AAV-1–mediated gene transfer to skeletal muscle in humans results in dose-dependent activation of capsid-specific T cells
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 high-dose cohort experienced a transient increase in the muscle enzyme creatine phosphokinase (CPK) 4 weeks after gene transfer. Simultaneous...
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| Published in | Blood Vol. 114; no. 10; pp. 2077 - 2086 |
|---|---|
| Main Authors | , , , , , , , , , , |
| Format | Journal Article |
| Language | English |
| Published |
Washington, DC
Elsevier Inc
03.09.2009
Americain Society of Hematology American Society of Hematology |
| Series | Gene Therapy |
| Subjects | |
| Online Access | Get full text |
| ISSN | 0006-4971 1528-0020 1528-0020 |
| DOI | 10.1182/blood-2008-07-167510 |
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| Abstract | 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 high-dose cohort experienced a transient increase in the muscle enzyme creatine phosphokinase (CPK) 4 weeks after gene transfer. Simultaneously, after an initial downward trend consistent with expression of LPL, plasma triglyceride levels returned to baseline. We characterized B- and T-cell responses to the vector and the transgene product in the subjects enrolled in this study. IFN-γ enzyme-linked immunosorbent spot (ELISpot) and intracellular cytokine staining assays performed on peripheral blood mononuclear cells (PBMCs) from the subject who experienced the CPK elevation showed the activation of capsid-specific CD4+ and CD8+ T cells. Four of 8 subjects had detectable T-cell responses to capsid with dose-dependent kinetics of appearance. Subjects with detectable T-cell responses to capsid also had higher anti–AAV-1 IgG3 antibody titer. No subject developed B- or T-cell responses to the LPL transgene product. These findings suggest that T-cell responses directed to the AAV-1 capsid are dose-dependent. Whether they also limit the duration of expression of the transgene at higher doses is unclear, and will require additional analyses at later time points. |
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| AbstractList | 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 high-dose cohort experienced a transient increase in the muscle enzyme creatine phosphokinase (CPK) 4 weeks after gene transfer. Simultaneously, after an initial downward trend consistent with expression of LPL, plasma triglyceride levels returned to baseline. We characterized B- and T-cell responses to the vector and the transgene product in the subjects enrolled in this study. IFN-γ enzyme-linked immunosorbent spot (ELISpot) and intracellular cytokine staining assays performed on peripheral blood mononuclear cells (PBMCs) from the subject who experienced the CPK elevation showed the activation of capsid-specific CD4+ and CD8+ T cells. Four of 8 subjects had detectable T-cell responses to capsid with dose-dependent kinetics of appearance. Subjects with detectable T-cell responses to capsid also had higher anti–AAV-1 IgG3 antibody titer. No subject developed B- or T-cell responses to the LPL transgene product. These findings suggest that T-cell responses directed to the AAV-1 capsid are dose-dependent. Whether they also limit the duration of expression of the transgene at higher doses is unclear, and will require additional analyses at later time points. 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 high-dose cohort experienced a transient increase in the muscle enzyme creatine phosphokinase (CPK) 4 weeks after gene transfer. Simultaneously, after an initial downward trend consistent with expression of LPL, plasma triglyceride levels returned to baseline. We characterized B- and T-cell responses to the vector and the transgene product in the subjects enrolled in this study. IFN-γ enzyme-linked immunosorbent spot (ELISpot) and intracellular cytokine staining assays performed on peripheral blood mononuclear cells (PBMCs) from the subject who experienced the CPK elevation showed the activation of capsid-specific CD4 + and CD8 + T cells. Four of 8 subjects had detectable T-cell responses to capsid with dose-dependent kinetics of appearance. Subjects with detectable T-cell responses to capsid also had higher anti–AAV-1 IgG3 antibody titer. No subject developed B- or T-cell responses to the LPL transgene product. These findings suggest that T-cell responses directed to the AAV-1 capsid are dose-dependent. Whether they also limit the duration of expression of the transgene at higher doses is unclear, and will require additional analyses at later time points. 