Validation in mesenchymal progenitor cells of a mutation-independent ex vivo approach to gene therapy for osteogenesis imperfecta

Human Molecular Genetics

Volumn 11, Issue 19, 2002, Pages 2201-2206

  Millington Ward, Sophia ^a   Allers, Carolina ^b   Tuohy, Gearóid ^a   Conget, Paulette ^b   Allen, Danny ^a   McMahon, Helena P ^a   Kenna, Paul F ^a   Humphries, Peter ^a   Jane Farrar, G ^a  

^a TRINITY COLLEGE DUBLIN   (Ireland)

^b UNIVERSITY OF CHILE   (Chile)

Author keywords

[No Author keywords available]

Indexed keywords

COLLAGEN TYPE 1; IMMUNOSUPPRESSIVE AGENT; RIBOZYME; RZPOL1A1; UNCLASSIFIED DRUG;

ARTICLE; AUTOTRANSPLANTATION; CELL TRANSPLANTATION; CONTROLLED STUDY; DNA POLYMORPHISM; DOWN REGULATION; GENE DELIVERY SYSTEM; GENE MUTATION; GENE TARGETING; GENE THERAPY; GENETIC TRANSCRIPTION; GRAFT VERSUS HOST REACTION; HUMAN; IMMUNOSUPPRESSIVE TREATMENT; MESENCHYME CELL; NUCLEOTIDE SEQUENCE; OSTEOGENESIS IMPERFECTA; PRIORITY JOURNAL; STEM CELL; VALIDATION PROCESS;

EID: 0037106533     PISSN: 09646906     EISSN: None     Source Type: Journal    
DOI: 10.1093/hmg/11.19.2201     Document Type: Article

References (23)

