'Investigacions Biomèdiques August Pi i Sunyer (IDIBAPS), 08036 Barcelona, Spain; Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER), 08036 Barcelona, Spain; Secció d'Errors Congènits del Metabolisme-IBC, Servei de Bioquímica i Genètica Molecular, Hospital Clínic de Barcelona, 08028 Barcelona, Spain., Fillat C; Institut d'Investigacions Biomèdiques August Pi i Sunyer (IDIBAPS), 08036 Barcelona, Spain; Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER), 08036 Barcelona, Spain; Facultat de Medicina i Ciències de la Salut, Universitat de Barcelona, 08036 Barcelona, Spain. Electronic address: cfillat@recerca.clinic.cat.
Source: Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2025 Oct 01; Vol. 33 (10), pp. 4820-4833. Date of Electronic Publication: 2025 Jul 17.
Publication Type: Journal Article
Journal Info: Publisher: Cell Press Country of Publication: United States NLM ID: 100890581 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1525-0024 (Electronic) Linking ISSN: 15250016 NLM ISO Abbreviation: Mol Ther Subsets: MEDLINE
Database: MEDLINE Ultimate
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  Data: Therapeutic AASS inhibition by AAV-miRNA rescues glutaric aciduria type I severe phenotype in mice.
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  Data: <searchLink fieldCode="AU" term="%22Segur-Bailach+E%22">Segur-Bailach E</searchLink>; Institut d'Investigacions Biomèdiques August Pi i Sunyer (IDIBAPS), 08036 Barcelona, Spain; Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER), 08036 Barcelona, Spain.<br /><searchLink fieldCode="AU" term="%22Mateu-Bosch+A%22">Mateu-Bosch A</searchLink>; Institut d'Investigacions Biomèdiques August Pi i Sunyer (IDIBAPS), 08036 Barcelona, Spain.<br /><searchLink fieldCode="AU" term="%22Bofill-De+Ros+X%22">Bofill-De Ros X</searchLink>; Department of Molecular Biology and Genetics, Aarhus University, Aarhus, Denmark.<br /><searchLink fieldCode="AU" term="%22Parés+M%22">Parés M</searchLink>; Institut d'Investigacions Biomèdiques August Pi i Sunyer (IDIBAPS), 08036 Barcelona, Spain.<br /><searchLink fieldCode="AU" term="%22da+Silva+Buttkus+P%22">da Silva Buttkus P</searchLink>; Institute of Experimental Genetics, German Mouse Clinic, Helmholtz Zentrum München, 85764 Neuherberg, Germany.<br /><searchLink fieldCode="AU" term="%22Rathkolb+B%22">Rathkolb B</searchLink>; Institute of Experimental Genetics, German Mouse Clinic, Helmholtz Zentrum München, 85764 Neuherberg, Germany; German Center for Diabetes Research (DZD), 85764 Neuherberg, Germany; Institute of Molecular Animal Breeding and Biotechnology, Genecenter, Ludwig-Maximilians-Universität München, 81377 Munich, Germany.<br /><searchLink fieldCode="AU" term="%22Gailus-Durner+V%22">Gailus-Durner V</searchLink>; Institute of Experimental Genetics, German Mouse Clinic, Helmholtz Zentrum München, 85764 Neuherberg, Germany.<br /><searchLink fieldCode="AU" term="%22Hrabě+de+Angelis+M%22">Hrabě de Angelis M</searchLink>; Institute of Experimental Genetics, German Mouse Clinic, Helmholtz Zentrum München, 85764 Neuherberg, Germany; Experimental Genetics, TUM School of Life Sciences, Technische Universität München, 85354 Freising, Germany; German Center for Diabetes Research (DZD), 85764 Neuherberg, Germany.<br /><searchLink fieldCode="AU" term="%22Moeini+P%22">Moeini P</searchLink>; DNA & RNA Medicine Division, Gene Therapy for Rare Diseases Department, Center for Applied Medical Research (CIMA), University of Navarra, IdisNA, Pamplona, Spain.<br /><searchLink fieldCode="AU" term="%22Gonzalez-Aseguinolaza+G%22">Gonzalez-Aseguinolaza G</searchLink>; DNA & RNA Medicine Division, Gene Therapy for Rare Diseases Department, Center for Applied Medical Research (CIMA), University of Navarra, IdisNA, Pamplona, Spain.<br /><searchLink fieldCode="AU" term="%22Tort+F%22">Tort F</searchLink>; Institut d'Investigacions Biomèdiques August Pi i Sunyer (IDIBAPS), 08036 Barcelona, Spain; Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER), 08036 Barcelona, Spain; Secció d'Errors Congènits del Metabolisme-IBC, Servei de Bioquímica i Genètica Molecular, Hospital Clínic de Barcelona, 08028 Barcelona, Spain.<br /><searchLink fieldCode="AU" term="%22Ribes+A%22">Ribes A</searchLink>; Institut d'Investigacions Biomèdiques August Pi i Sunyer (IDIBAPS), 08036 Barcelona, Spain; Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER), 08036 Barcelona, Spain; Secció d'Errors Congènits del Metabolisme-IBC, Servei de Bioquímica i Genètica Molecular, Hospital Clínic de Barcelona, 08028 Barcelona, Spain.<br /><searchLink fieldCode="AU" term="%22van+Karnebeek+CDM%22">van Karnebeek CDM</searchLink>; Departments of Pediatrics and Human Genetics, Emma Center for Personalized Medicine, Amsterdam Gastro-Enterology Endocrinology Metabolism, Amsterdam University Medical Centers, Amsterdam, the Netherlands.<br /><searchLink fieldCode="AU" term="%22García-Villoria+J%22">García-Villoria J</searchLink>; Institut d'Investigacions Biomèdiques August Pi i Sunyer (IDIBAPS), 08036 Barcelona, Spain; Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER), 08036 Barcelona, Spain; Secció d'Errors Congènits del Metabolisme-IBC, Servei de Bioquímica i Genètica Molecular, Hospital Clínic de Barcelona, 08028 Barcelona, Spain.<br /><searchLink fieldCode="AU" term="%22Fillat+C%22">Fillat C</searchLink>; Institut d'Investigacions Biomèdiques August Pi i Sunyer (IDIBAPS), 08036 Barcelona, Spain; Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER), 08036 Barcelona, Spain; Facultat de Medicina i Ciències de la Salut, Universitat de Barcelona, 08036 Barcelona, Spain. Electronic address: cfillat@recerca.clinic.cat.
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  Data: <searchLink fieldCode="JN" term="%22100890581%22">Molecular therapy : the journal of the American Society of Gene Therapy</searchLink> [Mol Ther] 2025 Oct 01; Vol. 33 (10), pp. 4820-4833. <i>Date of Electronic Publication: </i>2025 Jul 17.
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  Data: <i>Publisher: </i><searchLink fieldCode="PB" term="%22Cell+Press%22">Cell Press </searchLink><i>Country of Publication: </i>United States <i>NLM ID: </i>100890581 <i>Publication Model: </i>Print-Electronic <i>Cited Medium: </i>Internet <i>ISSN: </i>1525-0024 (Electronic) <i>Linking ISSN: </i><searchLink fieldCode="IS" term="%2215250016%22">15250016 </searchLink><i>NLM ISO Abbreviation: </i>Mol Ther <i>Subsets: </i>MEDLINE
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