Muscle-Directed Gene Transfer and Transient Immune Suppression Result in Sustained Partial Correction of Canine Hemophilia B Caused by a Null Mutation
Open Access
- 1 September 2001
- journal article
- Published by Elsevier in Molecular Therapy
- Vol. 4 (3) , 192-200
- https://doi.org/10.1006/mthe.2001.0442
Abstract
No abstract availableKeywords
This publication has 24 references indexed in Scilit:
- Systemic delivery of an adenoviral vector encoding canine factor VIII results in short-term phenotypic correction, inhibitor development, and biphasic liver toxicity in hemophilia A dogsBlood, 2001
- Posttranslational modifications of recombinant myotube-synthesized human factor IXBlood, 2001
- Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vectorNature Genetics, 2000
- Sustained Expression of Therapeutic Level of Factor IX in Hemophilia B Dogs by AAV-Mediated Gene Therapy in LiverMolecular Therapy, 2000
- Increased apoptosis of Huntington disease lymphoblasts associated with repeat length-dependent mitochondrial depolarizationNature Medicine, 1999
- Persistent expression of canine factor IX in hemophilia B caninesGene Therapy, 1999
- Gene therapy for the hemophiliasProceedings of the National Academy of Sciences, 1999
- Long-term correction of canine hemophilia B by gene transfer of blood coagulation factor IX mediated by adeno-associated viral vectorNature Medicine, 1999
- Stable gene transfer and expression of human blood coagulation factor IX after intramuscular injection of recombinant adeno-associated virusProceedings of the National Academy of Sciences, 1997
- Factor IX:Molecular structure, epitopes, and mutations associated with inhibitor formationPublished by Springer Nature ,1995