[HTML][HTML] Efficacy and safety of adeno‐associated viral vectors based on serotype 8 and 9 vs. lentiviral vectors for hemophilia B gene therapy

T VandenDriessche, L Thorrez… - Journal of Thrombosis …, 2007 - Elsevier
Background: Adeno‐associated viral (AAV) and lentiviral vectors are promising vectors for
gene therapy for hemophilia because they are devoid of viral genes and have the potential
for long‐term gene expression. Objectives: To compare the performance of different AAV
serotypes (AAV8 and AAV9) vs. lentiviral vectors expressing factor (F) IX. Methods and
results: AAV‐based and lentiviral vectors were generated that express FIX from the same
hepatocyte‐specific expression cassette. AAV9 transduced the liver as efficiently as AAV8 …