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NEW ENGLAND JOURNAL OF MEDICINE (2014)
Gene Therapy Prolongs Survival and Restores Function in Murine and Canine Models of Myotubular Myopathy
Martin K. Childers et al.
SCIENCE TRANSLATIONAL MEDICINE (2014)
Drawing a high-resolution functional map of adeno-associated virus capsid by massively parallel sequencing
Kei Adachi et al.
NATURE COMMUNICATIONS (2014)
Novel Adeno-associated Viruses Derived From Pig Tissues Transduce Most Major Organs in Mice
Alexander Bello et al.
SCIENTIFIC REPORTS (2014)
Toll-like receptor signaling pathways
Takumi Kawasaki et al.
FRONTIERS IN IMMUNOLOGY (2014)
Pre-clinical assessment of immune responses to adeno-associated virus (AAV) vectors
Etiena Basner-Tschakarjan et al.
FRONTIERS IN IMMUNOLOGY (2014)
Mapping the AAV capsid host antibody response toward the development of second generation gene delivery vectors
Yu-Shan Tseng et al.
FRONTIERS IN IMMUNOLOGY (2014)
Immune responses to AAV-vectors, the Glybera example from bencn to bedside
Valerie Ferreira et al.
FRONTIERS IN IMMUNOLOGY (2014)
B-cell depletion is protective against anti-AAV capsid immune response: a human subject case study
M. Corti et al.
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT (2014)
Systemic gene transfer reveals distinctive muscle transduction profile of tyrosine mutant AAV-1,-6, and-9 in neonatal dogs
Chady H. Hakim et al.
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT (2014)
Engineered AAV vector minimizes in vivo targeting of transduced hepatocytes by capsid-specific CD8+ T cells
Ashley T. Martino et al.
BLOOD (2013)
Prevalence and pharmacological modulation of humoral immunity to AAV vectors in gene transfer to synovial tissue
F. Mingozzi et al.
GENE THERAPY (2013)
Efficacy and long-term safety of alipogene tiparvovec (AAV1-LPLS447X) gene therapy for lipoprotein lipase deficiency: an open-label trial
D. Gaudet et al.
GENE THERAPY (2013)
Global CNS gene delivery and evasion of anti-AAV-neutralizing antibodies by intrathecal AAV administration in non-human primates
S. J. Gray et al.
GENE THERAPY (2013)
Kinetics of Adeno-Associated Virus Serotype 2 (AAV2) and AAV8 Capsid Antigen Presentation In Vivo Are Identical
Yi He et al.
HUMAN GENE THERAPY (2013)
Human Treg responses allow sustained recombinant adeno-associated virus-mediated transgene expression
Christian Mueller et al.
JOURNAL OF CLINICAL INVESTIGATION (2013)
Adeno-associated virus capsid antigen presentation is dependent on endosomal escape
Chengwen Li et al.
JOURNAL OF CLINICAL INVESTIGATION (2013)
CpG-depleted adeno-associated virus vectors evade immune detection
Susan M. Faust et al.
JOURNAL OF CLINICAL INVESTIGATION (2013)
Whole body correction of mucopolysaccharidosis IIIA by intracerebrospinal fluid gene therapy
Virginia Haurigot et al.
JOURNAL OF CLINICAL INVESTIGATION (2013)
C-Reactive Protein (CRP) Is Essential for Efficient Systemic Transduction of Recombinant Adeno-Associated Virus Vector 1 (rAAV-1) and rAAV-6 in Mice
Jerome Denard et al.
JOURNAL OF VIROLOGY (2013)
Capsid Antibodies to Different Adeno-Associated Virus Serotypes Bind Common Regions
Brittney L. Gurda et al.
JOURNAL OF VIROLOGY (2013)
Mapping the Structural Determinants Responsible for Enhanced T Cell Activation to the Immunogenic Adeno-Associated Virus Capsid from Isolate Rhesus 32.33
Lauren E. Mays et al.
JOURNAL OF VIROLOGY (2013)
Minimizing the Inhibitory Effect of Neutralizing Antibody for Efficient Gene Expression in the Liver With Adeno-associated Virus 8 Vectors
Jun Mimuro et al.
MOLECULAR THERAPY (2013)
Differential Type I Interferon-dependent Transgene Silencing of Helper-dependent Adenoviral vs. Adeno-associated Viral Vectors In Vivo
Masataka Suzuki et al.