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 high-dose cohort experienced a transient increase in the muscle enzyme creatine phosphokinase (CPK) 4 weeks after gene transfer. Simultaneously, after an initial downward trend consistent with expression of LPL, plasma triglyceride levels returned to baseline. We characterized B- and T-cell responses to the vector and the transgene product in the subjects enrolled in this study. IFN-gamma enzyme-linked immunosorbent spot (ELISpot) and intracellular cytokine staining assays performed on peripheral blood mononuclear cells (PBMCs) from the subject who experienced the CPK elevation showed the activation of capsid-specific CD4(+) and CD8(+) T cells. Four of 8 subjects had detectable T-cell responses to capsid with dose-dependent kinetics of appearance. Subjects with detectable T-cell responses to capsid also had higher anti-AAV-1 IgG3 antibody titer. No subject developed B- or T-cell responses to the LPL transgene product. These findings suggest that T-cell responses directed to the AAV-1 capsid are dose-dependent. Whether they also limit the duration of expression of the transgene at higher doses is unclear, and will require additional analyses at later time points.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 high-dose cohort experienced a transient increase in the muscle enzyme creatine phosphokinase (CPK) 4 weeks after gene transfer. Simultaneously, after an initial downward trend consistent with expression of LPL, plasma triglyceride levels returned to baseline. We characterized B- and T-cell responses to the vector and the transgene product in the subjects enrolled in this study. IFN-gamma enzyme-linked immunosorbent spot (ELISpot) and intracellular cytokine staining assays performed on peripheral blood mononuclear cells (PBMCs) from the subject who experienced the CPK elevation showed the activation of capsid-specific CD4(+) and CD8(+) T cells. Four of 8 subjects had detectable T-cell responses to capsid with dose-dependent kinetics of appearance. Subjects with detectable T-cell responses to capsid also had higher anti-AAV-1 IgG3 antibody titer. No subject developed B- or T-cell responses to the LPL transgene product. These findings suggest that T-cell responses directed to the AAV-1 capsid are dose-dependent. Whether they also limit the duration of expression of the transgene at higher doses is unclear, and will require additional analyses at later time points. 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 high-dose cohort experienced a transient increase in the muscle enzyme creatine phosphokinase (CPK) 4 weeks after gene transfer. Simultaneously, after an initial downward trend consistent with expression of LPL, plasma triglyceride levels returned to baseline. We characterized B- and T-cell responses to the vector and the transgene product in the subjects enrolled in this study. IFN-gamma enzyme-linked immunosorbent spot (ELISpot) and intracellular cytokine staining assays performed on peripheral blood mononuclear cells (PBMCs) from the subject who experienced the CPK elevation showed the activation of capsid-specific CD4(+) and CD8(+) T cells. Four of 8 subjects had detectable T-cell responses to capsid with dose-dependent kinetics of appearance. Subjects with detectable T-cell responses to capsid also had higher anti-AAV-1 IgG3 antibody titer. No subject developed B- or T-cell responses to the LPL transgene product. These findings suggest that T-cell responses directed to the AAV-1 capsid are dose-dependent. Whether they also limit the duration of expression of the transgene at higher doses is unclear, and will require additional analyses at later time points. |
| Author | Betts, Michael R. Hui, Daniel J. Mingozzi, Federico Basner-Tschakarjan, Etiena Hasbrouck, Nicole C. Meulenberg, Janneke J. Edmonson, Shyrie A. Hutnick, Natalie A. Kastelein, John J. Stroes, Erik S. High, Katherine A. |
