参考文献
[1]J Mahlangu,M Cerguiera,A Srivastava.Emerging therapies for Haemophilia-Global perspective[J].Haemophilia,2018,24(Suppl 6):15-21.
[2]G F Pierce,A Iorio Past,present and future of Haemophilia gene therapy:From vectors and transgenes to known and unknown outcomes [J].Haemophilia,2018, 24(Suppl.4):60-67.
[3]Paulk N K,Pekrun K,Zhu E,et al.Bioengineered AAV capsids with combined highhuman liver transduction in vivo and unique humoral seroreactivity [J].Molecular Therapy,2018,26:289-303.
[4]Cantore A,Milani M,Annoni A,et al.Liver-directed gene therapy for hemophilia B with immune stealth lentiviral vectors [J].Blood,2017, 130:605.
[5]Machin N,Ragni M V.Measuring success in hemophilia gene therapy using a factor level & outcomes yardstick [J].Expert Rev Hematol, 2018, 11:83-86.(https://www.daowen.com)
[6]Limperg P F,Terwee C B,Young N L,et al.Health-related quality of life questionnaires in individuals with haemophilia:a systematic review of their measurement properties[J].Haemophilia,2017, 23:1-14.
[7]Miesbach W,Meijer K,Coppens M,et al.Gene therapy with adeno-associated virus vector5-human factor Ⅸ in adults with hemophilia B[J].Blood,2018, 131:1022-1031.
[8]Hinderer C,Katz N,Buza E L,et al.Severe toxicity in nonhuman primates and pglets following high-dose intravenous administration of an AAV vector expressing human SMN [J].Hum Gene Ther,2018, 29:285-298.
[9]Flotte TR.Severe toxicity in non-human pimates and piglest with systemic high-dose administration of AAV9-like vectors:putting patients first [J].Hum Gene Ther,2018, 29:283-284.