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Gene Therapy, ISSN 0969-7128, 04/2013, Volume 20, Issue 4, pp. 361 - 369
Journal Article
Environmental Health Perspectives, ISSN 0091-6765, 09/2017, Volume 125, Issue 9, pp. 097013 - 097013
Journal Article
Journal of Biological Chemistry, ISSN 0021-9258, 04/2006, Volume 281, Issue 15, pp. 10105 - 10117
Journal Article
Journal Article
Nature Medicine, ISSN 1078-8956, 07/2013, Volume 19, Issue 7, pp. 889 - 891
Journal Article
Blood, ISSN 0006-4971, 2009, Volume 114, Issue 10, pp. 2077 - 2086
In a clinical trial for adeno-associated virus serotype 1 (AAV-1)-mediated gene transfer to muscle for lipoprotein lipase (LPL) deficiency, 1 subject from the... 
RESPONSES | AAV VECTOR | LIPOPROTEIN-LIPASE DEFICIENCY | ADENOASSOCIATED VIRAL VECTOR | LONG-TERM CORRECTION | VIRUS | BENEFICIAL MUTATION | CANINE HEMOPHILIA-B | FACTOR-IX | HEMATOLOGY | LEBERS CONGENITAL AMAUROSIS | Dependovirus - genetics | Genetic Therapy | Dose-Response Relationship, Immunologic | Immunoglobulin G - blood | Humans | Male | Antibodies, Viral - blood | Immunoenzyme Techniques | CD4-Positive T-Lymphocytes - immunology | Lymphocyte Activation - genetics | Lymphocyte Activation - immunology | Muscle, Skeletal - immunology | Immunoglobulin G - immunology | CD8-Positive T-Lymphocytes - metabolism | Female | Capsid - metabolism | Creatine Kinase - immunology | Muscle, Skeletal - enzymology | Transduction, Genetic | Lipoprotein Lipase - genetics | Transgenes - genetics | CD4-Positive T-Lymphocytes - metabolism | Hyperlipoproteinemia Type I - enzymology | Dependovirus - immunology | Lipoprotein Lipase - biosynthesis | Hyperlipoproteinemia Type I - genetics | Capsid - immunology | B-Lymphocytes - immunology | Creatine Kinase - biosynthesis | Hyperlipoproteinemia Type I - immunology | Interferon-gamma - immunology | Lipoprotein Lipase - immunology | Triglycerides - blood | Antibodies, Viral - immunology | Hyperlipoproteinemia Type I - therapy | CD8-Positive T-Lymphocytes - immunology | Transgenes - immunology | Interferon-gamma - biosynthesis | Index Medicus | Abridged Index Medicus | Gene Therapy
Journal Article
JAMA, ISSN 0098-7484, 03/2017, Volume 317, Issue 9, pp. 937 - 946
Journal Article