Concepedia

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, Robert J. Davidson, Catherine B. Zander, X. Long Zheng, Valder R. Arruda, Rodney M. Camire, Denise E. Sabatino

Journal of Thrombosis and Haemostasis · 2016 · 48 citations · 38 references

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References

38

Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response

Catherine S. Manno, Glenn F. Pierce, Valder R. Arruda et al. · Nature Medicine · 2006 · 2.1K citations

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Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B

Amit Nathwani, Ulreke M. Reiss, Edward G. D. Tuddenham et al. · New England Journal of Medicine · 2014 · 1.3K citations · Full text

, , +17

Arg-X-Lys/Arg-Arg motif as a signal for precursor cleavage catalyzed by furin within the constitutive secretory pathway

Masahiro Hosaka, Masami Nagahama, W.S. Kim et al. · Journal of Biological Chemistry · 1991 · 598 citations · Full text

, , +17

Adeno-associated virus vectors can be efficiently produced without helper virus

Takashi Matsushita, SS Elliger, CA Elliger et al. · Gene Therapy · 1998 · 495 citations

, , +11

Stabilization of Factor VIII in Plasma by the von Willebrand Factor

Harvey J. Weiss, Ira I. Sussman, Leon W. Hoyer · Journal of Clinical Investigation · 1977 · 422 citations · Full text

, , +17