| scholarly article | Q13442814 |
| P819 | ADS bibcode | 2009PNAS..10616363B |
| P356 | DOI | 10.1073/PNAS.0904514106 |
| P932 | PMC publication ID | 2752529 |
| P698 | PubMed publication ID | 19706466 |
| P5875 | ResearchGate publication ID | 26766540 |
| P50 | author | Michael R. Betts | Q88295419 |
| James Wilson | Q16947166 | ||
| P2093 | author name string | Lili Wang | |
| Roberto Calcedo | |||
| Christian Mueller | |||
| Barry J Byrne | |||
| Terence R Flotte | |||
| Mark L Brantly | |||
| Jeffrey D Chulay | |||
| Thomas J Conlon | |||
| Carolyn Spencer | |||
| Farshid Rouhani | |||
| L Terry Spencer | |||
| Margaret Humphries | |||
| P2860 | cites work | Alpha1-antitrypsin gene therapy modulates cellular immunity and efficiently prevents type 1 diabetes in nonobese diabetic mice | Q45859046 |
| Sustained and complete phenotype correction of hemophilia B mice following intramuscular injection of AAV1 serotype vectors | Q45879602 | ||
| Worldwide epidemiology of neutralizing antibodies to adeno-associated viruses. | Q45882349 | ||
| Preclinical evaluation of alpha-1-proteinase inhibitor. Pharmacokinetics and safety studies | Q46228948 | ||
| Use of a highly purified alpha 1-antitrypsin standard to establish ranges for the common normal and deficient alpha 1-antitrypsin phenotypes | Q46455576 | ||
| Clinical pharmacokinetics of alpha 1-antitrypsin in homozygous PiZ deficient patients | Q68026686 | ||
| Evaluation of the S-type of alpha-1-antitrypsin as an in vivo and in vitro inhibitor of neutrophil elastase | Q68090902 | ||
| Recombinant adeno-associated viral vectors mediate long-term transgene expression in muscle | Q73254110 | ||
| Evaluation of cellular immune responses in subjects chronically infected with HIV type 1 | Q79690113 | ||
| Augmentation therapy in alpha-1 antitrypsin deficiency | Q94693361 | ||
| Latent adeno-associated virus infection elicits humoral but not cell-mediated immune responses in a nonhuman primate model | Q24527302 | ||
| Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy | Q24535690 | ||
| HIV nonprogressors preferentially maintain highly functional HIV-specific CD8+ T cells | Q29616205 | ||
| Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response | Q29619095 | ||
| The alpha 1-antitrypsin gene and its deficiency states | Q34517557 | ||
| Production of clinical-grade recombinant adeno-associated virus vectors | Q35012878 | ||
| Adeno-associated viruses undergo substantial evolution in primates during natural infections | Q35022838 | ||
| Risk factors for emphysema. Cigarette smoking is associated with a reduction in the association rate constant of lung alpha 1-antitrypsin for neutrophil elastase | Q35820470 | ||
| Gene delivery to skeletal muscle results in sustained expression and systemic delivery of a therapeutic protein | Q35943644 | ||
| Sustained secretion of human alpha-1-antitrypsin from murine muscle transduced with adeno-associated virus vectors | Q36734619 | ||
| The alpha 1-antitrypsin gene and its mutations. Clinical consequences and strategies for therapy | Q38596155 | ||
| Production and purification of serotype 1, 2, and 5 recombinant adeno-associated viral vectors | Q40692830 | ||
| Impact of preexisting vector immunity on the efficacy of adeno-associated virus-based HIV-1 Gag vaccines. | Q42024301 | ||
| Intramuscular administration of recombinant adeno-associated virus 2 alpha-1 antitrypsin (rAAV-SERPINA1) vectors in a nonhuman primate model: safety and immunologic aspects | Q44136395 | ||
| Intravenous human plasma-derived augmentation therapy in alpha 1-antitrypsin deficiency: from pharmacokinetic analysis to individualizing therapy | Q44504515 | ||
