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Proceedings of the National Academy of Sciences of the United States of America, ISSN 0027-8424, 9/2009, Volume 106, Issue 38, pp. 16363 - 16368
Alpha-1 antitrypsin (AAT) deficiency is well-suited as a target for human gene transfer. We performed a phase 1, open-label, doseescalation clinical trial of a... 
T lymphocytes | Genetic vectors | Neutralizing antibodies | Enzyme linked immunosorbent assay | Antibodies | Clinical trials | Capsid | Transgenes | Gene therapy | Blood | CTL | Immune response | AAV | Pulmonary | MULTIDISCIPLINARY SCIENCES | pulmonary | gene therapy | NEUTROPHIL ELASTASE | VIRAL VECTORS | DEFICIENCY | SKELETAL-MUSCLE | PHARMACOKINETICS | immune response | INTRAMUSCULAR INJECTION | IMMUNE-RESPONSES | ADENOASSOCIATED VIRUS VECTORS | ALPHA-1-ANTITRYPSIN GENE | AUGMENTATION THERAPY | CD8-Positive T-Lymphocytes - cytology | Dependovirus - genetics | Genetic Vectors - administration & dosage | Humans | Middle Aged | Male | Capsid - enzymology | Antibodies, Viral - blood | Recombinant Fusion Proteins - metabolism | CD4-Positive T-Lymphocytes - immunology | T-Lymphocytes - metabolism | Time Factors | CD8-Positive T-Lymphocytes - metabolism | Adult | Female | alpha 1-Antitrypsin Deficiency - genetics | Cell Line | Gene Expression | alpha 1-Antitrypsin - genetics | alpha 1-Antitrypsin Deficiency - therapy | Enzyme-Linked Immunosorbent Assay | alpha 1-Antitrypsin - blood | CD4-Positive T-Lymphocytes - cytology | CD4-Positive T-Lymphocytes - metabolism | Dependovirus - immunology | Injections, Intramuscular | Genetic Vectors - genetics | Recombinant Fusion Proteins - blood | Capsid - immunology | T-Lymphocytes - cytology | Recombinant Fusion Proteins - genetics | T-Lymphocytes - immunology | Aged | alpha 1-Antitrypsin - metabolism | CD8-Positive T-Lymphocytes - immunology | Genetic Therapy - methods | Biological Sciences
Journal Article
Proceedings of the National Academy of Sciences of the United States of America, ISSN 0027-8424, 02/2017, Volume 114, Issue 7, pp. 1655 - 1659
Journal Article
Molecular Therapy, ISSN 1525-0016, 06/2017, Volume 25, Issue 6, pp. 1387 - 1394
Alpha-1 antitrypsin deficiency is a monogenic disorder resulting in emphysema due principally to the unopposed effects of neutrophil elastase. We previously... 
alpha-1 antitrypsin | clinical trial | rAAV | AAT | AAV | Tregs | gene therapy | A1AT | PD-1 | exhausted T cells | MEDICINE, RESEARCH & EXPERIMENTAL | CELLS | HEPATOCYTES | CLINICAL-TRIAL | TRANSFECTION | VECTOR | RESPONSES | TRANSGENE EXPRESSION | BIOTECHNOLOGY & APPLIED MICROBIOLOGY | LIVER | GENETICS & HEREDITY | ACCUMULATION | SECRETION | Immunohistochemistry | T-Lymphocyte Subsets - immunology | Dependovirus - genetics | Genetic Therapy | T-Lymphocytes, Regulatory - metabolism | Genetic Vectors - administration & dosage | Humans | Muscles - pathology | T-Lymphocytes, Regulatory - immunology | Time Factors | Epitopes, T-Lymphocyte - immunology | Muscles - metabolism | Transgenes | Neutrophils - metabolism | alpha 1-Antitrypsin Deficiency - genetics | Gene Expression | alpha 1-Antitrypsin - genetics | alpha 1-Antitrypsin Deficiency - therapy | Genetic Vectors - immunology | Neutrophils - enzymology | Dependovirus - immunology | Immunophenotyping | Genetic Vectors - genetics | alpha 1-Antitrypsin Deficiency - metabolism | Capsid - immunology | Biopsy | T-Lymphocyte Subsets - metabolism | Biomarkers | alpha 1-Antitrypsin - metabolism | Flow cytometry | Lung diseases | Neutrophils | Cytotoxicity | Histology | Lymphocytes T | Patients | Proteins | Lymphocytes | Degranulation | Elastase | Mutation | Gene therapy | Emphysema | Original
Journal Article
EMBO Molecular Medicine, ISSN 1757-4676, 03/2013, Volume 5, Issue 3, pp. 397 - 412
Alpha‐1‐anti‐trypsin deficiency is the most common genetic cause of liver disease in children and liver transplantation is currently the only available... 
