Gene therapy for hemophilia

Current Opinion in Molecular Therapeutics

Volumn 1, Issue 4, 1999, Pages 493-499

  Lynch, Carmel M ^a  

^a Targeted Genetics Corporation   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

COMPLEMENTARY DNA; RECOMBINANT BLOOD CLOTTING FACTOR 8; RECOMBINANT BLOOD CLOTTING FACTOR 9; VIRUS VECTOR;

DRUG RESEARCH; GENE TARGETING; GENE THERAPY; HEMOPHILIA A; HEMOPHILIA B; HUMAN; NONHUMAN; PROMOTER REGION; PROTEIN EXPRESSION; REVIEW; TISSUE DISTRIBUTION; TREATMENT OUTCOME;

ADENO-ASSOCIATED VIRUS; CANIS FAMILIARIS; MURINAE;

EID: 0032862997     PISSN: 14648431     EISSN: None     Source Type: Journal    
DOI: None     Document Type: Review

References (52)

1. Gitschier J, Kogan S, Diamond C, Levinson B: Genetic basis of hemophilia A. Thromb Haemost (1991) 66:37-39.
2. DiMichele D, Neufeld EJ: Hemophilia. A new approach to an old disease. Hematol Oncol Clin North Am (1998) 12:1315-1344.
3. Palmer TD, Thompson AR, Miller AD: Production of human factor IX in animals by genetically modified skin fibroblasts: potential therapy for hemophilia B. Blood (1989) 73:438-445.
4. Hoeben RC, van der Jagt RC, Schoute F, van Tilburg NH, Verbeet MP, Briët E, van Ormondt H, van der Eb AJ: Expression of functional factor VIII in primary human skin fibroblasts after retrovirus-mediated gene transfer. J Biol Chem (1990) 265:7318-7323.
5. Lynch CM, Israel DI, Kaufman RJ, Miller AD: Sequences in the coding region of clotting factor VIII act as dominant inhibitors of RNA accumulation and protein production. Hum Gene Ther (1993) 4:259-272.
6. Hoeben RC, Einerhand MP, Briët E, van Ormondt H, Valerio D, van der Eb AJ: Toward gene therapy in haemophilia A: retrovirus-mediated transfer of a factor VIII gene into murine haematopoietic progenitor cells. Thromb Haemost (1992) 67:341-345.
7. Chuah MK, Brems H, Vanslembrouck V, Collen D, Vandendriessche T: Bone marrow stromal cells as targets for gene therapy of hemophilia A. Hum Gene Ther (1998) 9:353-365.
8. Armentano D, Thompson AR, Darlington C, Woo SL: Expression of human factor IX in rabbit hepatocytes by retrovirus-mediated gene transfer: potential for gene therapy of hemophilia B. Proc Natl Acad Sci USA (1990) 87:6141-6145.
9. Kay MA: Hepatic gene therapy for haemophilia B. Haemophilia (1998) 4:389-392.
10. Roman M, Axelrod JH, Dai Y, Naviaux RK, Friedmann T, Verma IM: Circulating human or canine factor IX from retrovirally transduced primary myoblasts and established myoblast cell lines grafted into murine skeletal muscle. Somat Cell Mol Genet (1992) 18:247-258.
11. Yao SN, Kurachi K: Expression of human factor IX in mice after injection of genetically modified myoblasts. Proc Natl Acad Sci USA (1992) 89:3357-3361.
12. Hoeben RC, Fallaux FJ, Van Tilburg NH, Cramer SJ, Van Ormondt H, Briët E, Van Der Eb AJ: Toward gene therapy for hemophilia A: long-term persistence of factor VIII-secreting fibroblasts after transplantation into immunodeficient mice. Hum Gene Ther (1993) 4:179-186.
13. Zatloukal K, Cotten M, Berger M, Schmidt W, Wagner E, Bimstiel ML: In vivo production of human factor VII in mice after intrasplenic implantation of primary fibroblasts transfected by receptor-mediated, adenovirus-augmented gene delivery. Proc Natl Acad Sci USA (1994) 91:5148-5152.
14. Dwarki VJ, Belloni P, Nijjar T, Smith J, Couto L, Rabier M, Clift S, Bems A, Cohen LK: Gene therapy for hemophilia A: production of therapeutic levels of human factor VIII in vivo in mice. Proc Natl Acad Sci USA (1995) 92:1023-1027.
15. Miller DG, Adam MA, Miller AD: Gene transfer by retrovirus vectors occurs only in cells that are actively replicating at the time of infection. Mol Cell Biol (1990) 10:4239-4242.
16. Kay MA, Rothenberg S, Landen CN, Bellinger DA, Leland F, Toman C, Finegold M, Thompson AR, Read MS, Brinkhous KM, Woo SLC: In vivo gene therapy of hemophilia B: sustained partial correction in factor IX-deficient dogs. Science (1993) 262:117-119.