1. Sillence, D.O. and Rimoin, D.L. (1978) Classification of osteogenesis imperfect. Lancet, 1, 1041-1042.
2. Sillence, D.O., Senn, A. and Danks, D.M. (1979) Genetic heterogeneity in osteogenesis imperfecta. J. Med. Genet., 16, 101-116.
3. Willing, M.C., Pruchno, C.J., Atkinson, M. and Byers, P.H. (1992) Osteogenesis imperfecta type I is commonly due to a COL1A1 null allele of type I collagen. Am. J. Hum. Genet., 51, 508-515.
4. Grassi, G., Forlino, A. and Marini, J.C. (1997) Cleavage of collagen RNA transcripts by hammerhead ribozymes in vitro is mutation-specific and shows competitive binding effects. Nucleic Acids Res., 25, 3451-3458.
5. Dawson, P.A. and Marini, J.C. (2000) Hammerhead ribozymes selectively suppress mutant type I collagen mRNA in osteogenesis imperfecta fibroblasts. Nucleic Acids Res., 28, 4013-4020.
6. Toudjarska, I., Kilpatrick, M.W., Niu, J., Wenstrup, R.J. and Tsipouras, P. (2001) Delivery of a hammerhead ribozyme specifically downregulates mutant type I collagen mRNA in a murine model of osteogenesis imperfecta. Antisense Nucleic Acid Drug Dev., 11, 341-346.
7. Millington-Ward, S., O'Neill, B., Tuohy, G., Al-Jandal, N., Kiang, A.S., Kenna, P.F., Palfi, A., Hayden, P., Mansergh, F., Kennan, A. et al. (1997) Strategems in vitro for gene therapies directed to dominant mutations. Hum. Mol. Genet., 6, 1415-1426.
8. Millington-Ward, S., O'Neill, B., Kiang, A.S., Humphries, P., Kenna, P.F. and Farrar, G.J. (1999) A mutation-independent therapeutic strategem for osteogenesis imperfecta. Antisense Nucleic Acid Drug Dev., 9, 537-542.
9. O'Neill, B., Millington-Ward, S., O'Reilly, M., Tuohy, G., Kiang, A.S., Kenna, P.F., Humphries, P. and Farrar, G.J. (2000) Ribozyme-based therapeutic approaches for autosomal dominant retinitis pigmentosa. Invest. Ophthalmol. Vis. Sci., 41, 2863-2869.
10. Westerhausen, A., Leicht, W. and Heinz, F. (1990) A sequence polymorphism in the 3′-nontranslated region of the pro alpha 1 chain of type I procollagen. Nucleic Acids Res., 18, 4968.
11. Prockop, D.J. (1997) Marrow stromal cells as stem cells for nonhematopoietic tissues. Science, 276, 71-74.
12. Pittenger, M.F., Mackay, A.M., Beck, S.C., Jaiswal, R.K., Douglas, R., Mosca, J.D., Moorman, M.A., Simonetti, D.W., Craig, S. and Marshak, D.R. (1999) Multilineage potential of adult human mesenchymal stem cells. Science, 284, 143-147.
13. Zvaifler, N.J., Marinova-Mutafchieva, L., Adams, G., Edwards, C.J., Moss, J., Burger, J.A. and Maini, R.N. (2000) Mesenchymal precursor cells in the blood of normal individuals. Arthritis Res., 2, 477-488.
14. Horwitz, E.M., Prockop, D.J., Fitzpatrick, L.A., Koo, W.W., Gordon, P.L., Neel, M., Sussman, M., Orchard, P., Marx, J.C., Pyeritz, R.E. and Brenner, M.K. (1999) Transplantability and therapeutic effects of bone marrow-derived mesenchymal cells in children with osteogenesis imperfecta. Nat. Med., 5, 309-313.
15. Horwitz, E.M., Prockop, D.J., Gordon, P.L., Koo, W.W., Fitzpatrick, L.A., Neel, M.D., McCarville, M.E., Orchard, P.J., Pyeritz, R.E. and Brenner, M.K. (2001) Clinical responses to bone marrow transplantation in children with severe osteogenesis imperfecta. Blood, 97, 1227-1231.
16. Pereira, R.F., O'Hara, M.D., Laptev, A.V., Halford, K.W., Pollard, M.D., Class, R., Simon, D., Livezey, K. and Prockop, D.J. (1998) Marrow stromal cells as a source of progenitor cells for nonhematopoietic tissues in transgenic mice with a phenotype of osteogenesis imperfecta. Proc. Natl Acad. Sci. USA, 95, 1142-1147.
17. Onyia, J.E., Clapp, D.W., Long, H. and Hock, J.M. (1998) Osteoprogenitor cells as targets for ex vivo gene transfer. J. Bone Miner. Res., 13, 20-30.
18. Ding, L., Lu, S., Batchu, R., III, R.S. and Munshi, N. (1999) Bone marrow stromal cells as a vehicle for gene transfer. Gene Ther., 6, 1611-1616.
19. Primorac, D., Rowe, D.W., Mottes, M., Barisic, I., Anticevic, D., Mirandola, S., Gomez Lira, M., Kalajzic, I., Kusec, V. and Glorieux, F.H. (2001) Osteogenesis imperfecta at the beginning of bone and joint decade. Croat. Med. J., 42, 393-415.
20. Selvey, S., Thompson, E.W., Matthaei, K., Lea, R.A., Irving, M.G. and Griffiths, L.R. (2001) beta-Actin - an unsuitable internal control for RT-PCR. Mol. Cell Probes, 15, 307-311.
21. Conget, P.A. and Minguell, J.J. (1999) Phenotypical and functional properties of human bone marrow mesenchymal progenitor cells. J. Cell Physiol., 181, 67-73.
22. Conget, P.A. and Minguell, J.J. (2000) Adenoviral-mediated gene transfer into ex vivo expanded human bone marrow mesenchymal progenitor cells. Exp. Hematol., 28, 382-390.
23. Yee, J.K., Miyanohara, A., LaPorte, P., Bouic, K., Burns, J.C. and Friedmann, T. (1994) A general method for the generation of high-titer, pantropic retroviral vectors: highly efficient infection of primary hepatocytes. Proc. Natl Acad. Sci. USA, 91, 9564-9568.