Angiogenesis Enhances Factor IX Delivery and Persistence from Retrievable Human Bioengineered Muscle Implants

Molecular Therapy

Volumn 14, Issue 3, 2006, Pages 442-451

  Thorrez, Lieven ^a   Vandenburgh, Herman ^a,b   Callewaert, Nico ^a   Mertens, Nico ^a   Shansky, Janet ^b   Wang, Lin ^b   Arnout, Jef ^c   Collen, Désiré ^a   Chuah, Marinee ^a   VandenDriessche, Thierry ^a  

^a DEPARTMENT OF PLANT SYSTEMS BIOLOGY   (Belgium)

^b MIRIAM HOSPITAL   (United States)

^c UNIVERSITY OF LEUVEN   (Belgium)

Author keywords

angiogenesis; factor IX; muscle; tissue engineering; VEGF

Indexed keywords

BLOOD CLOTTING FACTOR 9; GREEN FLUORESCENT PROTEIN; LENTIVIRUS VECTOR; RECOMBINANT BLOOD CLOTTING FACTOR 9; VASCULOTROPIN; VASCULOTROPIN A;

ADULT; ANGIOGENESIS; ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL TISSUE; ARTICLE; BIOARTIFICIAL ORGAN; BIOENGINEERING; CELL ACTIVITY; CELLULAR DISTRIBUTION; CONTROLLED STUDY; FEMALE; GENE LOCATION; GENE TRANSFER; HUMAN; HUMAN CELL; HUMAN TISSUE; MALE; MITOSIS; MOUSE; MUSCLE CELL; MYOBLAST; NONHUMAN; PROTEIN PROCESSING; SAFETY; STEM CELL GENE THERAPY;

ANIMALS; FACTOR IX; GENE THERAPY; GENETIC VECTORS; GREEN FLUORESCENT PROTEINS; HEMOPHILIA A; HUMANS; LENTIVIRUS; MICE; MICE, SCID; MYOBLASTS, SKELETAL; NEOVASCULARIZATION, PHYSIOLOGIC; TISSUE ENGINEERING; TRANSDUCTION, GENETIC; VASCULAR ENDOTHELIAL GROWTH FACTOR A;

EID: 33746867991     PISSN: 15250016     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.ymthe.2006.03.019     Document Type: Article

References (47)