MOLECULAR THERAPY (2013)
The Role of Apoptosis in Immune Hyporesponsiveness Following AAV8 Liver Gene Transfer
Susan M. Faust et al.
MOLECULAR THERAPY (2013)
Modulation of CD8+ T cell responses to AAV vectors with IgG-derived MHC class II epitopes
Daniel J. Hui et al.
MOLECULAR THERAPY (2013)
Overcoming Preexisting Humoral Immunity to AAV Using Capsid Decoys
Federico Mingozzi et al.
SCIENCE TRANSLATIONAL MEDICINE (2013)
Pre-existing Anti-Adeno-Associated Virus Antibodies as a Challenge in AAV Gene Therapy
Vedell Louis Jeune et al.
HUMAN GENE THERAPY METHODS (2013)
Bioengineering of AAV2 Capsid at Specific Serine, Threonine, or Lysine Residues Improves Its Transduction Efficiency in Vitro and in Vivo
Nishanth Gabriel et al.
HUMAN GENE THERAPY METHODS (2013)
Gene Therapy for Leber Congenital Amaurosis Caused by RPE65 Mutations Safety and Efficacy in 15 Children and Adults Followed Up to 3 Years
Samuel G. Jacobson et al.
ARCHIVES OF OPHTHALMOLOGY (2012)
Factor IX expression in skeletal muscle of a severe hemophilia B patient 10 years after AAV-mediated gene transfer
George Buchlis et al.
BLOOD (2012)
Intracellular transport of recombinant adeno-associated virus vectors
M. Nonnenmacher et al.
GENE THERAPY (2012)
Toll-like receptor 2-mediated innate immune response in human nonparenchymal liver cells toward adeno-associated viral vectors
Marianna Hoesel et al.
HEPATOLOGY (2012)
Adeno-Associated Virus Serotype 9 Transduction in the Central Nervous System of Nonhuman Primates
Lluis Samaranch et al.
HUMAN GENE THERAPY (2012)
Transient and intensive pharmacological immunosuppression fails to improve AAV-based liver gene transfer in non-human primates
Carmen Unzu et al.
JOURNAL OF TRANSLATIONAL MEDICINE (2012)
Human Galectin 3 Binding Protein Interacts with Recombinant Adeno-Associated Virus Type 6
Jerome Denard et al.
JOURNAL OF VIROLOGY (2012)
Mapping a Neutralizing Epitope onto the Capsid of Adeno-Associated Virus Serotype 8
Brittney L. Gurda et al.
JOURNAL OF VIROLOGY (2012)
Single Amino Acid Modification of Adeno-Associated Virus Capsid Changes Transduction and Humoral Immune Profiles
Chengwen Li et al.
JOURNAL OF VIROLOGY (2012)
Self-complementary AAVs Induce More Potent Transgene Product-specific Immune Responses Compared to a Single-stranded Genome
TeLang Wu et al.
MOLECULAR THERAPY (2012)
Neutralizing Antibodies Against AAV Serotypes 1, 2, 6, and 9 in Sera of Commonly Used Animal Models
Kleopatra Rapti et al.
MOLECULAR THERAPY (2012)
MyD88 Signaling in B Cells Regulates the Production of Th1-dependent Antibodies to AAV
Muriel Sudres et al.
MOLECULAR THERAPY (2012)
Pharmacological Modulation of Humoral Immunity in a Nonhuman Primate Model of AAV Gene Transfer for Hemophilia B
Federico Mingozzi et al.
MOLECULAR THERAPY (2012)
Phase 1 Gene Therapy for Duchenne Muscular Dystrophy Using a Translational Optimized AAV Vector
Dawn E. Bowles et al.
MOLECULAR THERAPY (2012)
AAV2 Gene Therapy Readministration in Three Adults with Congenital Blindness
Jean Bennett et al.
SCIENCE TRANSLATIONAL MEDICINE (2012)
The genome of self-complementary adeno-associated viral vectors increases Toll-like receptor 9-dependent innate immune responses in the liver
Ashley T. Martino et al.
