Gene Therapy for Hemophilia: A Step Closer to Reality
Open Access
- 31 March 2000
- journal article
- editorial
- Published by Elsevier in Molecular Therapy
- Vol. 1 (3) , 207-208
- https://doi.org/10.1006/mthe.2000.0048
Abstract
No abstract availableKeywords
This publication has 9 references indexed in Scilit:
- 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
- Sustained correction of bleeding disorder in hemophilia B mice by gene therapyProceedings 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
- Adeno-Associated Viral Vector-Mediated Gene Transfer of Human Blood Coagulation Factor IX Into Mouse LiverBlood, 1998
- Direct intramuscular injection with recombinant AAV vectors results in sustained expression in a dog model of hemophiliaGene Therapy, 1998
- Persistent and therapeutic concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectorsNature Genetics, 1997