메뉴 건너뛰기




Volumn 41, Issue 4, 2003, Pages 445-451

Haemophilia B: From molecular diagnosis to gene therapy

Author keywords

Denaturing HPLC (D HPLC); Factor 9 (F9); Factor IX (FIX); Mutations

Indexed keywords

ADENOVIRUS VECTOR; BLOOD CLOTTING FACTOR 9; BLOOD CLOTTING FACTOR 9 CONCENTRATE; DNA; RETROVIRUS VECTOR;

EID: 0038735501     PISSN: 14346621     EISSN: None     Source Type: Journal    
DOI: 10.1515/CCLM.2003.067     Document Type: Review
Times cited : (19)

References (55)
  • 1
    • 0014499005 scopus 로고
    • Hemophilia in the Talmud and Rabbinic writings
    • Rosner F. Hemophilia in the Talmud and Rabbinic writings. Ann Int Med 1969; 70:833-7.
    • (1969) Ann. Int. Med. , vol.70 , pp. 833-837
    • Rosner, F.1
  • 2
    • 0017138012 scopus 로고
    • The history of haemophilia
    • Ingram GIC. The history of haemophilia. J Clin Pathol 1976; 29:469-79
    • (1976) J. Clin. Pathol. , vol.29 , pp. 469-479
    • Ingram, G.I.C.1
  • 3
    • 0036218048 scopus 로고    scopus 로고
    • Haemophilia A and haemophilia B: Molecular insights
    • Bowen DJ. Haemophilia A and haemophilia B: molecular insights. Mol Pathol 2002; 55:127-44.
    • (2002) Mol. Pathol. , vol.55 , pp. 127-144
    • Bowen, D.J.1
  • 4
    • 0036135026 scopus 로고    scopus 로고
    • Mutations in the factor IX gene (F9) during the past 150 years have relative rates similar to ancient mutations
    • Feng J, Drost JB, Scaringe WA, Liu Q, Sommer SS. Mutations in the factor IX gene (F9) during the past 150 years have relative rates similar to ancient mutations. Hum Mutat 2002; 19:49-57.
    • (2002) Hum. Mutat. , vol.19 , pp. 49-57
    • Feng, J.1    Drost, J.B.2    Scaringe, W.A.3    Liu, Q.4    Sommer, S.S.5
  • 5
    • 0025948892 scopus 로고
    • Missense mutations and evolutionary conservation of amino acids: Evidence that many of the amino acids in factor IX function as "spacer" elements
    • Bottema CD, Ketterling RP, Li S, Yoon HS, Phillips JA III, Sommer SS. Missense mutations and evolutionary conservation of amino acids: evidence that many of the amino acids in factor IX function as "spacer" elements. Am J Hum Genet 1991; 49:820-38.
    • (1991) Am. J. Hum. Genet. , vol.49 , pp. 820-838
    • Bottema, C.D.1    Ketterling, R.P.2    Li, S.3    Yoon, H.S.4    Phillips J.A. III5    Sommer, S.S.6
  • 6
    • 18744422146 scopus 로고    scopus 로고
    • Germline origins in the human F9 gene: Frequent G:C→A:T mosaicism and increased mutations with advanced maternal age
    • Ketterling RP, Vielhaber E, Li X, Drost J, Schaid DJ, Kasper CK, et al. Germline origins in the human F9 gene: frequent G:C→A:T mosaicism and increased mutations with advanced maternal age. Hum Genet 1999; 105:629-40.
    • (1999) Hum. Genet. , vol.105 , pp. 629-640
    • Ketterling, R.P.1    Vielhaber, E.2    Li, X.3    Drost, J.4    Schaid, D.J.5    Kasper, C.K.6
  • 7
    • 0033358737 scopus 로고    scopus 로고
    • Mutation rates in humans. II. Sporadic mutation-specific rates and rate of detrimental human mutations inferred from hemophilia B
    • Giannelli F, Anagnostopoulos T, Green PM. Mutation rates in humans. II. Sporadic mutation-specific rates and rate of detrimental human mutations inferred from hemophilia B. Am J Hum Genet 1999; 65:1580-7.
