5 Year Expression and Neutrophil Defect Repair after Gene Therapy in Alpha-1 Antitrypsin Deficiency

Alpha-1 antitrypsin deficiency is a monogenic disorder resulting in emphysema due principally to the unopposed effects of neutrophil elastase. We previously reported achieving plasma wild-type alpha-1 antitrypsin concentrations at 2.5%–3.8% of the purported therapeutic level at 1 year after a single...

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Veröffentlicht in:	Molecular therapy 2017-06, Vol.25 (6), p.1387-1394
Hauptverfasser:	Mueller, Christian, Gernoux, Gwladys, Gruntman, Alisha M., Borel, Florie, Reeves, Emer P., Calcedo, Roberto, Rouhani, Farshid N., Yachnis, Anthony, Humphries, Margaret, Campbell-Thompson, Martha, Messina, Louis, Chulay, Jeffrey D., Trapnell, Bruce, Wilson, James M., McElvaney, Noel G., Flotte, Terence R.
Format:	Artikel
Sprache:	eng
Schlagworte:	A1AT AAT AAV alpha 1-Antitrypsin - genetics alpha 1-Antitrypsin - metabolism alpha 1-Antitrypsin Deficiency - genetics alpha 1-Antitrypsin Deficiency - metabolism alpha 1-Antitrypsin Deficiency - therapy alpha-1 antitrypsin Biomarkers Biopsy Capsid - immunology clinical trial Cytotoxicity Degranulation Dependovirus - genetics Dependovirus - immunology Elastase Emphysema Epitopes, T-Lymphocyte - immunology exhausted T cells Flow cytometry Gene Expression Gene therapy Genetic Therapy Genetic Vectors - administration & dosage Genetic Vectors - genetics Genetic Vectors - immunology Histology Humans Immunohistochemistry Immunophenotyping Lung diseases Lymphocytes Lymphocytes T Muscles - metabolism Muscles - pathology Mutation Neutrophils Neutrophils - enzymology Neutrophils - metabolism Original Patients PD-1 Proteins rAAV T-Lymphocyte Subsets - immunology T-Lymphocyte Subsets - metabolism T-Lymphocytes, Regulatory - immunology T-Lymphocytes, Regulatory - metabolism Time Factors Transgenes Tregs
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