17. Kay MA, Landen CN, Rothenberg SR, Taylor LA, Leland F, Wienie S, Fang B, Bellinger D, Finegold M, Thompson AR, Read M, Brinkhouse KM, Woo SLC: In vivo hepatic gene therapy: complete albeit transient correction of factor IX deficiency in hemophilia B dogs. Proc Natl Acad Sci USA (1994) 91:2353-2357.
18. Hoeben RC, Fallaux FJ, Cramer SJ, van den Wollenberg DJ, van Ormondt H, Briët E, van der Eb AJ: Expression of the blood-clotting factor-VIII cDNA is repressed by a transcriptional silencer located in its coding region. Blood (1995) 85:2447-2454.
19. Koeberl DD, Halbert CL, Krumm A, Miller AD: Sequences within the coding regions of clotting factor VIII and CFTR block transcriptional elongation. Hum Gene Ther (1995) 6:469-479.
20. Chuah MK, Vandendriessche T, Morgan RA: Development and analysis of retroviral vectors expressing human factor VIII as a potential gene therapy for hemophilia A. Hum Gene Ther (1995) 6:1363-1377.
21. Smith TA, Mehaffey MG, Kayda DB, Saunders JM, Yei S, Trapnell BC, McClelland A, Kaleko M: Adenovirus mediated expression of therapeutic plasma levels of human factor IX in mice. Nature Genet (1993) 5:397-402.
22. Connelly S, Smith TA, Dhir G, Gardner JM, Mehaffey MG, Zaret KS, McClelland A, Kaleko M: In vivo gene delivery and expression of physiological levels of functional human factor VIII in mice. Hum Gene Ther (1995) 6:185-193.
23. Connelly S, Mount J, Mauser A, Gardner JM, Kaleko M, McClelland A, Le CD Jr. Complete short-term correction of canine nemophilia A by in vivo gene therapy. Stood (1996) 88:3846-3853.
24. Connelly S, Gardner JM, McClelland A, Kaleko M: High-level tissue-specific expression of functional human factor VIII in mice. Hum Gene Ther (1996) 7:183-195.
25. Connelly S, Andrews JL, Gallo AM, Kayda DB, Qian J, Hoyer L, Kadan MJ, Gorziglia MI, Trapnell BC, McClelland A, Kaleko M: Sustained phenotypic correction of murine hemophilia A by in vivo gene therapy. Blood (1998) 91:3273-3281.
26. Connelly S, Kaleko M: Haemophilia A gene therapy. Haemophilia (1998) 4:380-388.
27. Clemens PR, Kochanek S, Sunada Y, Chan S, Chen HH, Campbell KP, Caskey CT: In vivo muscle gene transfer of full-length dystrophin with an adenoviral vector that lacks all viral genes. Gene Ther (1996) 3:965-972.
28. Lieber A, He CY, Kirillova I, Kay MA: Recombinant adenoviruses with large deletions generated by Cremediated excision exhibit different biological properties compared with first-generation vectors in vitro and in vivo. J Virol (1996) 70:8944-8960.
29. Gao GP, Yang Y, Wilson JM: Biology of adenovirus vectors with E1 and E4 deletions for liver- directed gene therapy. J Virol (1996) 70:8934-8943.
30. Flotte TR, Afione SA, Conrad C, McGrath SA, Solow R, Oka H, Zeitlin PL, Guggino WB, Carter BJ: Stable in vivo expression of the cystic fibrosis transmembrane conductance regulator with an adeno-associated virus vector. Proc Natl Acad Sci USA (1993) 90:10613-10617.
31. Clark KR, Sferra TJ, Johnson PR: Recombinant adeno-associated viral vectors mediate long-term transgene expression in muscle. Hum Gene Ther (1997) 8:659-669.
32. Fisher KJ, Jooss K, Alston J, Yang Y, Haecker SE, High K, Pathak R, Raper SE, Wilson JM: Recombinant adeno-associated virus for muscle directed gene therapy. Nature Med (1997) 3:306-312.
33. Kessler PD, Podsakoff GM, Chen X, McQuiston SA, Colosi PC, Matelis LA, Kurtzman GJ, Byme BJ: Gene delivery to skeletal muscle results in sustained expression and systemic delivery of a therapeutic protein. Proc Natl Acad Sci USA (1996) 93:14082-14087.
34. Xiao X, Li J, Samulski RJ: Efficient long-term gene transfer into muscle tissue of immunocompetent mice by adeno-associated virus vector. J Virol (1996) 70:8098-8108.
35. Koeberl DD, Alexander IE, Halbert CL, Russell DW, Miller AD: Persistent expression of human clotting factor IX from mouse liver after intravenous injection of adeno-associated virus vectors. Proc Natl Acad Sci USA (1997) 94:1426-1431.