    • (1999) Am. J. Hum. Genet. , vol.65 , pp. 1580-1587
    • Giannelli, F.1    Anagnostopoulos, T.2    Green, P.M.3
  • 8
    • 0035501319 scopus 로고    scopus 로고
    • Human germline mutation in the factor IX gene
    • Sommer SS, Scaringe WA, Hill KA. Human germline mutation in the factor IX gene. Mut Res 2001; 487:1-17.
    • (2001) Mut. Res. , vol.487 , pp. 1-17
    • Sommer, S.S.1    Scaringe, W.A.2    Hill, K.A.3
  • 9
    • 0036305116 scopus 로고    scopus 로고
    • Model of a ternary complex between-activated factor VII, tissue factor and factor IX
    • Chen SW, Pellequer JL, Schved JF, Giansily-Blaizot M. Model of a ternary complex between-activated factor VII, tissue factor and factor IX. Thromb Haemost 2002; 88: 74-82.
    • (2002) Thromb. Haemost. , vol.88 , pp. 74-82
    • Chen, S.W.1    Pellequer, J.L.2    Schved, J.F.3    Giansily-Blaizot, M.4
  • 10
    • 0033932856 scopus 로고    scopus 로고
    • Experience of a single Italian center in genetic counseling for hemophilia: From linkage analysis to molecular diagnosis
    • Tagariello G, Belvini D, Salviato R, Are A, De Biasi E, Goodeve A, et al. Experience of a single Italian center in genetic counseling for hemophilia: from linkage analysis to molecular diagnosis. Haematologica 2000; 85:525-9.
    • (2000) Haematologica , vol.85 , pp. 525-529
    • Tagariello, G.1    Belvini, D.2    Salviato, R.3    Are, A.4    De Biasi, E.5    Goodeve, A.6
  • 11
    • 0021340565 scopus 로고
    • Characterisation and use of an intragenic polymorphic marker for detection of carriers of haemophilia B (factor IX deficiency)
    • Giannelli F, Anson DS, Choo KH, Rees DJ, Winship PR, Ferrari N, et al. Characterisation and use of an intragenic polymorphic marker for detection of carriers of haemophilia B (factor IX deficiency). Lancet 1984; 1:239-41.
    • (1984) Lancet , vol.1 , pp. 239-241
    • Giannelli, F.1    Anson, D.S.2    Choo, K.H.3    Rees, D.J.4    Winship, P.R.5    Ferrari, N.6
  • 12
    • 0021322792 scopus 로고
    • Carrier detection of hemophilia B by using a restriction site polymorphism associated with the coagulation factor IX gene
    • Grunebaum L, Cazenave JP, Camerino G, Kloepfer C, Mandel JL, Tolstoshev P, et al. Carrier detection of hemophilia B by using a restriction site polymorphism associated with the coagulation factor IX gene. J Clin Invest 1984; 73: 1491-5.
    • (1984) J. Clin. Invest. , vol.73 , pp. 1491-1495
    • Grunebaum, L.1    Cazenave, J.P.2    Camerino, G.3    Kloepfer, C.4    Mandel, J.L.5    Tolstoshev, P.6
  • 13
    • 0025054983 scopus 로고
    • Diagnosis of haemophilia B using the polymerase chain reaction
    • Reiss J, Neufeldt U, Wieland K, Zoll B. Diagnosis of haemophilia B using the polymerase chain reaction. Blut 1990; 60:31-6.
    • (1990) Blut , vol.60 , pp. 31-36
    • Reiss, J.1    Neufeldt, U.2    Wieland, K.3    Zoll, B.4
  • 14
    • 0025040423 scopus 로고
    • Hemophilia B: Diagnostic value of RFLP analysis in 19 of the 20 known Finnish families
    • Lehesjoki AE, Rasi V, de la Chapelle A. Hemophilia B: diagnostic value of RFLP analysis in 19 of the 20 known Finnish families. Clin Genet 1990; 38:187-97.
