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AAV-based gene therapy prevents and halts the progression of dilated cardiomyopathy in a mouse model of phosphoglucomutase 1 deficiency (PGM1-CDG).
Balakrishnan, Bijina; Altassan, Ruqaiah; Budhraja, Rohit; Liou, Willisa; Lupo, Arielle; Bryant, Sarah; Mankouski, Anastasiya; Radenkovic, Silvia; Preston, Graeme J; Pandey, Akhilesh; Boudina, Sihem; Kozicz, Tamas; Morava-Kozicz, Eva; Lai, Kent.
Afiliación
  • Balakrishnan B; Division of Medical Genetics, Department of Pediatrics, University of Utah, Salt Lake City, Utah, USA.
  • Altassan R; Department of Medical Genomics, Centre for Genomic Medicine, King Faisal Specialist Hospital and Research Center, Riyadh, Saudi Arabia.
  • Budhraja R; Department of Laboratory Medicine and Pathology, Mayo Clinic, Rochester, Minnesota, USA.
  • Liou W; Electron Microscopy Core Facility, University of Utah, Salt Lake City, Utah, USA.
  • Lupo A; Division of Medical Genetics, Department of Pediatrics, University of Utah, Salt Lake City, Utah, USA.
  • Bryant S; Division of Medical Genetics, Department of Pediatrics, University of Utah, Salt Lake City, Utah, USA.
  • Mankouski A; Division of Neonatology, Department of Pediatrics, University of Utah, Salt Lake City, Utah, USA.
  • Radenkovic S; Department of Clinical Genomics, Center of Individualized Medicine, Mayo Clinic, Rochester, Minnesota, USA.
  • Preston GJ; Department of Clinical Genomics, Center of Individualized Medicine, Mayo Clinic, Rochester, Minnesota, USA.
  • Pandey A; Department of Laboratory Medicine and Pathology, Mayo Clinic, Rochester, Minnesota, USA; Manipal Academy of Higher Education (MAHE), Manipal, Karnataka, India.
  • Boudina S; Department of Nutrition and Integrative Physiology, College of Health, University of Utah, Salt Lake City, Utah, USA.
  • Kozicz T; Department of Laboratory Medicine and Pathology, Mayo Clinic, Rochester, Minnesota, USA; Department of Clinical Genomics, Center of Individualized Medicine, Mayo Clinic, Rochester, Minnesota, USA; Department of Anatomy, University of Pecs School of Medicine, Pecs, Hungary.
  • Morava-Kozicz E; Department of Laboratory Medicine and Pathology, Mayo Clinic, Rochester, Minnesota, USA; Department of Clinical Genomics, Center of Individualized Medicine, Mayo Clinic, Rochester, Minnesota, USA; Department of Medical Genetics, University of Pecs, School of Medicine, Pecs, Hungary.
  • Lai K; Division of Medical Genetics, Department of Pediatrics, University of Utah, Salt Lake City, Utah, USA; Department of Nutrition and Integrative Physiology, College of Health, University of Utah, Salt Lake City, Utah, USA. Electronic address: kent.lai@hsc.utah.edu.
Transl Res ; 257: 1-14, 2023 07.
Article en En | MEDLINE | ID: mdl-36709920
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Texto completo: 1 Base de datos: MEDLINE Asunto principal: Cardiomiopatía Dilatada / Enfermedad del Almacenamiento de Glucógeno Idioma: En Revista: Transl Res Asunto de la revista: MEDICINA / TECNICAS E PROCEDIMENTOS DE LABORATORIO Año: 2023 Tipo del documento: Article
Texto completo
Imprimir
XML
PubMed Links
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Texto completo: 1 Base de datos: MEDLINE Asunto principal: Cardiomiopatía Dilatada / Enfermedad del Almacenamiento de Glucógeno Idioma: En Revista: Transl Res Asunto de la revista: MEDICINA / TECNICAS E PROCEDIMENTOS DE LABORATORIO Año: 2023 Tipo del documento: Article