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Will gene therapy trump factor treatment in hemophilia?

Will gene therapy trump factor treatment in hemophilia?

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Will gene therapy trump factor treatment in hemophilia?

http://www.wikidata.org/entity/Q38078426

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Will gene therapy trump factor treatment in hemophilia?

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Will gene therapy trump factor treatment in hemophilia?

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Will gene therapy trump factor treatment in hemophilia?

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Expert Review of Hematology

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Will gene therapy trump factor treatment in hemophilia?

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Louis Aledort

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Savita Rangarajan

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P2860

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

Rational design of a fully active, long-acting PEGylated factor VIII for hemophilia A treatment.

Intraarticular factor IX protein or gene replacement protects against development of hemophilic synovitis in the absence of circulating factor IX.

Eradication of neutralizing antibodies to factor VIII in canine hemophilia A after liver gene therapy.

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

Coagulation factor concentrates: past, present, and future.

X-linked thrombophilia with a mutant factor IX (factor IX Padua).

Adenovirus-associated virus vector-mediated gene transfer in hemophilia B

Recombinant factor IX-Fc fusion protein (rFIXFc) demonstrates safety and prolonged activity in a phase 1/2a study in hemophilia B patients.

Regional intravascular delivery of AAV-2-F.IX to skeletal muscle achieves long-term correction of hemophilia B in a large animal model

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43-48

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scholarly article

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10.1586/EHM.12.70

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1

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6

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2013-02-01T00:00:00Z

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23373779

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