36. Snyder RO, Miao CH, Patijn GA, Spratt SK, Danos O, Nagy D, Gown AM, Winther B, Meuse L, Cohen LK, Thompson AR, Kay MA: Persistent and therapeutic concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectors. Nature Genet (1997) 16:270-276.
37. Dranoff G, Jaffee E, Lazenby A, Golumbek P, Levitsky H, Brose K, Jackson V, Hamada H, Pardoll D, Mulligan RC: Vaccination with irradiated tumor cells engineered to secrete murine granulocyte-macrophage colony-stimulating factor stimulates potent, specific, and long-lasting anti-tumor immunity. Proc Natl Acad Sci USA (1993) 90:3539-3543.
38. Nakai H, Herzog RW, Hagstrom JN, Walter J, Kung SH, Yang EY, Tai SJ, Iwaki Y, Kurtzman GJ, Fisher KJ, Colosi P, Couto LB, High KA: Adeno-associated viral vector-mediated gene transfer of human blood coagulation factor IX into mouse liver. Blood (1998) 91:4600-4607.
39. Wang L, Takabe K, Bidlingmaier SM, III CR, Verma IM: Sustained correction of bleeding disorder in hemophilia B mice by gene therapy. Proc Natl Acad Sci USA (1999) 96:3906-3910.
40. Kay MA, Li Q, Liu TJ, Leland F, Toman C, Finegold M, Woo SL: Hepatic gene therapy: persistent expression of human alpha 1-antitrypsin in mice after direct gene delivery in vivo. Hum Gene Ther (1992) 3:641-647.
41. Guo ZS, Wang LH, Eisensmith RC, Woo SL: Evaluation of promoter strength for hepatic gene expression in vivo following adenovirus-mediated gene transfer. Gene Ther (1996) 3:802-810.
42. Baskar JF, Smith PP, Nilaver G, Jupp RA, Hoffmann S, Peffer NJ, Tenney DJ, Colberg-Poley AM, Ghazal P, Nelson JA: The enhancer domain of the human cytomegalovirus major immediate-early promoter determines cell type-specific expression in transgenic mice. J Virol (1996) 70:3207-3214.
43. Snyder RO, Spratt SK, Lagarde C, Bohl D, Kaspar B, Sloan B, Cohen LK, Danos O: Efficient and stable adeno-associated virus-mediated transduction in the skeletal muscle of adult immunocompetent mice. Hum Gene Ther (1997) 8:1891-1900.
44. Lin HF, Maeda N, Smithies O, Straight DL, Stafford DW: A coagulation factor IX-deficient mouse model for human hemophilia B. Blood (1997) 90:3962-3966.
45. Wang L, Zoppe M, Hackeng TM, Griffin JH, Lee KF, Verma IM: A factor IX-deficient mouse model for hemophilia B gene therapy. Proc Natl Acad Sci USA (1997) 94:11563-11566.
46. Kundu RK, Sangiorgi F, Wu LY, Kurachi K, Anderson WF, Maxson R, Gordon EM: Targeted Inactivation of the coagulation factor IX gene causes hemophilia B in mice. Blood (1998) 92:168-174.
47. Snyder RO, Miao C, Meuse L, Tubb J, Donahue BA, Lin HF, Stafford DW, Patel S, Thompson AR, Nichols T, Read MS, Bellinger DA, Brinkhous KM, Kay MA: Correction of hemophilia B in canine and murine models using recombinant adeno-associated viral vectors. Nat Med (1999) 5:64-70.
48. Herzog RW, Hagstrom JN, Kung SH, Tai SJ, Wilson JM, Fisher KJ, High KA: Stable gene transfer and expression of human blood coagulation factor IX after intramuscular injection of recombinant adeno-associated virus. Proc Natl Acad Sci USA (1997) 94:5804-5809.
49. Herzog RW, Yang EY, Couto LB, Hagstrom JN, Elwell D, Fields PA, Burton M, Bellinger DA, Read MS, Brinkhous KM, Podsakoff GM, Nichols TC, Kurtzman GJ, High KA: Long-term correction of canine hemophilia B by gene transfer of blood coagulation factor IX mediated by adeno-associated viral vector. Nature Med (1999) 5:56-63.
50. Dong JY, Fan PD, Frizzell RA: Quantitative analysis of the packaging capacity of recombinant adeno-associated virus. Hum Gene Ther (1996) 7:2101-2112.
51. Gnatenko DV, Saenko EL, Jesty J, Cao LX, Hearing P, Bahou WF: Human factor VIII can be packaged and functionally expressed in an adeno-associated virus background: applicability to haemophilia A gene therapy. Br J Haematol (1999) 104:27-36.
52. Chao H, Mao L, Walsh CE: Long-term therapeutic levels of human factor VIII expressed in mice. Second Ann Am Soc Gene Ther. (1999):Abs 936.