BLOOD (2011)
microRNA122-regulated transgene expression increases specificity of cardiac gene transfer upon intravenous delivery of AAV9 vectors
A. Geisler et al.
GENE THERAPY (2011)
Liver-specific microRNA-122 target sequences incorporated in AAV vectors efficiently inhibits transgene expression in the liver
C. Qiao et al.
GENE THERAPY (2011)
Impact of Pre-Existing Immunity on Gene Transfer to Nonhuman Primate Liver with Adeno-Associated Virus 8 Vectors
Lili Wang et al.
HUMAN GENE THERAPY (2011)
Phase 2 Clinical Trial of a Recombinant Adeno-Associated Viral Vector Expressing α1-Antitrypsin: Interim Results
Terence R. Flotte et al.
HUMAN GENE THERAPY (2011)
The Assembly-Activating Protein Promotes Capsid Assembly of Different Adeno-Associated Virus Serotypes
F. Sonntag et al.
JOURNAL OF VIROLOGY (2011)
A 10 Patient Case Report on the Impact of Plasmapheresis Upon Neutralizing Factors Against Adeno-associated Virus (AAV) Types 1, 2, 6, and 8
Virginie Monteilhet et al.
MOLECULAR THERAPY (2011)
Capsid-specific T-cell Responses to Natural Infections With Adeno-associated Viruses in Humans Differ From Those of Nonhuman Primates
Hua Li et al.
MOLECULAR THERAPY (2011)
Nonredundant Roles of IL-10 and TGF-β in Suppression of Immune Responses to Hepatic AAV-Factor IX Gene Transfer
Brad E. Hoffman et al.
MOLECULAR THERAPY (2011)
MicroRNA-regulated, Systemically Delivered rAAV9: A Step Closer to CNS-restricted Transgene Expression
Jun Xie et al.
MOLECULAR THERAPY (2011)
Adenovirus-Associated Virus Vector-Mediated Gene Transfer in Hemophilia B
Amit C. Nathwani et al.
NEW ENGLAND JOURNAL OF MEDICINE (2011)
Dosage Thresholds for AAV2 and AAV8 Photoreceptor Gene Therapy in Monkey
Luk H. Vandenberghe et al.
SCIENCE TRANSLATIONAL MEDICINE (2011)
Prevention and reversal of antibody responses against factor IX in gene therapy for hemophilia B
Sushrusha Nayak et al.
FRONTIERS IN MICROBIOLOGY (2011)
Prevalence of Serum IgG and Neutralizing Factors Against Adeno-Associated Virus (AAV) Types 1, 2, 5, 6, 8, and 9 in the Healthy Population: Implications for Gene Therapy Using AAV Vectors
Sylvie Boutin et al.
HUMAN GENE THERAPY (2010)
Host Range, Prevalence, and Genetic Diversity of Adenoviruses in Bats
Yan Li et al.
JOURNAL OF VIROLOGY (2010)
Cross-reactive immunologic material status affects treatment outcomes in Pompe disease infants
Priya S. Kishnani et al.
MOLECULAR GENETICS AND METABOLISM (2010)
High-efficiency Transduction and Correction of Murine Hemophilia B Using AAV2 Vectors Devoid of Multiple Surface-exposed Tyrosines
David M. Markusic et al.
MOLECULAR THERAPY (2010)
Proteasome Inhibitors Decrease AAV2 Capsid-derived Peptide Epitope Presentation on MHC Class I Following Transduction
Jonathan D. Finn et al.
MOLECULAR THERAPY (2010)
Brief Report: Dystrophin Immunity in Duchenne's Muscular Dystrophy.
Jerry R. Mendell et al.
NEW ENGLAND JOURNAL OF MEDICINE (2010)
Optimization of stealth adeno-associated virus vectors by randomization of immunogenic epitopes
Stephan Maersch et al.
VIROLOGY (2010)
Long-term correction of inhibitor-prone hemophilia B dogs treated with liver-directed AAV2-mediated factor IX gene therapy
Glenn P. Niemeyer et al.
BLOOD (2009)
Isolation and evaluation of novel adeno-associated virus sequences from porcine tissues
A. Bello et al.
GENE THERAPY (2009)
Hepatic Regulatory T Cells and Kupffer Cells Are Crucial Mediators of Systemic T Cell Tolerance to Antigens Targeting Murine Liver
Ekaterina Breous et al.
HEPATOLOGY (2009)
Natural and Adaptive Foxp3+ Regulatory T Cells: More of the Same or a Division of Labor?
Maria A. Curotto de Lafaille et al.