    • (1990) Clin. Genet. , vol.38 , pp. 187-197
    • Lehesjoki, A.E.1    Rasi, V.2    de la Chapelle, A.3
  • 15
    • 0027756995 scopus 로고
    • Carrier detection and prenatal diagnosis of hemophilia B with more advanced techniques
    • Caprino D, Acquila M, Mori PG. Carrier detection and prenatal diagnosis of hemophilia B with more advanced techniques. Ann Hematol 1993; 67:289-93.
    • (1993) Ann. Hematol. , vol.67 , pp. 289-293
    • Caprino, D.1    Acquila, M.2    Mori, P.G.3
  • 16
    • 0028289158 scopus 로고
    • A comparison of the allelic frequencies of ten DNA polymorphisms associated with factor VIII and factor IX genes in Thai and Western European populations
    • Goodeve AC, Chuansumrit A, Sasanakul W, Isarangkura P, Preston FE, Peake IR. A comparison of the allelic frequencies of ten DNA polymorphisms associated with factor VIII and factor IX genes in Thai and Western European populations. Blood Coagul Fibrinolysis 1994; 5:29-35.
    • (1994) Blood Coagul. Fibrinolysis , vol.5 , pp. 29-35
    • Goodeve, A.C.1    Chuansumrit, A.2    Sasanakul, W.3    Isarangkura, P.4    Preston, F.E.5    Peake, I.R.6
  • 17
    • 0035293431 scopus 로고    scopus 로고
    • Carrier detection and prenatal diagnosis in families with haemophilia
    • Shetty S, Ghosh K, Bhide A, Mohanty D. Carrier detection and prenatal diagnosis in families with haemophilia. Natl Med J India 2001; 14:81-3.
    • (2001) Natl. Med. J. India , vol.14 , pp. 81-83
    • Shetty, S.1    Ghosh, K.2    Bhide, A.3    Mohanty, D.4
  • 18
    • 0025205085 scopus 로고
    • Common intragenic and extragenic polymorphisms of blood coagulation factors VIII and IX are different in Chinese and Caucasian populations
    • De la Salle C, Wu Q, Baas MJ, Hanauer A, Ruan C, Cazeneuve JP. Common intragenic and extragenic polymorphisms of blood coagulation factors VIII and IX are different in Chinese and Caucasian populations. Clin Genet 1990; 38:434-40.
    • (1990) Clin. Genet. , vol.38 , pp. 434-440
    • De la Salle, C.1    Wu, Q.2    Baas, M.J.3    Hanauer, A.4    Ruan, C.5    Cazeneuve, J.P.6
  • 19
    • 0026063167 scopus 로고
    • Molecular defects in haemophilia B: Detection by direct restriction enzyme analysis
    • Chan VV, Yip B, Tong TM, Chan TP, Lau K, Yam I, et al. Molecular defects in haemophilia B: detection by direct restriction enzyme analysis. Br J Haematol 1991; 791:63-9.
    • (1991) Br. J. Haematol. , vol.791 , pp. 63-69
    • Chan, V.V.1    Yip, B.2    Tong, T.M.3    Chan, T.P.4    Lau, K.5    Yam, I.6
  • 20
    • 0026089883 scopus 로고
    • Restriction fragment length polymorphisms associated with the factor VIII and factor IX genes in Polynesians
    • Van-de-Water NS, Ridgway D, Ockelford PA. Restriction fragment length polymorphisms associated with the factor VIII and factor IX genes in Polynesians. J Med Genet 1991; 28:171-6.
    • (1991) J. Med. Genet. , vol.28 , pp. 171-176
    • Van-de-Water, N.S.1    Ridgway, D.2    Ockelford, P.A.3
  • 21
    • 0028074245 scopus 로고
    • Factor IX gene haplotypes in Brazilian blacks and characterization of unusual Dde I alleles
    • Figueiredo MS, Bowen DJ, Silva Junior WA, Zago MA. Factor IX gene haplotypes in Brazilian blacks and characterization of unusual Dde I alleles. Br J Haematol 1994; 87: 789-96.