TFEB | gene therapy | alpha‐1‐anti‐trypsin | autophagy | helper‐dependent adenoviral vector | Alpha-1-anti-trypsin | Helper-dependent adenoviral vector | Gene therapy | Autophagy | NONHUMAN-PRIMATES | MEDICINE, RESEARCH & EXPERIMENTAL | DEPENDENT ADENOVIRAL VECTORS | ALPHA-ANTITRYPSIN DEFICIENCY | LIVER-DISEASE | helper-dependent adenoviral vector | TRANSGENE EXPRESSION | alpha-1-anti-trypsin | HEMOPHILIA-B | ENDOPLASMIC-RETICULUM | ALPHA-1-ANTITRYPSIN DEFICIENCY | NF-KAPPA-B | MUTANT ALPHA-ANTITRYPSIN-Z | Liver - pathology | Liver - enzymology | Microtubule-Associated Proteins - genetics | Humans | NF-kappa B - metabolism | Mutation, Missense | Lysosomes - metabolism | Transfection | Time Factors | Liver Cirrhosis - metabolism | Autophagy - genetics | Microtubule-Associated Proteins - deficiency | Interleukin-6 - metabolism | Liver Cirrhosis - genetics | Disease Models, Animal | alpha 1-Antitrypsin Deficiency - genetics | Gene Transfer Techniques | Genetic Predisposition to Disease | alpha 1-Antitrypsin - genetics | alpha 1-Antitrypsin Deficiency - therapy | Mice, Inbred C57BL | Mice, Transgenic | Liver Cirrhosis - therapy | alpha 1-Antitrypsin Deficiency - metabolism | Mice, Knockout | Papio | Autophagy-Related Protein 7 | Phenotype | Animals | alpha 1-Antitrypsin Deficiency - complications | Basic Helix-Loop-Helix Leucine Zipper Transcription Factors - genetics | Liver Cirrhosis - pathology | Basic Helix-Loop-Helix Leucine Zipper Transcription Factors - metabolism | Mice | alpha 1-Antitrypsin - metabolism | HeLa Cells | Apoptosis | Genetic Therapy - methods | Proteins | Trypsin | Liver diseases | Genes | Genetic engineering | Genetic transcription | Health aspects | Phenotypes | Gene transfer | Syngeneic grafts | Cloning | Transgenic mice | Gene expression | Medical screening | Studies | Allografts | Plasmids | Fibrosis | Polymers | Phagocytosis | Deoxyribonucleic acid--DNA | Liver transplantation | Index Medicus
Journal Article
Human Gene Therapy, ISSN 1043-0342, 08/2018, Volume 29, Issue 8, pp. 853 - 860
Journal Article
Lancet, The, ISSN 0140-6736, 2015, Volume 386, Issue 9991, pp. 360 - 368
Journal Article
Journal of Clinical Investigation, ISSN 0021-9738, 12/2013, Volume 123, Issue 12, pp. 5310 - 5318
Journal Article
Journal of Clinical Investigation, ISSN 0021-9738, 01/2014, Volume 124, Issue 1, pp. 251 - 261
Journal Article