IMMUNITY (2009)
Calcium Upregulation by Percutaneous Administration of Gene Therapy in Cardiac Disease (CUPID Trial), a First-in-Human Phase 1/2 Clinical Trial
Brian E. Jaski et al.
JOURNAL OF CARDIAC FAILURE (2009)
Capsid antigen presentation flags human hepatocytes for destruction after transduction by adeno-associated viral vectors
Gary C. Pien et al.
JOURNAL OF CLINICAL INVESTIGATION (2009)
The TLR9-MyD88 pathway is critical for adaptive immune responses to adeno-associated virus gene therapy vectors in mice
Jiangao Zhu et al.
JOURNAL OF CLINICAL INVESTIGATION (2009)
Adeno-Associated Virus Capsid Structure Drives CD4-Dependent CD8+ T Cell Response to Vector Encoded Proteins
Lauren E. Mays et al.
JOURNAL OF IMMUNOLOGY (2009)
Worldwide Epidemiology of Neutralizing Antibodies to Adeno-Associated Viruses
Roberto Calcedo et al.
JOURNAL OF INFECTIOUS DISEASES (2009)
Cytotoxic-T-Lymphocyte-Mediated Elimination of Target Cells Transduced with Engineered Adeno-Associated Virus Type 2 Vector In Vivo
Chengwen Li et al.
JOURNAL OF VIROLOGY (2009)
Striatal Readministration of rAAV Vectors Reveals an Immune Response Against AAV2 Capsids That Can Be Circumvented
Carmen S. Peden et al.
MOLECULAR THERAPY (2009)
Impact of the Underlying Mutation and the Route of Vector Administration on Immune Responses to Factor IX in Gene Therapy for Hemophilia B
Ou Cao et al.
MOLECULAR THERAPY (2009)
Cellular immune response to cryptic epitopes during therapeutic gene transfer
Chengwen Li et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2009)
Distinct immune responses to transgene products from rAAV1 and rAAV8 vectors
Yuanqing Lu et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2009)
Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy
Mark L. Brantly et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2009)
Activation of natural regulatory T cells by IgG Fc-derived peptide Tregitopes
Anne S. De Groot et al.
BLOOD (2008)
Regulatory T cells and immune tolerance
Shimon Sakaguchi et al.
CELL (2008)
Complement is an essential component of the immune response to adeno-associated virus vectors
Anne K. Zaiss et al.
JOURNAL OF VIROLOGY (2008)
In vitro and in vivo gene therapy vector evolution via multispecies interbreeding and retargeting of adeno-associated viruses
Dirk Grimm et al.
JOURNAL OF VIROLOGY (2008)
DNA shuffling of adeno-associated virus yields functionally diverse viral progeny
James T. Koerber et al.
MOLECULAR THERAPY (2008)
Effect of gene therapy on visual function in Leber's congenital amaurosis
James W. B. Bainbridge et al.
NEW ENGLAND JOURNAL OF MEDICINE (2008)
Next generation of adeno-associated virus 2 vectors: Point mutations in tyrosines lead to high-efficiency transduction at lower doses
Li Zhong et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2008)
Adeno-associated virus Type 12 (AAV12): A novel AAV serotype with sialic acid- and heparan sulfate proteoglycan-independent transduction activity
Michael Schmidt et al.
JOURNAL OF VIROLOGY (2008)
A microRNA-regulated lentiviral vector mediates stable correction of hemophilia B mice
Brian D. Brown et al.
BLOOD (2007)
Modulation of tolerance to the transgene product in a nonhuman primate model of AAV-mediated gene transfer to liver
Federico Mingozzi et al.
BLOOD (2007)
Immune responses to AAV in clinical trials
Federico Mingozzi et al.
CURRENT GENE THERAPY (2007)
Induction and role of regulatory CD4+CD25+ T cells in tolerance to the transgene product following hepatic in vivo gene transfer
Ou Cao et al.
BLOOD (2007)
A role for exposed mannosylations in presentation of human therapeutic self-proteins to CD4+T lymphocytes
Suryasarathi Dasgupta et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2007)
CD8+ T-cell responses to adeno-associated virus capsid in humans
Federico Mingozzi et al.
NATURE MEDICINE (2007)
Effects of transient immunosuppression on adenoassociated, virus-mediated, liver-directed gene transfer in rhesus macaques and implications for human gene therapy
Haiyan Jiang et al.