    • (1994) Br. J. Haematol. , vol.87 , pp. 789-796
    • Figueiredo, M.S.1    Bowen, D.J.2    Silva Junior, W.A.3    Zago, M.A.4
  • 22
    • 0027169164 scopus 로고
    • Detection of a molecular defect in 40 of 44 patients with haemophilia B by PCR and denaturing gradient gel electrophoresis
    • Tartary M, Vidaud D, Piao Y, Costa JM, Bahnak BR, Fressinaud E, et al. Detection of a molecular defect in 40 of 44 patients with haemophilia B by PCR and denaturing gradient gel electrophoresis. Br J Haematol 1993; 84:662-9.
    • (1993) Br. J. Haematol. , vol.84 , pp. 662-669
    • Tartary, M.1    Vidaud, D.2    Piao, Y.3    Costa, J.M.4    Bahnak, B.R.5    Fressinaud, E.6
  • 23
    • 0032749906 scopus 로고    scopus 로고
    • Factor IX gene analysis in 70 unrelated patients with haemophilia B: Description of 13 new mutations
    • Attali O, Vinciguerra C, Trzeciak MC, Durin A, Pernod G, Gay V, et al. Factor IX gene analysis in 70 unrelated patients with haemophilia B: description of 13 new mutations. Thromb Haemost 1999; 82:1437-42.
    • (1999) Thromb. Haemost. , vol.82 , pp. 1437-1442
    • Attali, O.1    Vinciguerra, C.2    Trzeciak, M.C.3    Durin, A.4    Pernod, G.5    Gay, V.6
  • 24
    • 0028037894 scopus 로고
    • Factor IX gene mutations causing haemophilia B: Comparison of SSC screening versus systematic DNA sequencing and diagnostic applications
    • Aguilar-Martinez P, Romey MC, Schved JF, Gris JC, Demaille J, Claustres M. Factor IX gene mutations causing haemophilia B: comparison of SSC screening versus systematic DNA sequencing and diagnostic applications. Hum Genet 1994; 94:287-90.
    • (1994) Hum. Genet. , vol.94 , pp. 287-290
    • Aguilar-Martinez, P.1    Romey, M.C.2    Schved, J.F.3    Gris, J.C.4    Demaille, J.5    Claustres, M.6
  • 25
    • 0027244742 scopus 로고
    • Hemophilia B carrier determination based on family-specific mutation detection by DNA single-strand conformation analysis
    • Poon MC, Anand S, Fraser BM, Hoar DI, Sinclair GD. Hemophilia B carrier determination based on family-specific mutation detection by DNA single-strand conformation analysis. J Lab Clin Med 1993; 122:55-63.
    • (1993) J. Lab. Clin. Med. , vol.122 , pp. 55-63
    • Poon, M.C.1    Anand, S.2    Fraser, B.M.3    Hoar, D.I.4    Sinclair, G.D.5
  • 26
    • 0035464731 scopus 로고    scopus 로고
    • Study on factor IX gene mutation in 74 haemophilia B patients
    • Liu J, Xiang H, Liu L. [Study on factor IX gene mutation in 74 haemophilia B patients]. Zhonghua Xue Ye Xue Za Zhi 2001; 22:467-9.
    • (2001) Zhonghua Xue Ye Xue Za Zhi , vol.22 , pp. 467-469
    • Liu, J.1    Xiang, H.2    Liu, L.3
  • 28
    • 0033631005 scopus 로고    scopus 로고
    • Nine independent F9 mutations in the Mexican hemophilia B population: Nonrandom recurrences of point mutation events in the human germline
    • Jaloma-Cruz AR, Scaringe WA, Drost JB, Roberts S, Li X, Barros-Nunez P, et al. Nine independent F9 mutations in the Mexican hemophilia B population: nonrandom recurrences of point mutation events in the human germline. Hum Mutat 2000; 15:116-7.
    • (2000) Hum. Mutat. , vol.15 , pp. 116-117
    • Jaloma-Cruz, A.R.1    Scaringe, W.A.2    Drost, J.B.3    Roberts, S.4    Li, X.5    Barros-Nunez, P.6
  • 29
    • 0031957550 scopus 로고    scopus 로고
    • Germline mutations in Peruvian patients with hemophilia B: Pattern of mutation in Amerindians is similar to the putative endogenous germline pattern
    • Heit JA, Thorland EC, Ketterling RP, Lind TJ, Daniels TM, Zapata RE, 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; 11:372-6.
