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Molecular Therapy - Methods & Clinical Development, ISSN 2329-0501, 06/2019, Volume 13, pp. 233 - 242
Phase 1 and phase 2 gene therapy trials using intramuscular (IM) administration of a recombinant adeno-associated virus serotype 1 (rAAV1) for replacement of... 
Journal Article
Journal Article
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
Clinical Infectious Diseases, ISSN 1058-4838, 10/2002, Volume 35, Issue 7, pp. 825 - 833
Journal Article
Journal of Clinical Investigation, ISSN 0021-9738, 12/2013, Volume 123, Issue 12, pp. 5310 - 5318
Journal Article
Journal Article
Molecular Therapy, ISSN 1525-0016, 08/2017, Volume 25, Issue 8, pp. 1866 - 1880
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
Molecular Therapy, ISSN 1525-0016, 05/2015, Volume 23, pp. S186 - S187
Journal Article
Journal Article
Clinical Infectious Diseases, ISSN 1058-4838, 10/2001, Volume 33, Issue 7, pp. 1015 - 1021
Journal Article
Investigative ophthalmology & visual science, ISSN 0146-0404, 12/2018, Volume 59, Issue 15, pp. 5941 - 5956
PURPOSE. Prospective evaluation of patients with X-linked retinoschisis (XLRS). METHODS. Fifty-six males XLRS patients, age >7 years, had retinal structure and... 
Cyst cavity volume | XLRS | Retinoschisis | VISUAL-ACUITY | retinoschisis | ACETAZOLAMIDE | TOPICAL DORZOLAMIDE | VARIABILITY | cyst cavity volume | MOUSE MODEL | CYSTIC MACULAR LESIONS | OPHTHALMOLOGY | JUVENILE RETINOSCHISIS | GENE-THERAPY | MUTATIONS | CARBONIC-ANHYDRASE INHIBITORS | Retina
Journal Article
Molecular Therapy, ISSN 1525-0016, 05/2015, Volume 23, pp. S79 - S79
Journal Article
Proceedings of the National Academy of Sciences of the United States of America, ISSN 0027-8424, 09/2009, Volume 106, Issue 38, p. 16363
  Alpha-1 antitrypsin (AAT) deficiency is well-suited as a target for human gene transfer. We performed a phase 1, open-label, dose-escalation clinical trial... 
Enzymes | Lymphocytes | Genomics | Viruses | Gene expression | Gene therapy | Cells
Journal Article