Joshua I. Siner

AAV liver expression of FIX-Padua prevents and eradicates FIX inhibitor without increasing thrombogenicity in hemophilia B dogs and mice

Julie M. Crudele, Jonathan D. Finn, Joshua I. Siner et al. · Blood · 2015 · 154 citations · Full text

Medicine, Hematology, Pathobiology +6

Minimal modification in the factor VIII B-domain sequence ameliorates the murine hemophilia A phenotype

Joshua I. Siner, Nicholas P. Iacobelli, Denise E. Sabatino et al. · Blood · 2013 · 83 citations · Full text

Medicine, Hematology, Molecular Biology +11

Novel factor VIII variants with a modified furin cleavage site improve the efficacy of gene therapy for hemophilia A

Giang N. Nguyen, Lindsey A. George, Joshua I. Siner et al. · Journal of Thrombosis and Haemostasis · 2016 · 48 citations · Full text

Medicine, Hematology, Molecular Biology +13

Circumventing furin enhances factor VIII biological activity and ameliorates bleeding phenotypes in hemophilia models

Joshua I. Siner, Benjamin J. Samelson‐Jones, Julie M. Crudele et al. · JCI Insight · 2016 · 36 citations · Full text

Medicine, Hematology, Molecular Biology +17

Safety of Liver Gene Transfer Following Peripheral Intravascular Delivery of Adeno-Associated Virus (AAV)-5 and AAV-6 in a Large Animal Model

Patricia Favaro, Jonathan D. Finn, Joshua I. Siner et al. · Human Gene Therapy · 2010 · 24 citations · Full text

Pharmacy, Molecular Medicine, Drug Delivery +12