    • (1998) Hum. Mutat. , vol.11 , pp. 372-376
    • Heit, J.A.1    Thorland, E.C.2    Ketterling, R.P.3    Lind, T.J.4    Daniels, T.M.5    Zapata, R.E.6
  • 30
    • 0028237968 scopus 로고
    • Six novel and three recurrent mutations in nine Austrian patients with hemophilia B
    • Walter J, Pabinger-Fasching I, Watzke HH. Six novel and three recurrent mutations in nine Austrian patients with hemophilia B. Thromb Haemost 1994; 72:74-7.
    • (1994) Thromb. Haemost. , vol.72 , pp. 74-77
    • Walter, J.1    Pabinger-Fasching, I.2    Watzke, H.H.3
  • 31
    • 0034537118 scopus 로고    scopus 로고
    • Factor IX gene sequencing by a simple and sensitive 15-hour procedure for haemophilia B diagnosis: Identification of two novel mutations
    • Vidal F, Farssac E, Altisent C, Puig L, Gallardo D. Factor IX gene sequencing by a simple and sensitive 15-hour procedure for haemophilia B diagnosis: identification of two novel mutations. Br J Haematol 2000; 111:549-51.
    • (2000) Br. J. Haematol. , vol.111 , pp. 549-551
    • Vidal, F.1    Farssac, E.2    Altisent, C.3    Puig, L.4    Gallardo, D.5
  • 32
    • 0034998368 scopus 로고    scopus 로고
    • A haemophilia A and B molecular genetic diagnostic programme in Hungary: A highly informative and cost-effective strategy
    • Klein I, Andrikovics H, Bors A, Nemes L, Tordai A, Varadi A. A haemophilia A and B molecular genetic diagnostic programme in Hungary: a highly informative and cost-effective strategy. Haemophilia 2001; 7:306-12.
    • (2001) Haemophilia , vol.7 , pp. 306-312
    • Klein, I.1    Andrikovics, H.2    Bors, A.3    Nemes, L.4    Tordai, A.5    Varadi, A.6
  • 33
    • 0035020939 scopus 로고    scopus 로고
    • Complete and rapid scanning of the cystic fibrosis transmembrane conductance regulator (CFTR) gene by denaturing high-performance liquid chromatography (D-HPLC): Major implications for genetic counselling
    • Le Maréchal C, Audrézet MP, Quéré I, Raguénès O, Lagonné S, Férec C. Complete and rapid scanning of the cystic fibrosis transmembrane conductance regulator (CFTR) gene by denaturing high-performance liquid chromatography (D-HPLC): major implications for genetic counselling. Hum Genet 2001; 108:290-8.
    • (2001) Hum. Genet. , vol.108 , pp. 290-298
    • Le Maréchal, C.1    Audrézet, M.P.2    Quéré, I.3    Raguénès, O.4    Lagonné, S.5    Férec, C.6
  • 36
    • 0034999807 scopus 로고    scopus 로고
    • Denaturing high-performance liquid chromatography: A review
    • Xiao W, Oefner PJ. Denaturing high-performance liquid chromatography: a review. Hum Mutat 2001; 17:439-74.
    • (2001) Hum. Mutat. , vol.17 , pp. 439-474
    • Xiao, W.1    Oefner, P.J.2
  • 37
    • 0034078332 scopus 로고    scopus 로고
    • A comparison of fluorescent SSCP and denaturing HPLC for high throughput mutation scanning
    • Ellis LA, Taylor GR. A comparison of fluorescent SSCP and denaturing HPLC for high throughput mutation scanning. Hum Mutat 2000; 15:556-64.
    • (2000) Hum. Mutat. , vol.15 , pp. 556-564
    • Ellis, L.A.1    Taylor, G.R.2
  • 39
    • 0031045680 scopus 로고    scopus 로고
    • HLA genotype of patients with severe haemophilia A due to intron 22 inversion with and without inhibitors of factor VIII
    • Oldenburg J, Picard JK, Schwaab R. HLA genotype of patients with severe haemophilia A due to intron 22 inversion with and without inhibitors of factor VIII. Thromb Haemost 1997; 77:238-42.
