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Long-term safety and efficacy of factor IX gene therapy in hemophilia B.
Amit C Nathwani
1 ,
Ulreke M Reiss ,
Edward G D Tuddenham ,
Cecilia Rosales ,
Pratima Chowdary ,
Jenny McIntosh ,
Marco Della Peruta ,
Elsa Lheriteau ,
Nishal Patel ,
Deepak Raj ,
Anne Riddell ,
Jun Pie ,
Savita Rangarajan ,
David Bevan ,
Michael Recht ,
Yu-Min Shen ,
Kathleen G Halka ,
Etiena Basner-Tschakarjan ,
Federico Mingozzi ,
Katherine A High ,
James Allay ,
Mark A Kay ,
Catherine Y C Ng ,
Junfang Zhou ,
Maria Cancio ,
Christopher L Morton ,
John T Gray ,
Deokumar Srivastava ,
Arthur W Nienhuis ,
Andrew M Davidoff
Nov 20 2014
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Abstract
In patients with severe hemophilia B, gene therapy that is mediated by a novel self-complementary adeno-associated virus serotype 8 (AAV8) vector has been shown to raise factor IX levels for periods of up to 16 months. We wanted to determine the durability of transgene expression, the vector dose-response relationship, and the level of persistent or late toxicity.