| Characterization of plasmid DNA transfer into mouse skeletal muscle: evaluation of uptake mechanism, expression and secretion of gene products into blood. | Q45207733 | ||
| Preclinical characterization of a recombinant adeno-associated virus type 1-pseudotyped vector demonstrates dose-dependent injection site inflammation and dissemination of vector genomes to distant sites | Q45406023 | ||
| Phase I trial of intramuscular injection of a recombinant adeno-associated virus serotype 2 alphal-antitrypsin (AAT) vector in AAT-deficient adults | Q45409947 | ||
| Novel tools for production and purification of recombinant adenoassociated virus vectors | Q45752072 | ||
| P433 | issue | 38 | |
| P407 | language of work or name | English | Q1860 |
| P921 | main subject | gene therapy | Q213901 |
| lymphocyte | Q715347 | ||
| P1104 | number of pages | 6 | |
| P304 | page(s) | 16363-16368 | |
| P577 | publication date | 2009-08-12 | |
| P1433 | published in | Proceedings of the National Academy of Sciences of the United States of America | Q1146531 |
| P1476 | title | Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy | |
| P478 | volume | 106 |
| Q38837735 | 5 Year Expression and Neutrophil Defect Repair after Gene Therapy in Alpha-1 Antitrypsin Deficiency. |
| Q57819244 | A Destabilizing Domain Allows for Fast, Noninvasive, Conditional Control of Protein Abundance in the Mouse Eye - Implications for Ocular Gene Therapy |
| Q91814799 | A Novel Small Molecule Inhibits Intrahepatocellular Accumulation of Z-Variant Alpha 1-Antitrypsin In Vitro and In Vivo |
| Q34994549 | A barrel of monkeys: scAAV8 gene therapy for hemophilia in nonhuman primates. |
| Q34268108 | A review of augmentation therapy for alpha-1 antitrypsin deficiency |
| Q39615423 | A versatile adeno-associated virus vector producer cell line method for scalable vector production of different serotypes |
| Q39675325 | AAV Natural Infection Induces Broad Cross-Neutralizing Antibody Responses to Multiple AAV Serotypes in Chimpanzees |
| Q92876148 | AAV Vector Immunogenicity in Humans: A Long Journey to Successful Gene Transfer |
| Q36106408 | AAV capsid CD8+ T-cell epitopes are highly conserved across AAV serotypes |
| Q42206263 | AAV vectors expressing LDLR gain-of-function variants demonstrate increased efficacy in mouse models of familial hypercholesterolemia |
| Q33659389 | AAV's anatomy: roadmap for optimizing vectors for translational success |
| Q35733790 | AAV-mediated gene transfer in the perinatal period results in expression of FVII at levels that protect against fatal spontaneous hemorrhage. |
| Q34370111 | Adeno-associated virus (AAV) vectors in gene therapy: immune challenges and strategies to circumvent them. |
| Q41606960 | Adeno-associated virus serotype 1 (AAV1)- and AAV5-antibody complex structures reveal evolutionary commonalities in parvovirus antigenic reactivity |
| Q64088191 | Adeno-associated virus vectored immunoprophylaxis to prevent HIV in healthy adults: a phase 1 randomised controlled trial |
| Q34621723 | Adeno-associated virus vectors serotype 2 induce prolonged proliferation of capsid-specific CD8+ T cells in mice. |
| Q27026177 | Adeno-associated virus-mediated cancer gene therapy: current status |
| Q37272261 | Adeno-associated virus-mediated gene therapy for metabolic myopathy |
| Q35691659 | Adenovirus-associated virus vector-mediated gene transfer in hemophilia B |
| Q34810340 | Adipose tissue-derived mesenchymal stem cell-based liver gene delivery |
| Q38114852 | Alpha-1 antitrypsin deficiency: new developments in augmentation and other therapies |
| Q35063726 | Alpha-1 proteinase inhibitors for the treatment of alpha-1 antitrypsin deficiency: safety, tolerability, and patient outcomes |
| Q35892390 | Analyzing cellular immunity to AAV in a canine model using ELISPOT assay |