    • (1997) Thromb. Haemost. , vol.77 , pp. 238-242
    • Oldenburg, J.1    Picard, J.K.2    Schwaab, R.3
  • 40
    • 0037157787 scopus 로고    scopus 로고
    • Genotype-phenotype correlation in cystic fibrosis: The role of modifier genes
    • Salvatore F, Scudiero O, Castaldo G. Genotype-phenotype correlation in cystic fibrosis: the role of modifier genes. Am J Med Genet 2002; 111:88-95.
    • (2002) Am. J. Med. Genet. , vol.111 , pp. 88-95
    • Salvatore, F.1    Scudiero, O.2    Castaldo, G.3
  • 41
    • 0029658954 scopus 로고    scopus 로고
    • Safety and efficacy of monoclonal antibody purified factor IX concentrate in previously untreated patients with hemophilia B
    • Shapiro AD, Ragni MV, Lusher JM, Culbert S, Koerper MA, Bergman GE, et al. Safety and efficacy of monoclonal antibody purified factor IX concentrate in previously untreated patients with hemophilia B. Thromb Haemost 1996; 75: 30-5.
    • (1996) Thromb. Haemost. , vol.75 , pp. 30-35
    • Shapiro, A.D.1    Ragni, M.V.2    Lusher, J.M.3    Culbert, S.4    Koerper, M.A.5    Bergman, G.E.6
  • 42
    • 0032932329 scopus 로고    scopus 로고
    • Efficacy of a high purity, chemically treated and nanofiltered factor IX concentrate for continous infusion in haemophilia patients undergoing surgery
    • Schulman S, Wallensten R, White B, Smith OP. Efficacy of a high purity, chemically treated and nanofiltered factor IX concentrate for continous infusion in haemophilia patients undergoing surgery. Haemophilia 1999; 5:96-100.
    • (1999) Haemophilia , vol.5 , pp. 96-100
    • Schulman, S.1    Wallensten, R.2    White, B.3    Smith, O.P.4
  • 43
    • 10144234803 scopus 로고    scopus 로고
    • Recombinant human factor IX replacement therapy, prophylaxis, and pharmacokinetics in canine haemophilia B
    • Brinkhous KM, Sigman JL, Read MS, Stewart PF, McCarthy KP, Timony GA, et al. Recombinant human factor IX replacement therapy, prophylaxis, and pharmacokinetics in canine haemophilia B. Blood 1996; 88:2603-10.
    • (1996) Blood , vol.88 , pp. 2603-2610
    • Brinkhous, K.M.1    Sigman, J.L.2    Read, M.S.3    Stewart, P.F.4    McCarthy, K.P.5    Timony, G.A.6
  • 45
    • 0035895058 scopus 로고    scopus 로고
    • Human recombinant factor IX: Safety and efficacy studies in haemophilia B patients previously treated with plasma-derived factor IX concentrates
    • Recombinant Factor IX Study Group
    • Roth DA, Kessler CM, Pasi KJ, Rup B, Courter SG, Tubridy KL. Recombinant Factor IX Study Group. Human recombinant factor IX: safety and efficacy studies in haemophilia B patients previously treated with plasma-derived factor IX concentrates. Blood 2001; 98:3600-6.
    • (2001) Blood , vol.98 , pp. 3600-3606
    • Roth, D.A.1    Kessler, C.M.2    Pasi, K.J.3    Rup, B.4    Courter, S.G.5    Tubridy, K.L.6
  • 47
    • 0035077128 scopus 로고    scopus 로고
    • Intratracheal administration of recombinant human factor IX (BeneFix) achieves therapeutic levels in hemophilia B dogs
    • Russel KE, Read MS, Bellinger DA, Leitermann K, Rup BJ, McCarthy KP, et al. Intratracheal administration of recombinant human factor IX (BeneFix) achieves therapeutic levels in hemophilia B dogs. Thromb Haemost 2001; 85:445-9.
