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

Molecular Therapy

Volumn 25, Issue 6, 2017, Pages 1387-1394

  Mueller, Christian ^a   Gernoux, Gwladys ^a   Gruntman, Alisha M ^a,b   Borel, Florie ^a   Reeves, Emer P ^c   Calcedo, Roberto ^d   Rouhani, Farshid N ^e   Yachnis, Anthony ^e   Humphries, Margaret ^a   Campbell Thompson, Martha ^e   Messina, Louis ^a   Chulay, Jeffrey D ^f   Trapnell, Bruce ^g   Wilson, James M ^d   McElvaney, Noel G ^c   Flotte, Terence R ^a  

^a UNIVERSITY OF MASSACHUSETTS MEDICAL SCHOOL   (United States)

^b TUFTS UNIVERSITY   (United States)

^c BEAUMONT HOSPITAL   (Ireland)

^d UNIVERSITY OF PENNSYLVANIA   (United States)

^e UNIVERSITY OF FLORIDA COLLEGE OF MEDICINE   (United States)

^f APPLIED GENETIC TECHNOLOGIES CORPORATION   (United States)

^g CINCINNATI CHILDREN'S HOSPITAL MEDICAL CENTER   (United States)

Author keywords

A1AT; AAT; AAV; alpha 1 antitrypsin; clinical trial; exhausted T cells; gene therapy; PD 1; rAAV; Tregs

Indexed keywords

ADENO ASSOCIATED VIRUS VECTOR; ALPHA 1 ANTITRYPSIN; GRANULOCYTE ANTIBODY; LEUKOCYTE ELASTASE; BIOLOGICAL MARKER; EPITOPE;

ADENO ASSOCIATED VIRUS 1; ALPHA 1 ANTITRYPSIN DEFICIENCY; ALPHA 1 ANTITRYPSIN GENE; ANTIGEN BINDING; ARTICLE; CELL DAMAGE; CELL REGENERATION; CELLULAR IMMUNITY; COHORT ANALYSIS; CONTROLLED STUDY; CYTOTOXIC T LYMPHOCYTE; DEGRANULATION; DISEASE ASSOCIATION; DRUG MEGADOSE; ENZYME INHIBITION; GENE THERAPY; HUMAN; HUMAN CELL; HUMAN TISSUE; IMMUNOLOGICAL TOLERANCE; MEMBRANE BINDING; MULTICENTER STUDY; NEUTROPHIL; NONHUMAN; OPEN STUDY; PHASE 2 CLINICAL TRIAL; PROTEIN EXPRESSION; REGULATORY T LYMPHOCYTE; VIRUS CAPSID; VIRUS RECOMBINANT; ADMINISTRATION AND DOSAGE; BIOPSY; DEPENDOPARVOVIRUS; ENZYMOLOGY; GENE EXPRESSION; GENE VECTOR; GENETICS; IMMUNOHISTOCHEMISTRY; IMMUNOLOGY; IMMUNOPHENOTYPING; METABOLISM; MUSCLE; PATHOLOGY; T LYMPHOCYTE SUBPOPULATION; TIME FACTOR; TRANSGENE;

ALPHA 1-ANTITRYPSIN; ALPHA 1-ANTITRYPSIN DEFICIENCY; BIOMARKERS; BIOPSY; CAPSID; DEPENDOVIRUS; EPITOPES, T-LYMPHOCYTE; GENE EXPRESSION; GENETIC THERAPY; GENETIC VECTORS; HUMANS; IMMUNOHISTOCHEMISTRY; IMMUNOPHENOTYPING; MUSCLES; NEUTROPHILS; T-LYMPHOCYTE SUBSETS; T-LYMPHOCYTES, REGULATORY; TIME FACTORS; TRANSGENES;

EID: 85017403198     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1016/j.ymthe.2017.03.029     Document Type: Article

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