| Q34059643 | Anti-gp120 minibody gene transfer to female genital epithelial cells protects against HIV-1 virus challenge in vitro |
| Q38963940 | Assaying the Stability and Inactivation of AAV Serotype 1 Vectors. |
| Q37774788 | Augmentation therapy in alpha-1 antitrypsin deficiency: advances and controversies |
| Q41197115 | Autoimmunity, recessive diseases, and gene replacement therapy. |
| Q34763584 | B-Cell Depletion is Protective Against Anti-AAV Capsid Immune Response: A Human Subject Case Study |
| Q56889545 | Bioengineered Viral Platform for Intramuscular Passive Vaccine Delivery to Human Skeletal Muscle |
| Q37316887 | Birth of a new therapeutic platform: 47 years of adeno-associated virus biology from virus discovery to licensed gene therapy |
| Q35568563 | Capsid-specific T-cell responses to natural infections with adeno-associated viruses in humans differ from those of nonhuman primates |
| Q36084447 | Cell and gene therapy for genetic diseases: inherited disorders affecting the lung and those mimicking sudden infant death syndrome |
| Q33938435 | Cell-Mediated Immunity to AAV Vectors, Evolving Concepts and Potential Solutions |
| Q36401337 | Center for fetal monkey gene transfer for heart, lung, and blood diseases: an NHLBI resource for the gene therapy community |
| Q28080450 | Challenges and Prospects for Alpha-1 Antitrypsin Deficiency Gene Therapy |
| Q34211507 | Characterization of a recombinant adeno-associated virus type 2 Reference Standard Material |
| Q64977765 | Circumventing cellular immunity by miR142-mediated regulation sufficiently supports rAAV-delivered OVA expression without activating humoral immunity. |
| Q40358542 | Class I-restricted T-cell responses to a polymorphic peptide in a gene therapy clinical trial for α-1-antitrypsin deficiency. |
| Q37980864 | Cocaine hydrolase gene therapy for cocaine abuse |
| Q35180848 | Combination therapy utilizing shRNA knockdown and an optimized resistant transgene for rescue of diseases caused by misfolded proteins |
| Q33832790 | Comparison of adeno-associated virus pseudotype 1, 2, and 8 vectors administered by intramuscular injection in the treatment of murine phenylketonuria |
| Q36966955 | CpG-depleted adeno-associated virus vectors evade immune detection. |
| Q37277411 | Current Challenges and Future Directions in Recombinant AAV-Mediated Gene Therapy of Duchenne Muscular Dystrophy |
| Q38264336 | Current status of gene therapy for α-1 antitrypsin deficiency |
| Q38584256 | Delivery of Adeno-Associated Virus Gene Therapy by Intravascular Limb Infusion Methods |
| Q57493992 | Destabilizing Domains Enable Long-Term and Inert Regulation of GDNF Expression in the Brain |
| Q38003108 | Detection of EPO gene doping in blood |
| Q90059174 | Detection of intra-articular gene therapy in horses using quantitative real time PCR in synovial fluid and plasma |
| Q38069585 | Development of Novel Recombinant AAV Vectors and Strategies for the Potential Gene Therapy of Hemophilia |
| Q30152887 | Development of Patient-specific AAV Vectors After Neutralizing Antibody Selection for Enhanced Muscle Gene Transfer |
| Q33832987 | Development of operational immunologic tolerance with neonatal gene transfer in nonhuman primates: preliminary studies |
| Q41590387 | Dystrophin immunity in Duchenne's muscular dystrophy. |
| Q36571141 | Effects of FVIII immunity on hepatocyte and hematopoietic stem cell-directed gene therapy of murine hemophilia A. |
| Q34311144 | Efficacy and long-term safety of alipogene tiparvovec (AAV1-LPLS447X) gene therapy for lipoprotein lipase deficiency: an open-label trial |
