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Gene Therapy 2017: Progress and Future Directions

Clinical and translational science, 2017-07, Vol.10 (4), p.242-248

2017

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Gene Therapy Progress and Prospects: Recombinant adeno-associated virus (rAAV) vectors

Gene therapy, 2004-05, Vol.11 (10), p.805-810

FLOTTE, T. R

2004

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Stable therapeutic serum levels of human alpha-1 antitrypsin (AAT) after portal vein injection of recombinant adeno-associated virus (rAAV) vectors

Gene therapy, 2001-09, Vol.8 (17), p.1299-1306

2001

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Production and purification of serotype 1, 2, and 5 recombinant adeno-associated viral vectors

Methods (San Diego, Calif.), 2002-10, Vol.28 (2), p.158-167

2002

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The Short Apical Membrane Half-life of Rescued ΔF508-Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Results from Accelerated Endocytosis of ΔF508-CFTR in Polarized Human Airway Epithelial Cells

The Journal of biological chemistry, 2005-11, Vol.280 (44), p.36762-36772

2005

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Adeno-associated virus-based gene therapy for inherited disorders

Pediatric research, 2005-12, Vol.58 (6), p.1143-1147

FLOTTE, Terence R

2005

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Phase I trial of intramuscular injection of a recombinant adeno-associated virus alpha 1-antitrypsin (rAAV2-CB-hAAT) gene vector to AAT-deficient adults

Human gene therapy, 2004-01, Vol.15 (1), p.93-128

2004

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Phase I trial of intranasal and endobronchial administration of a recombinant adeno-associated virus serotype 2 (rAAV2)-CFTR vector in adult cystic fibrosis patients: A two-part clinical study

Human gene therapy, 2003-07, Vol.14 (11), p.1079-1088

2003

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IL-10 suppresses chemokines, inflammation, and fibrosis in a model of chronic renal disease

Journal of the American Society of Nephrology, 2005-12, Vol.16 (12), p.3651-3660

2005

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Improved Method of Recombinant AAV2 Delivery for Systemic Targeted Gene Therapy

Molecular therapy, 2002-07, Vol.6 (1), p.106-112

2002

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Recent developments in recombinant AAV-mediated gene therapy for lung diseases

Current gene therapy, 2005-06, Vol.5 (3), p.361-366

Flotte, Terence R

2005

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A phase II, double-blind, randomized, placebo-controlled clinical trial of tgAAVCF using maxillary sinus delivery in patients with cystic fibrosis with antrostomies

Human gene therapy, 2002-07, Vol.13 (11), p.1349-1359

2002

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Adeno-associated virus : A ubiquitous commensal of mammals

Human gene therapy, 2005-04, Vol.16 (4), p.401-407

2005

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DNA-Dependent PK Inhibits Adeno-Associated Virus DNA Integration

Proceedings of the National Academy of Sciences - PNAS, 2004-02, Vol.101 (7), p.2112-2116

2004

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Viral Vector–mediated and Cell-based Therapies for Treatment of Cystic Fibrosis

Molecular therapy, 2007-02, Vol.15 (2), p.229-241

2007

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Interleukin 10 Attenuates Neointimal Proliferation and Inflammation in Aortic Allografts by a Heme Oxygenase-Dependent Pathway

Proceedings of the National Academy of Sciences - PNAS, 2005-05, Vol.102 (20), p.7251-7256

2005

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Sustained Secretion of Human Alpha-1-Antitrypsin from Murine Muscle Transduced with Adeno-Associated Virus Vectors

Proceedings of the National Academy of Sciences - PNAS, 1998-11, Vol.95 (24), p.14384-14388

1998

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Gene Therapy in Cystic Fibrosis

Chest, 2001-09, Vol.120 (3), p.124S-131S

2001

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CMV-β-actin promoter directs higher expression from an adeno-associated viral vector in the liver than the cytomegalovirus or elongation factor 1α promoter and results in therapeutic levels of human factor X in mice

Human gene therapy, 2001-03, Vol.12 (5), p.563-573

2001

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Systemic Overexpression of IL-10 Induces CD4+CD25+ Cell Populations In Vivo and Ameliorates Type 1 Diabetes in Nonobese Diabetic Mice in a Dose-Dependent Fashion

