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Directed Engineering of a High-expression Chimeric Transgene as a Strategy for Gene Therapy of Hemophilia A

Christopher B. Doering, Gabriela Denning, Kerry L. Dooriss, Bagirath Gangadharan, J M Johnston, Keith Kerstann, David McCarty, H. Trent Spencer

Molecular Therapy · 2009 · 78 citations · 29 references

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References

29

Characterization of the human factor VIII gene

Jane Gitschier, William I. Wood, Therese M. Goralka et al. · Nature · 1984 · 985 citations

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Bioengineering of coagulation factor VIII for improved secretion

Hongzhi Miao, Nongnuch Sirachainan, L. E. Palmer et al. · Blood · 2004 · 209 citations

Efficient gene transfer into rhesus repopulating hematopoietic stem cells using a simian immunodeficiency virus–based lentiviral vector system

Hideki Hanawa, Peiman Hematti, Keyvan Keyvanfar et al. · Blood · 2004 · 160 citations · Full text

Therapeutic levels of human factor VIII and IX using HIV-1–based lentiviral vectors in mouse liver

Frank Park, Kazuo Ohashi, Mark A. Kay · Blood · 2000 · 151 citations

Lentivirus‐mediated platelet‐derived factor VIII gene therapy in murine haemophilia A

Qizhen Shi, David A. Wilcox, Scot A. Fahs et al. · Journal of Thrombosis and Haemostasis · 2007 · 133 citations · Full text

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