Our journey to successful gene therapy for hemophilia B.
1 Katharine Dormandy Haemophilia Centre and Thrombosis Unit , Royal Free NHS Foundation Trust, London NW3 2QG, United Kingdom .
Amit C
AC
Nathwani
Arthur W
AW
Nienhuis
Andrew M
AM
Davidoff
D000818:Animals; D000229:Dependovirus / Q000235:genetics; D005164:Factor IX / Q000235:genetics*; D015316:Genetic Therapy; D002836:Hemophilia B / Q000235:genetics / Q000628:therapy*; D006801:Humans; D014161:Transduction, Genetic
D005164:Factor IX