BLOOD (2006)
Adeno-associated virus (AAV) capsid genes isolated from rat and mouse liver genomic DNA define two new AAV species distantly related to AAV-5
Michael A. Lochrie et al.
VIROLOGY (2006)
Heparin binding directs activation of T cells against adeno-associated virus serotype 2 capsid
Luk H. Vandenberghe et al.
NATURE MEDICINE (2006)
Loss of Siglec expression on T lymphocytes during human evolution
Dzung H. Nguyen et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2006)
Identification and characterization of novel adeno-associated virus isolates in ATCC virus stocks
Michael Schmidt et al.
JOURNAL OF VIROLOGY (2006)
APOBEC3A is a potent inhibitor of adeno-associated virus and retrotransposons
H Chen et al.
CURRENT BIOLOGY (2006)
Human immunoglobulin inhibits liver transduction by AAV vectors at low AAV2 neutralizing titers in SCID mice
CD Scallan et al.
BLOOD (2006)
Successful transduction of liver in hemophilia by AAV-factor IX and limitations imposed by the host immune response
CS Manno et al.
NATURE MEDICINE (2006)
Directed evolution of adeno-associated virus yields enhanced gene delivery vectors
N Maheshri et al.
NATURE BIOTECHNOLOGY (2006)
Mutations on the external surfaces of adeno-associated virus type 2 capsids that affect transduction and neutralization
MA Lochrie et al.
JOURNAL OF VIROLOGY (2006)
Novel caprine adeno-associated virus (AAV) capsid (AAV-Go.1) is closely related to the primate AAV-5 and has unique tropism and neutralization properties
AE Arbetman et al.
JOURNAL OF VIROLOGY (2005)
Utility of PEGylated recombinant adeno-associated viruses for gene transfer
HT Le et al.
JOURNAL OF CONTROLLED RELEASE (2005)
PEG conjugation moderately protects adeno-associated viral vectors against antibody neutralization
GK Lee et al.
BIOTECHNOLOGY AND BIOENGINEERING (2005)
Rapamycin selectively expands CD4+CD25+FoxP3+ regulatory T cells
M Battaglia et al.
BLOOD (2005)
Striatal delivery of rAAV-hAADC to rats with preexisting immunity to AAV
LM Sanftner et al.
MOLECULAR THERAPY (2004)
Induction of antigen-specific CD4+T-cell anergy and deletion by in vivo viral gene transfer
E Dobrzynski et al.
BLOOD (2004)
Adeno-associated virus and the development of adeno-associated virus vectors: A historical perspective
BJ Carter
MOLECULAR THERAPY (2004)
Safety and efficacy of factor IX gene transfer to skeletal muscle in murine and canine hemophilia B models by adeno-associated viral vector serotype 1
VR Arruda et al.
BLOOD (2004)
Clades of Adeno-associated viruses are widely disseminated in human tissues
GP Gao et al.
JOURNAL OF VIROLOGY (2004)
Genetic modifications of the adeno-associated virus type 2 capsid reduce the affinity and the neutralizing effects of human serum antibodies
NA Huttner et al.
GENE THERAPY (2003)
Induction of immune tolerance to coagulation factor IX antigen by in vivo hepatic gene transfer
F Mingozzi et al.
JOURNAL OF CLINICAL INVESTIGATION (2003)
AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B
CS Manno et al.
BLOOD (2003)
Differential activation of innate immune responses by adenovirus and adeno-associated virus vectors
AK Zaiss et al.
JOURNAL OF VIROLOGY (2002)
A deviant immune response to viral proteins and transgene product is generated on subretinal administration of adenovirus and adeno-associated virus
V Anand et al.
MOLECULAR THERAPY (2002)
Muscle-directed gene transfer and transient immune suppression result in sustained partial correction of canine hemophilia B caused by a null mutation
RW Herzog et al.
MOLECULAR THERAPY (2001)
Cutting edge:: Control of CD8+ T cell activation by CD4+CD25+ immunoregulatory cells
CA Piccirillo et al.
JOURNAL OF IMMUNOLOGY (2001)
Epitope mapping of human anti-adeno-associated virus type 2 neutralizing antibodies: Implications for gene therapy and virus structure
M Moskalenko et al.
JOURNAL OF VIROLOGY (2000)
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