Page 191 - 2021_03-Haematologica-web
P. 191
Porcine model of hemophilia B
14(6):1216-1225.
30.Heit JA, Thorland EC, Ketterling RP, et al.
Germline mutations in Peruvian patients with hemophilia B: pattern of mutation in AmerIndians is similar to the putative endogenous germline pattern. Hum Mutat. 1998;11(5):372-376.
31.Ludwig M, Grimm T, Brackmann HH, Olek K. Parental origin of factor IX gene muta- tions, and their distribution in the gene. Am J Hum Genet. 1992;50(1):164-173.
32.Poon MC, Anand S, Fraser BM, Hoar DI, Sinclair GD. Hemophilia B carrier determi- nation based on family-specific mutation detection by DNA single-strand conforma- tion analysis. J Lab Clin Med. 1993;122 (1):55-63.
33. Wulff K, Bykowska K, Lopaciuk S, Herrmann FH. Molecular analysis of hemo- philia B in Poland: 12 novel mutations of the factor IX gene. Acta Biochim Pol. 1999; 46(3):721-726.
34.Guan Y, Ma Y, Li Q, et al. CRISPR/Cas9- mediated somatic correction of a novel coagulator factor IX gene mutation amelio- rates hemophilia in mouse. EMBO Mol Med. 2016;8(5):477-488.
35.High KA. Gene therapy for hemophilia: the clot thickens. Hum Gene Ther. 2014; 25(11):915-922.
36. Nathwani AC, Davidoff AM, Tuddenham EGD. Advances in gene therapy for hemo- philia. Hum Gene Ther. 2017;28(11):1004- 1012.
37. Wang L, Yang Y, Breton CA, et al. CRISPR/Cas9-mediated in vivo gene target- ing corrects hemostasis in newborn and adult factor IX-knockout mice. Blood. 2019;133(26):2745-2752.
38.VandenDriessche T, Collen D, Chuah MK. Gene therapy for the hemophilias. J Thromb Haemost. 2003;1(7):1550-1558.
39.Xu X, Wan T, Xin H, et al. Delivery of CRISPR/Cas9 for therapeutic genome edit- ing. J Gene Med. 2019;21(7):e3107.
40.Wagner DL, Amini L, Wendering DJ, et al. High prevalence of Streptococcus pyogenes Cas9-reactive T cells within the adult human population. Nat Med. 2019;25(2): 242-248.
haematologica | 2021; 106(3)
837