| Q35078437 | Employing a gain-of-function factor IX variant R338L to advance the efficacy and safety of hemophilia B human gene therapy: preclinical evaluation supporting an ongoing adeno-associated virus clinical trial |
| Q33833473 | Engineering liver-detargeted AAV9 vectors for cardiac and musculoskeletal gene transfer |
| Q41881145 | Enhanced long-term transduction and multilineage engraftment of human hematopoietic stem cells transduced with tyrosine-modified recombinant adeno-associated virus serotype 2. |
| Q34080880 | Ex vivo transduction and transplantation of bone marrow cells for liver gene delivery of alpha1-antitrypsin |
| Q38071683 | Expert opinion in biological therapy: update on developments in lung gene transfer |
| Q91938979 | Five Years of Successful Inducible Transgene Expression Following Locoregional Adeno-Associated Virus Delivery in Nonhuman Primates with No Detectable Immunity |
| Q33731154 | Gel-mediated delivery of AAV1 vectors corrects ventilatory function in Pompe mice with established disease |
| Q52721830 | Gene Therapy With Regulatory T Cells: A Beneficial Alliance. |
| Q38937862 | Gene Therapy for Alpha-1 Antitrypsin Deficiency Lung Disease |
| Q40103301 | Gene Therapy in Tyrosinemia: Potential and Pitfalls. |
| Q37969759 | Gene and cell therapy based treatment strategies for inflammatory bowel diseases |
| Q38260248 | Gene replacement therapy for genetic hepatocellular jaundice |
| Q57677438 | Gene therapy for Rett syndrome: prospects and challenges |
| Q37866000 | Gene therapy for alpha-1 antitrypsin deficiency |
| Q33881920 | Gene therapy for monogenic liver diseases: clinical successes, current challenges and future prospects. |
| Q41106554 | Gene transfer of master autophagy regulator TFEB results in clearance of toxic protein and correction of hepatic disease in alpha-1-anti-trypsin deficiency |
| Q40611143 | Generation and characterization of anti-Adeno-associated virus serotype 8 (AAV8) and anti-AAV9 monoclonal antibodies |
| Q35571114 | Generation and in vivo evaluation of IL10-treated dendritic cells in a nonhuman primate model of AAV-based gene transfer |
| Q37384103 | Human Treg responses allow sustained recombinant adeno-associated virus-mediated transgene expression |
| Q36086772 | Human galectin 3 binding protein interacts with recombinant adeno-associated virus type 6 |
| Q37236099 | Humoral Immune Response to AAV. |
| Q38195598 | Immune Responses to AAV-Vectors, the Glybera Example from Bench to Bedside |
| Q36983740 | Immune responses to AAV vectors: overcoming barriers to successful gene therapy |
| Q38287263 | Immune responses to intramuscular administration of alipogene tiparvovec (AAV1-LPL(S447X)) in a phase II clinical trial of lipoprotein lipase deficiency gene therapy |
| Q37943306 | Immunity and AAV-Mediated Gene Therapy for Muscular Dystrophies in Large Animal Models and Human Trials |
| Q36114228 | Immunosuppression decreases inflammation and increases AAV6-hSERCA2a-mediated SERCA2a expression |
| Q35575706 | Impact of pre-existing immunity on gene transfer to nonhuman primate liver with adeno-associated virus 8 vectors |
| Q35952312 | In Silico Reconstruction of the Viral Evolutionary Lineage Yields a Potent Gene Therapy Vector |
| Q52431137 | In vivo genome editing partially restores alpha1-antitrypsin in a murine model of AAT deficiency. |
| Q47110966 | Increasing the Clinical Potential and Applications of Anti-HIV Antibodies |
| Q38874623 | Induction of T-Cell Infiltration and Programmed Death Ligand 2 Expression by Adeno-Associated Virus in Rhesus Macaque Skeletal Muscle and Modulation by Prednisone |
| Q37082190 | Interception of cocaine by enzyme or antibody delivered with viral gene transfer: a novel strategy for preventing relapse in recovering drug users |
