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Article

Risk and Prevention of Anti-factor IX Formation in AAV-Mediated Gene Transfer in the Context of a Large Deletion of F9

1 Sept 2001

Abstract excerpt

The safety of several gene therapy approaches for treatment of the severe, X-linked bleeding disorder hemophilia is currently being evaluated in early phase clinical trials. One strategy seeks to correct deficiency of functional coagulation factor IX (hemophilia B) by intramuscular (IM) administration of an adeno-associated viral (AAV) vector. A potentially serious complication of any treatment for hemophilia is...

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Identifiers

PMID: 11545610
DOI: 10.1006/mthe.2001.0441

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Risk and Prevention of Anti-factor IX Formation in AAV-Mediated Gene Transfer in the Context of a Large Deletion of F9PMID 11545610Read ↗

Effective gene therapy for haemophilic mice with pathogenic factor IX antibodiesSemantic proximity Read ↗

Muscle-Directed Gene Transfer and Transient Immune Suppression Result in Sustained Partial Correction of Canine Hemophilia B Caused by a Null MutationSemantic proximity Read ↗

Impact of the underlying mutation and the route of vector administration on immune responses to factor IX in gene therapy for hemophilia B.Semantic proximity Read ↗

Prevention and Reversal of Antibody Responses Against Factor IX in Gene Therapy for Hemophilia BSemantic proximity Read ↗

Characterization of a genetically engineered mouse model of hemophilia A with complete deletion of the F8 gene.Semantic proximity Read ↗

Role of the vector genome and underlying factor IX mutation in immune responses to AAV gene therapy for hemophilia BSemantic proximity Read ↗

Creation of a mouse expressing defective human factor IX.Semantic proximity Read ↗

Factor IX variants improve gene therapy efficacy for hemophilia BSemantic proximity Read ↗

Role of Vector in Activation of T Cell Subsets in Immune Responses against the Secreted Transgene Product Factor IXSemantic proximity Read ↗

Engineered factor IX variants bypass FVIII and correct hemophilia A phenotype in mice.Semantic proximity Read ↗

Permanent phenotypic correction of hemophilia B in immunocompetent mice by prenatal gene therapy.Semantic proximity Read ↗

Novel hemophilia B mouse models exhibiting a range of mutations in the Factor IX gene.Semantic proximity Read ↗

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