The Journal of immunology (1950), 2003-09, Vol.171 (5), p.2270-2278

2003

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Effect of DNA-Dependent Protein Kinase on the Molecular Fate of the rAAV2 Genome in Skeletal Muscle

Proceedings of the National Academy of Sciences - PNAS, 2001-03, Vol.98 (7), p.4084-4088

2001

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Efficient and persistent gene transfer of AAV-CFTR in maxillary sinus

The Lancet (British edition), 1998-06, Vol.351 (9117), p.1702-1703

1998

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Targeting recombinant adeno-associated virus vectors to enhance gene transfer to pancreatic islets and liver

Gene therapy, 2003-09, Vol.10 (18), p.1551-1558

2003

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Recombinant adeno-associated virus-mediated alpha-1 antitrypsin gene therapy prevents type I diabetes in NOD mice

Gene therapy, 2004-01, Vol.11 (2), p.181-186

2004

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Latent Adeno-Associated Virus Infection Elicits Humoral but Not Cell-Mediated Immune Responses in a Nonhuman Primate Model

Journal of Virology, 1999-10, Vol.73 (10), p.8549-8558

1999

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Gene Therapy 2017: Progress and Future Directions

Gene Therapy Progress and Prospects: Recombinant adeno-associated virus (rAAV) vectors

Stable therapeutic serum levels of human alpha-1 antitrypsin (AAT) after portal vein injection of recombinant adeno-associated virus (rAAV) vectors

Production and purification of serotype 1, 2, and 5 recombinant adeno-associated viral vectors

The Short Apical Membrane Half-life of Rescued ΔF508-Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Results from Accelerated Endocytosis of ΔF508-CFTR in Polarized Human Airway Epithelial Cells

Adeno-associated virus-based gene therapy for inherited disorders

Phase I trial of intramuscular injection of a recombinant adeno-associated virus alpha 1-antitrypsin (rAAV2-CB-hAAT) gene vector to AAT-deficient adults

Phase I trial of intranasal and endobronchial administration of a recombinant adeno-associated virus serotype 2 (rAAV2)-CFTR vector in adult cystic fibrosis patients: A two-part clinical study

IL-10 suppresses chemokines, inflammation, and fibrosis in a model of chronic renal disease

Improved Method of Recombinant AAV2 Delivery for Systemic Targeted Gene Therapy

Recent developments in recombinant AAV-mediated gene therapy for lung diseases

A phase II, double-blind, randomized, placebo-controlled clinical trial of tgAAVCF using maxillary sinus delivery in patients with cystic fibrosis with antrostomies

Adeno-associated virus : A ubiquitous commensal of mammals

DNA-Dependent PK Inhibits Adeno-Associated Virus DNA Integration

Viral Vector–mediated and Cell-based Therapies for Treatment of Cystic Fibrosis

Interleukin 10 Attenuates Neointimal Proliferation and Inflammation in Aortic Allografts by a Heme Oxygenase-Dependent Pathway

Sustained Secretion of Human Alpha-1-Antitrypsin from Murine Muscle Transduced with Adeno-Associated Virus Vectors

Gene Therapy in Cystic Fibrosis

CMV-β-actin promoter directs higher expression from an adeno-associated viral vector in the liver than the cytomegalovirus or elongation factor 1α promoter and results in therapeutic levels of human factor X in mice

Systemic Overexpression of IL-10 Induces CD4+CD25+ Cell Populations In Vivo and Ameliorates Type 1 Diabetes in Nonobese Diabetic Mice in a Dose-Dependent Fashion

Effect of DNA-Dependent Protein Kinase on the Molecular Fate of the rAAV2 Genome in Skeletal Muscle

Efficient and persistent gene transfer of AAV-CFTR in maxillary sinus

Targeting recombinant adeno-associated virus vectors to enhance gene transfer to pancreatic islets and liver

Recombinant adeno-associated virus-mediated alpha-1 antitrypsin gene therapy prevents type I diabetes in NOD mice

Latent Adeno-Associated Virus Infection Elicits Humoral but Not Cell-Mediated Immune Responses in a Nonhuman Primate Model

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