1. Chuah M.K., Collen D., and Vandendriessche T. Preclinical and clinical gene therapy for haemophilia. Haemophilia 10 Suppl. 4 (2004) 119-125
2. Manno C.S., et al. Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response. Nat. Med. 12 (2006) 342-347
3. Chuah M.K., et al. Therapeutic factor VIII levels and negligible toxicity in mouse and dog models of hemophilia A following gene therapy with high-capacity adenoviral vectors. Blood 101 (2003) 1734-1743
4. Qiu X., et al. Implantation of autologous skin fibroblast genetically modified to secrete clotting factor IX partially corrects the hemorrhagic tendencies in two hemophilia B patients. Chin. Med. J. (Engl.) 109 (1996) 832-839
5. Roth D.A., Tawa Jr. N.E., O'Brien J.M., Treco D.A., and Selden R.F. Nonviral transfer of the gene encoding coagulation factor VIII in patients with severe hemophilia A. N. Engl. J. Med. 344 (2001) 1735-1742
6. Cavazzana-Calvo M., et al. Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease. Science 288 (2000) 669-672
7. Hacein-Bey-Abina S., et al. LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science 302 (2003) 415-419
8. Dwarki V.J., et al. Gene therapy for hemophilia A: production of therapeutic levels of human factor VIII in vivo in mice. Proc. Natl. Acad. Sci. USA 92 (1995) 1023-1027
9. Arruda V.R., et al. Posttranslational modifications of recombinant myotube-synthesized human factor IX. Blood 97 (2001) 130-138
10. Arruda V.R., et al. Safety and efficacy of factor IX gene transfer to skeletal muscle in murine and canine hemophilia B models by adeno-associated viral vector serotype 1. Blood 103 (2004) 85-92
11. Dai Y., Roman M., Naviaux R.K., and Verma I.M. Gene therapy via primary myoblasts: long-term expression of factor IX protein following transplantation in vivo. Proc. Natl. Acad. Sci. USA 89 (1992) 10892-10895
12. Wang J.M., Zheng H., Blaivas M., and Kurachi K. Persistent systemic production of human factor IX in mice by skeletal myoblast-mediated gene transfer: feasibility of repeat application to obtain therapeutic levels. Blood 90 (1997) 1075-1082
13. Dhawan J., Pan L.C., Pavlath G.K., Travis M.A., Lanctot A.M., and Blau H.M. Systemic delivery of human growth hormone by injection of genetically engineered myoblasts. Science 254 (1991) 1509-1512
14. Cooper R.N., Thiesson D., Furling D., Di Santo J.P., Butler-Browne G.S., and Mouly V. Extended amplification in vitro and replicative senescence: key factors implicated in the success of human myoblast transplantation. Hum. Gene Ther. 14 (2003) 1169-1179
15. Renault V., Thornell L.E., Eriksson P.O., Butler-Browne G., and Mouly V. Regenerative potential of human skeletal muscle during aging. Aging Cell 1 (2002) 132-139
16. Gussoni E., Blau H.M., and Kunkel L.M. The fate of individual myoblasts after transplantation into muscles of DMD patients. Nat. Med. 3 (1997) 970-977
17. Menasche P. Skeletal muscle satellite cell transplantation. Cardiovasc. Res. 58 (2003) 351-357
18. Vandenburgh H., et al. Tissue-engineered skeletal muscle organoids for reversible gene therapy. Hum. Gene Ther. 7 (1996) 2195-2200
19. Powell C.A., Smiley B.L., Mills J., and Vandenburgh H.H. Mechanical stimulation improves tissue-engineered human skeletal muscle. Am. J. Physiol. Cell Physiol. 283 (2002) C1557-C1565
20. VandenDriessche T., et al. Lentiviral vectors containing the human immunodeficiency virus type-1 central polypurine tract can efficiently transduce nondividing hepatocytes and antigen-presenting cells in vivo. Blood 100 (2002) 813-822
21. Hagstrom J.N., et al. Improved muscle-derived expression of human coagulation factor IX from a skeletal actin/CMV hybrid enhancer/promoter. Blood 95 (2000) 2536-2542
22. Axelrod J.H., Read M.S., Brinkhous K.M., and Verma I.M. Phenotypic correction of factor IX deficiency in skin fibroblasts of hemophilic dogs. Proc. Natl. Acad. Sci. USA 87 (1990) 5173-5177
23. Krebsbach P.H., Zhang K., Malik A.K., and Kurachi K. Bone marrow stromal cells as a genetic platform for systemic delivery of therapeutic proteins in vivo: human factor IX model. J. Gene Med. 5 (2003) 11-17
24. Powell C., et al. Tissue-engineered human bioartificial muscles expressing a foreign recombinant protein for gene therapy. Hum. Gene Ther. 10 (1999) 565-577