| Author_xml | – sequence: 1 givenname: Federico surname: Mingozzi fullname: Mingozzi, Federico organization: Division of Hematology and Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, PA – sequence: 2 givenname: Janneke J. surname: Meulenberg fullname: Meulenberg, Janneke J. organization: Amsterdam Molecular Therapeutics, Amsterdam, The Netherlands – sequence: 3 givenname: Daniel J. surname: Hui fullname: Hui, Daniel J. organization: Division of Hematology and Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, PA – sequence: 4 givenname: Etiena surname: Basner-Tschakarjan fullname: Basner-Tschakarjan, Etiena organization: Division of Hematology and Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, PA – sequence: 5 givenname: Nicole C. surname: Hasbrouck fullname: Hasbrouck, Nicole C. organization: Division of Hematology and Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, PA – sequence: 6 givenname: Shyrie A. surname: Edmonson fullname: Edmonson, Shyrie A. organization: Division of Hematology and Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, PA – sequence: 7 givenname: Natalie A. surname: Hutnick fullname: Hutnick, Natalie A. organization: Department of Microbiology, University of Pennsylvania, Philadelphia – sequence: 8 givenname: Michael R. surname: Betts fullname: Betts, Michael R. organization: Department of Microbiology, University of Pennsylvania, Philadelphia – sequence: 9 givenname: John J. surname: Kastelein fullname: Kastelein, John J. organization: Department of Vascular Medicine, Academic Medical Center, Amsterdam, The Netherlands – sequence: 10 givenname: Erik S. surname: Stroes fullname: Stroes, Erik S. organization: Department of Vascular Medicine, Academic Medical Center, Amsterdam, The Netherlands – sequence: 11 givenname: Katherine A. surname: High fullname: High, Katherine A. email: high@email.chop.edu organization: Division of Hematology and Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, PA |
| BackLink | http://pascal-francis.inist.fr/vibad/index.php?action=getRecordDetail&idt=21904920$$DView record in Pascal Francis https://www.ncbi.nlm.nih.gov/pubmed/19506302$$D View this record in MEDLINE/PubMed |
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| Issue | 10 |
| Keywords | Human Adenoviridae Acquired immunity Cellular immunity Activation Striated muscle Genetic transfer Virus Adenovirus 1 Human adenovirus T-Lymphocyte Mastadenovirus Capsid Gene therapy Dose Vector |
| Language | English |
| License | This article is made available under the Elsevier license. CC BY 4.0 |
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| PublicationSeriesTitle | Gene Therapy |
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| Publisher | Elsevier Inc Americain Society of Hematology American Society of Hematology |
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| Snippet | 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... 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... |
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| SubjectTerms | Antibodies, Viral - blood Antibodies, Viral - immunology B-Lymphocytes - immunology Biological and medical sciences Capsid - immunology Capsid - metabolism CD4-Positive T-Lymphocytes - immunology CD4-Positive T-Lymphocytes - metabolism CD8-Positive T-Lymphocytes - immunology CD8-Positive T-Lymphocytes - metabolism Creatine Kinase - biosynthesis Creatine Kinase - immunology Dependovirus - genetics Dependovirus - immunology Dose-Response Relationship, Immunologic Female Fundamental and applied biological sciences. Psychology Gene Therapy Genetic Therapy Humans Hyperlipoproteinemia Type I - enzymology Hyperlipoproteinemia Type I - genetics Hyperlipoproteinemia Type I - immunology Hyperlipoproteinemia Type I - therapy Immunoenzyme Techniques Immunoglobulin G - blood Immunoglobulin G - immunology Interferon-gamma - biosynthesis Interferon-gamma - immunology Lipoprotein Lipase - biosynthesis Lipoprotein Lipase - genetics Lipoprotein Lipase - immunology Lymphocyte Activation - genetics Lymphocyte Activation - immunology Male Microbiology Muscle, Skeletal - enzymology Muscle, Skeletal - immunology Techniques used in virology Transduction, Genetic Transgenes - genetics Transgenes - immunology Triglycerides - blood Virology |
| Title | AAV-1–mediated gene transfer to skeletal muscle in humans results in dose-dependent activation of capsid-specific T cells |
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