Gene transfer for hemophilia: can therapeutic efficacy in large animals be safely translated to patients?
Author:
Publisher
Wiley
Subject
Hematology
Link
http://onlinelibrary.wiley.com/wol1/doi/10.1111/j.1538-7836.2005.01460.x/fullpdf
Reference86 articles.
1. Therapeutic factor VIII levels and negligible toxicity in mouse and dog models of hemophilia A following gene therapy with high-capacity adenoviral vectors
2. Neonatal gene transfer leads to widespread correction of pathology in a murine model of lysosomal storage disease
3. Systemic delivery of genes to striated muscles using adeno-associated viral vectors
4. Stable gene transfer and expression of human blood coagulation factor IX after intramuscular injection of recombinant adeno-associated virus
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2. Gene therapy – are we ready now?;Haemophilia;2022-05
3. Treating Hemophilia by Gene Therapy;Translating Regenerative Medicine to the Clinic;2016
4. Translational Data from Adeno-Associated Virus-Mediated Gene Therapy of Hemophilia B in Dogs;Human Gene Therapy Clinical Development;2015-03
5. Translational Data from AAV-Mediated Gene Therapy of Hemophilia B in Dogs;Human Gene Therapy Clinical Development;2014-12-30
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