25. Carmeliet P. VEGF gene therapy: stimulating angiogenesis or angioma-genesis?. Nat. Med. 6 (2000) 1102-1103
26. Lin Y., Chang L., Solovey A., Healey J.F., Lollar P., and Hebbel R.P. Use of blood outgrowth endothelial cells for gene therapy for hemophilia A. Blood 99 (2002) 457-462
27. Brauker J., et al. Sustained expression of high levels of human factor IX from human cells implanted within an immunoisolation device into athymic rodents. Hum. Gene Ther. 9 (1998) 879-888
28. Hortelano G., Wang L., Xu N., and Ofosu F.A. Sustained and therapeutic delivery of factor IX in nude haemophilia B mice by encapsulated C2C12 myoblasts: concurrent tumourigenesis. Haemophilia 7 (2001) 207-214
29. Balland A., et al. Characterisation of two differently processed forms of human recombinant factor IX synthesised in CHO cells transformed with a polycistronic vector. Eur. J. Biochem. 172 (1988) 565-572
30. Diuguid D.L., Rabiet M.J., Furie B.C., Liebman H.A., and Furie B. Molecular basis of hemophilia B: a defective enzyme due to an unprocessed propeptide is caused by a point mutation in the factor IX precursor. Proc. Natl. Acad. Sci. USA 83 (1986) 5803-5807
31. Mount J.D., et al. Sustained phenotypic correction of hemophilia B dogs with a factor IX null mutation by liver-directed gene therapy. Blood 99 (2002) 2670-2676
32. Ashwell G., and Harford J. Carbohydrate-specific receptors of the liver. Annu. Rev. Biochem. 51 (1982) 531-554
33. Fischer B.E., and Dorner F. Recombinant coagulation factor IX: glycosylation analysis and in vitro conversion into human-like sialylation pattern. Thromb. Res. 89 (1998) 147-150
34. Shapiro A.D., et al. The safety and efficacy of recombinant human blood coagulation factor IX in previously untreated patients with severe or moderately severe hemophilia B. Blood 105 (2005) 518-525
35. Poon M.C., Lillicrap D., Hensman C., Card R., and Scully M.F. Recombinant factor IX recovery and inhibitor safety: a Canadian post-licensure surveillance study. Thromb. Haemostasis 87 (2002) 431-435
36. Yao S.N., and Kurachi K. Expression of human factor IX in mice after injection of genetically modified myoblasts. Proc. Natl. Acad. Sci. USA 89 (1992) 3357-3361
37. Chuah M.K., et al. Long-term persistence of human bone marrow stromal cells transduced with factor VIII-retroviral vectors and transient production of therapeutic levels of human factor VIII in nonmyeloablated immunodeficient mice. Hum. Gene Ther. 11 (2000) 729-738
38. Aiuti A., et al. Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning. Science 296 (2002) 2410-2413
39. Luttun A., et al. Revascularization of ischemic tissues by PlGF treatment, and inhibition of tumor angiogenesis, arthritis and atherosclerosis by anti-Flt1. Nat. Med. 8 (2002) 831-840
40. Park F., Ohashi K., and Kay M.A. Therapeutic levels of human factor VIII and IX using HIV-1-based lentiviral vectors in mouse liver. Blood 96 (2000) 1173-1176
41. Follenzi A., Ailles L.E., Bakovic S., Geuna M., and Naldini L. Gene transfer by lentiviral vectors is limited by nuclear translocation and rescued by HIV-1 pol sequences. Nat. Genet. 25 (2000) 217-222
42. Shultz L.D., et al. Human lymphoid and myeloid cell development in NOD/LtSz-scid IL2R{gamma}null mice engrafted with mobilized human hemopoietic stem cells. J. Immunol. 174 (2005) 6477-6489
43. Hennessey J.V., Chromiak J.A., Della Ventura S., Guertin J., and MacLean D.B. Increase in percutaneous muscle biopsy yield with a suction-enhancement technique. J. Appl. Physiol. 82 (1997) 1739-1742
44. Powell C., Shansky J., Del Tatto M., and Vandenburgh H.H. Bioartificial muscles in gene therapy. In: Morgan J.R. (Ed). Methods in Molecular Medicine, vol. 69: Gene Therapy Protocols (2001), Humana Press, Totowa, NJ 149-160
45. Niwa H., Yamamura K., and Miyazaki J. Efficient selection for high-expression transfectants with a novel eukaryotic vector. Gene 108 (1991) 193-199
46. Kay M.A., et al. Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector. Nat. Genet. 24 (2000) 257-261
47. Lu Y., Shansky J., Del Tatto M., Ferland P., Wang X., and Vandenburgh H. Recombinant vascular endothelial growth factor secreted from tissue-engineered bioartificial muscles promotes localized angiogenesis. Circulation 104 (2001) 594-599