| Q40487347 | Intramuscular administration of AAV overcomes pre-existing neutralizing antibodies in rhesus macaques. |
| Q35418068 | Intramuscular injection of AAV8 in mice and macaques is associated with substantial hepatic targeting and transgene expression |
| Q64964706 | Intrapleural Gene Therapy for Alpha-1 Antitrypsin Deficiency-Related Lung Disease. |
| Q39639059 | Lack of immunotoxicity after regional intravenous (RI) delivery of rAAV to nonhuman primate skeletal muscle |
| Q30496514 | Liver-directed recombinant adeno-associated viral gene delivery rescues a lethal mouse model of methylmalonic acidemia and provides long-term phenotypic correction |
| Q36940126 | Liver-specific microRNA-122 target sequences incorporated in AAV vectors efficiently inhibits transgene expression in the liver |
| Q45868167 | Longevity of rAAV vector and plasmid DNA in blood after intramuscular injection in nonhuman primates: implications for gene doping |
| Q26750799 | Manufacturing of recombinant adeno-associated viral vectors for clinical trials |
| Q38187651 | Mapping the AAV Capsid Host Antibody Response toward the Development of Second Generation Gene Delivery Vectors |
| Q41363155 | Measuring immune responses to recombinant AAV gene transfer |
| Q36740284 | Microdystrophin ameliorates muscular dystrophy in the canine model of duchenne muscular dystrophy |
| Q39400043 | Microvesicle-associated AAV vector as a novel gene delivery system |
| Q33731233 | Modulation of exaggerated-IgE allergic responses by gene transfer-mediated antagonism of IL-13 and IL-17e |
| Q41347222 | Non-clinical Safety and Efficacy of an AAV2/8 Vector Administered Intravenously for Treatment of Mucopolysaccharidosis Type VI. |
| Q52429666 | Nuclease-Mediated Gene Therapies for Inherited Metabolic Diseases of the Liver. |
| Q40097321 | Optimized AAV rh.10 Vectors That Partially Evade Neutralizing Antibodies during Hepatic Gene Transfer |
| Q26858992 | Parvovirus glycan interactions |
| Q30358434 | Passive immunization against HIV/AIDS by antibody gene transfer. |
| Q33931862 | Peripheral transvenular delivery of adeno-associated viral vectors to skeletal muscle as a novel therapy for hemophilia B. |
| Q40709550 | Pharmacodynamics of anti-HIV gene therapy using viral vectors and targeted endonucleases. |
| Q34633598 | Phase 1 gene therapy for Duchenne muscular dystrophy using a translational optimized AAV vector |
| Q35496285 | Phase 2 clinical trial of a recombinant adeno-associated viral vector expressing α1-antitrypsin: interim results |
| Q36945774 | Phase I/II trial of adeno-associated virus-mediated alpha-glucosidase gene therapy to the diaphragm for chronic respiratory failure in Pompe disease: initial safety and ventilatory outcomes |
| Q34994501 | Phenotypic correction of a mouse model for primary hyperoxaluria with adeno-associated virus gene transfer |
| Q38012709 | Phoenix rising: gene therapy makes a comeback |
| Q40216716 | Plasmacytoid and conventional dendritic cells cooperate in crosspriming AAV capsid-specific CD8+ T cells. |
| Q34982843 | Pompe disease gene therapy. |
| Q38191212 | Pre-Clinical Assessment of Immune Responses to Adeno-Associated Virus (AAV) Vectors |
| Q34589262 | Preclinical Evaluation of a Recombinant Adeno-Associated Virus Vector Expressing Human Alpha-1 Antitrypsin Made Using a Recombinant Herpes Simplex Virus Production Method |
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| Q36322970 | Prevalence and pharmacological modulation of humoral immunity to AAV vectors in gene transfer to synovial tissue |
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| Q38168940 | Safety profile of recombinant adeno-associated viral vectors: focus on alipogene tiparvovec (Glybera®). |
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