Gene therapy for hemophilia: an imperative to succeed
Open Access
- 9 June 2005
- journal article
- review article
- Published by Elsevier in Journal of Thrombosis and Haemostasis
- Vol. 3 (6) , 1195-1205
- https://doi.org/10.1111/j.1538-7836.2005.01401.x
Abstract
No abstract availableThis publication has 116 references indexed in Scilit:
- Factor IX variants improve gene therapy efficacy for hemophilia BBlood, 2005
- Side Effects Sideline Hemophilia TrialScience, 2004
- Sustained transgene expression by human cord blood derived CD34+ cells transduced with simian immunodeficiency virus agmTYO1‐based vectors carrying the human coagulation factor VIII gene in NOD/SCID miceThe Journal of Gene Medicine, 2004
- Induction of megakaryocytes to synthesize and store a releasable pool of human factor VIIIJournal of Thrombosis and Haemostasis, 2003
- Preclinical in vivo evaluation of pseudotyped adeno-associated virus vectors for liver gene therapyBlood, 2003
- Recombinant factor VIII expression in hematopoietic cells following lentiviral transductionGene Therapy, 2003
- Bone marrow stromal cells as a genetic platformfor systemic delivery of therapeutic proteins in vivo: human factor IX modelThe Journal of Gene Medicine, 2002
- Site-specific genomic integration produces therapeutic Factor IX levels in miceNature Biotechnology, 2002
- Expression of Biologically Active Human Factor IX in Human Hematopoietic Cells after Retroviral Vector-Mediated Gene TransductionHuman Gene Therapy, 1995
- Liver Transplantation in a HemophiliacNew England Journal of Medicine, 1985