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Human gene therapy, ISSN 1043-0342, 10/2019, Volume 30, Issue 10, pp. 1204 - 1210
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Blood, ISSN 0006-4971, 08/2013, Volume 122, Issue 6, pp. 863 - 871
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PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, ISSN 0027-8424, 10/2019, Volume 116, Issue 42, pp. 21150 - 21159
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HUMAN GENE THERAPY, ISSN 1043-0342, 10/2019, Volume 30, Issue 10, pp. 1190 - 1203
In vertebrates, the liver is the central metabolic organ of the body, which carries out an estimated 500 functions that range from general detoxification to... 
MEDICINE, RESEARCH & EXPERIMENTAL | LONG-TERM CORRECTION | liver | DEPENDENT ADENOVIRUS VECTOR | ADENOASSOCIATED VIRUS VECTOR | gene editing | silencing | MESSENGER-RNA THERAPY | MURINE MODEL | GLYCOGEN-STORAGE-DISEASE | BIOTECHNOLOGY & APPLIED MICROBIOLOGY | ORNITHINE TRANSCARBAMYLASE DEFICIENCY | MOUSE MODEL | IN-VIVO | GENETICS & HEREDITY | rare inherited diseases | gene addition | METHYLMALONIC ACIDEMIA | Hemophilia B - therapy | Dependovirus - genetics | Liver - pathology | Liver Transplantation | Metabolic Diseases - pathology | Humans | Liver Diseases - pathology | Hepatocytes - pathology | Hepatocytes - metabolism | Lentivirus - metabolism | Metabolic Diseases - therapy | Hemophilia B - pathology | Lentivirus - genetics | Gene Transfer Techniques | Liver Diseases - genetics | Dependovirus - metabolism | Genetic Vectors - chemistry | Liver - metabolism | Genetic Vectors - metabolism | Liver Diseases - therapy | Clinical Trials as Topic | Hemophilia B - genetics | Hemophilia A - therapy | Hemophilia A - pathology | Hemophilia A - metabolism | Animals | Metabolic Diseases - metabolism | Metabolic Diseases - genetics | Gene Editing - methods | Hemophilia A - genetics | Liver Diseases - metabolism | Hemophilia B - metabolism | Genetic Therapy - methods | Syngeneic grafts | Liver | Disorders | Clinical trials | Transplantation | Bilirubin | Protein turnover | Proteins | Allografts | Restoration | Hemophilia | Genetic modification | Carbohydrates | Liver diseases | Immune response | Detoxification | Protein biosynthesis | Metabolism | Gene expression | Vertebrates | Hepatocytes | Protein synthesis | Fats | Gene therapy | Liver transplantation | Bile
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American Journal of Hematology, ISSN 0361-8609, 11/2015, Volume 90, Issue 11, pp. 1027 - 1035
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American Journal of Hematology, ISSN 0361-8609, 02/2013, Volume 88, Issue 2, pp. 135 - 140
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Blood, ISSN 0006-4971, 03/2006, Volume 107, Issue 5, pp. 1903 - 1907
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