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

2014; Massachusetts Medical Society; Volume: 371; Issue: 21 Linguagem: Inglês

10.1056/nejmoa1407309

1533-4406

Amit Nathwani, Ulreke M. Reiss, Edward G. D. Tuddenham, Cecilia Rosales, Pratima Chowdary, Jenny McIntosh, Marco Della Peruta, Elsa Lhériteau, Nishal Patel, Deepak B. Thimiri Govinda 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 A. Allay, Mark A. Kay, Catherine Y. Ng, Junfang Zhou, Maria Cancio, Christopher L. Morton, John T. Gray, Deo Kumar Srivastava, Arthur W. Nienhuis, Andrew M. Davidoff,

Viral Infectious Diseases and Gene Expression in Insects

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.