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24. Genetic engineering for haemophilia A. Gan SU; Kon OL; Calne RY Expert Opin Biol Ther; 2006 Oct; 6(10):1023-30. PubMed ID: 16989584 [TBL] [Abstract][Full Text] [Related]
25. Employing a gain-of-function factor IX variant R338L to advance the efficacy and safety of hemophilia B human gene therapy: preclinical evaluation supporting an ongoing adeno-associated virus clinical trial. Monahan PE; Sun J; Gui T; Hu G; Hannah WB; Wichlan DG; Wu Z; Grieger JC; Li C; Suwanmanee T; Stafford DW; Booth CJ; Samulski JJ; Kafri T; McPhee SW; Samulski RJ Hum Gene Ther; 2015 Feb; 26(2):69-81. PubMed ID: 25419787 [TBL] [Abstract][Full Text] [Related]
26. Gene Therapy for Hemophilia and Duchenne Muscular Dystrophy in China. Liu X; Liu M; Wu L; Liang D Hum Gene Ther; 2018 Feb; 29(2):146-150. PubMed ID: 29366352 [TBL] [Abstract][Full Text] [Related]
27. Gene therapy trials in the UK: is haemophilia a suitable 'model'? Uk Haemophilia Centre Doctors' Organisation Working Party Clin Med (Lond); 2004; 4(1):54-6. PubMed ID: 14998268 [TBL] [Abstract][Full Text] [Related]
28. Establishing the appropriate primary endpoint in haemophilia gene therapy pivotal studies. Pierce GF; Ragni MV; van den Berg HM; Weill A; O'Mahony B; Skinner MW; Pipe SW Haemophilia; 2017 Sep; 23(5):643-644. PubMed ID: 28833947 [No Abstract] [Full Text] [Related]
31. The gene therapy journey for hemophilia: are we there yet? High KA Hematology Am Soc Hematol Educ Program; 2012; 2012():375-81. PubMed ID: 23233607 [TBL] [Abstract][Full Text] [Related]
32. Gene transfer for hemophilia: can therapeutic efficacy in large animals be safely translated to patients? High K J Thromb Haemost; 2005 Aug; 3(8):1682-91. PubMed ID: 16102034 [TBL] [Abstract][Full Text] [Related]
33. Problems and prospects in gene therapy for hemophilia. Herzog RW; High KA Curr Opin Hematol; 1998 Sep; 5(5):321-6. PubMed ID: 9776210 [TBL] [Abstract][Full Text] [Related]
34. State of the art: gene therapy of haemophilia. Spencer HT; Riley BE; Doering CB Haemophilia; 2016 Jul; 22 Suppl 5():66-71. PubMed ID: 27405679 [TBL] [Abstract][Full Text] [Related]
35. Preclinical and clinical gene therapy for haemophilia. Chuah MK; Collen D; Vandendriessche T Haemophilia; 2004 Oct; 10 Suppl 4():119-25. PubMed ID: 15479384 [TBL] [Abstract][Full Text] [Related]
36. [Development of gene therapy for hemophilia: current states and future perspectives]. Ohmori T Rinsho Ketsueki; 2018; 59(10):2238-2246. PubMed ID: 30305531 [TBL] [Abstract][Full Text] [Related]