    • (2001) Thromb. Haemost. , vol.85 , pp. 445-449
    • Russel, K.E.1    Read, M.S.2    Bellinger, D.A.3    Leitermann, K.4    Rup, B.J.5    McCarthy, K.P.6
  • 48
    • 0036041476 scopus 로고    scopus 로고
    • Use of a non-depleting anti-CD4 antibody to modulate the immune response to coagulation factors VIII and IX
    • Salooja N, Kemball-Cook G, Tuddenham EG, Dyson J. Use of a non-depleting anti-CD4 antibody to modulate the immune response to coagulation factors VIII and IX. Br J Haematol 2002; 118:839-42.
    • (2002) Br. J. Haematol. , vol.118 , pp. 839-842
    • Salooja, N.1    Kemball-Cook, G.2    Tuddenham, E.G.3    Dyson, J.4
  • 49
    • 0035123581 scopus 로고    scopus 로고
    • Gene therapy: A 2001 perspective
    • High KA. Gene therapy: a 2001 perspective. Haemophilia 2001; 7 Suppl 1:23-7.
    • (2001) Haemophilia , vol.7 , Issue.SUPPL. 1 , pp. 23-27
    • High, K.A.1
  • 50
    • 0035676918 scopus 로고    scopus 로고
    • Gene therapy for haemophilia
    • Pasi KJ. Gene therapy for haemophilia. Br J Haematol 2001; 115:744-57.
    • (2001) Br. J. Haematol. , vol.115 , pp. 744-757
    • Pasi, K.J.1
  • 51
    • 0036264622 scopus 로고    scopus 로고
    • Parvovirus-mediated gene transfer for the haemophilias
    • Walsh CE, Chao H. Parvovirus-mediated gene transfer for the haemophilias. Haemophilia 2002; 8 Suppl 2:60-7.
    • (2002) Haemophilia , vol.8 , Issue.SUPPL. 2 , pp. 60-67
    • Walsh, C.E.1    Chao, H.2
  • 53
    • 0034103009 scopus 로고    scopus 로고
    • Somatic integration and long-term transgene expression in normal and haemophilic mice using a DNA transposon system
    • Yant SR, Meuse L, Chiu W, Ivics Z, Izsvak Z, Kay MA. Somatic integration and long-term transgene expression in normal and haemophilic mice using a DNA transposon system. Nat Genet 2000; 25:35-41.
    • (2000) Nat. Genet. , vol.25 , pp. 35-41
    • Yant, S.R.1    Meuse, L.2    Chiu, W.3    Ivics, Z.4    Izsvak, Z.5    Kay, M.A.6
  • 54
    • 0033222971 scopus 로고    scopus 로고
    • Therapeutic plasma concentrations of human factor IX in mice after gene delivery into the amniotic cavity: A model for the prenatal treatment of haemophilia B
    • Schneider H, Adebakin S, Themis M, Cook T, Douar AM, Pavirani A, et al. Therapeutic plasma concentrations of human factor IX in mice after gene delivery into the amniotic cavity: a model for the prenatal treatment of haemophilia B. J Gene Med 1999; 1:424-32.
    • (1999) J. Gene Med. , vol.1 , pp. 424-432
    • Schneider, H.1    Adebakin, S.2    Themis, M.3    Cook, T.4    Douar, A.M.5    Pavirani, A.6
  • 55
    • 0036371236 scopus 로고    scopus 로고
    • Sustained delivery of therapeutic concentrations of human clotting factor IX: A comparision of adenoviral and AAV vectors administered in utero
    • Schneider H, Muhle C, Douar AM, Waddington S, Jiang QJ, von der Mark K, etal. Sustained delivery of therapeutic concentrations of human clotting factor IX: a comparision of adenoviral and AAV vectors administered in utero. J Gene Med 2002; 4:46-53.
    • (2002) J. Gene Med. , vol.4 , pp. 46-53
    • Schneider, H.1    Muhle, C.2    Douar, A.M.3    Waddington, S.4    Jiang, Q.J.5    von der Mark, K.6


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.