Enhanced response to enzyme replacement therapy in Pompe disease after the induction of immune tolerance.
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Enzyme replacement therapy for infantile-onset Pompe diseaseTargeted approaches to induce immune tolerance for Pompe disease therapyNew insights into therapeutic options for Pompe diseaseUse of cardiac magnetic resonance imaging to evaluate cardiac structure, function and fibrosis in children with infantile Pompe disease on enzyme replacement therapy.Tolerance induction to cytoplasmic beta-galactosidase by hepatic AAV gene transfer: implications for antigen presentation and immunotoxicityImmunomodulatory gene therapy prevents antibody formation and lethal hypersensitivity reactions in murine pompe disease.Hepatic gene transfer as a means of tolerance induction to transgene productsHepatocyte-targeted HFE and TFR2 control hepcidin expression in mice.The role of hepatocyte hemojuvelin in the regulation of bone morphogenic protein-6 and hepcidin expression in vivo.Cell-Mediated Immunity to AAV Vectors, Evolving Concepts and Potential Solutions.Immunomodulatory gene therapy in lysosomal storage disordersMapping the T helper cell response to acid α-glucosidase in Pompe miceTherapeutic approaches for lysosomal storage diseasesThe impact of antibodies on clinical outcomes in diseases treated with therapeutic protein: lessons learned from infantile Pompe disease.Development of Gene Transfer for Induction of Antigen-specific Tolerance.Enhanced efficacy from gene therapy in Pompe disease using coreceptor blockadeEarly treatment with alglucosidase alpha prolongs long-term survival of infants with Pompe diseaseAssessment of toxicity and biodistribution of recombinant AAV8 vector-mediated immunomodulatory gene therapy in mice with Pompe disease.Systemic administration of AAV8-α-galactosidase A induces humoral tolerance in nonhuman primates despite low hepatic expressionComparative impact of AAV and enzyme replacement therapy on respiratory and cardiac function in adult Pompe micePreclinical Development of New Therapy for Glycogen Storage Diseases.Preclinical dose-finding study with a liver-tropic, recombinant AAV-2/8 vector in the mouse model of galactosialidosis.Toward reducing immunogenicity of enzyme replacement therapy: altering the specificity of human β-glucuronidase to compensate for α-iduronidase deficiency.Immunodominant liver-specific expression suppresses transgene-directed immune responses in murine pompe diseaseOral delivery of Acid Alpha Glucosidase epitopes expressed in plant chloroplasts suppresses antibody formation in treatment of Pompe mice.Synergistic Efficacy from Gene Therapy with Coreceptor Blockade and a β2-Agonist in Murine Pompe Disease.Neutralizing antibodies to therapeutic enzymes: considerations for testing, prevention and treatmentAdjunctive β2-agonists reverse neuromuscular involvement in murine Pompe disease.Copackaged AAV9 Vectors Promote Simultaneous Immune Tolerance and Phenotypic Correction of Pompe Disease.Deficiency in MyD88 Signaling Results in Decreased Antibody Responses to an Adeno-Associated Virus Vector in Murine Pompe Disease.Pre-existing Antibody: Biotherapeutic Modality-Based Review.Salmeterol enhances the cardiac response to gene therapy in Pompe disease.Antibody formation and mannose-6-phosphate receptor expression impact the efficacy of muscle-specific transgene expression in murine Pompe disease.Cross-reactive immunologic material status affects treatment outcomes in Pompe disease infants.Immunity to adeno-associated virus vectors in animals and humans: a continued challenge.Potential of AAV vectors in the treatment of metabolic disease.Therapeutic approaches in glycogen storage disease type II/Pompe Disease.A Modified Enzymatic Method for Measurement of Glycogen Content in Glycogen Storage Disease Type IV.Non-depleting anti-CD4 monoclonal antibody induces immune tolerance to ERT in a murine model of Pompe disease.Low-Dose Liver-Targeted Gene Therapy for Pompe Disease Enhances Therapeutic Efficacy of ERT via Immune Tolerance Induction.
P2860
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P2860
Enhanced response to enzyme replacement therapy in Pompe disease after the induction of immune tolerance.
description
2007 nî lūn-bûn
@nan
2007年の論文
@ja
2007年学术文章
@wuu
2007年学术文章
@zh-cn
2007年学术文章
@zh-hans
2007年学术文章
@zh-my
2007年学术文章
@zh-sg
2007年學術文章
@yue
2007年學術文章
@zh
2007年學術文章
@zh-hant
name
Enhanced response to enzyme re ...... induction of immune tolerance.
@ast
Enhanced response to enzyme re ...... induction of immune tolerance.
@en
type
label
Enhanced response to enzyme re ...... induction of immune tolerance.
@ast
Enhanced response to enzyme re ...... induction of immune tolerance.
@en
prefLabel
Enhanced response to enzyme re ...... induction of immune tolerance.
@ast
Enhanced response to enzyme re ...... induction of immune tolerance.
@en
P2093
P2860
P356
P1476
Enhanced response to enzyme re ...... induction of immune tolerance.
@en
P2093
Andrew Bird
Baodong Sun
Dwight D Koeberl
Priya S Kishnani
Sarah P Young
P2860
P304
P356
10.1086/522236
P407
P577
2007-09-21T00:00:00Z