Long-term safety and efficacy of factor IX gene therapy in hemophilia B.
Nathwani AC1, Reiss UM, Tuddenham EG, Rosales C, Chowdary P, McIntosh J, Della Peruta M, Lheriteau E, Patel N, Raj D, Riddell A, Pie J, Rangarajan S, Bevan D, Recht M, Shen YM, Halka KG, Basner-Tschakarjan E, Mingozzi F, High KA, Allay J, Kay MA, Ng CY, Zhou J, Cancio M, Morton CL, Gray JT, Srivastava D, Nienhuis AW, Davidoff AM.
N Engl J Med. 2014 Nov 20 ;371(21):1994-2004. doi : 10.1056/NEJMoa1407309.