Chemistry: molecular biology and microbiology – Process of mutation – cell fusion – or genetic modification – Introduction of a polynucleotide molecule into or...
Patent
1997-07-10
1999-09-28
Saunders, David
Chemistry: molecular biology and microbiology
Process of mutation, cell fusion, or genetic modification
Introduction of a polynucleotide molecule into or...
435456, 435325, 435372, 435373, 435384, 435385, 435386, 435405, 4353201, 530350, 5303871, 53038823, 536 245, C12N 1512, C12N 510, C07K 1624, C07H 2104
Patent
active
059587741
DESCRIPTION:
BRIEF SUMMARY
BACKGROUND OF THE INVENTION
Generally, the invention relates to a method for gene transfer into cells activated from a quiescent state, in particular hematopoietic stem cells, and the cells thus obtained.
DESCRIPTION OF THE RELATED ART
Progress in the identification and cloning of genes that are responsible for human genetic diseases has led to a major program for the improvement of gene transfer technology (Anderson, W. F. (1992) Science, 256: 808-813; Miller, A. D. (1992) Nature, 357: 455-460; Morgan, R. A., & Anderson, W. F. (1993) Annu. Rev. Biochem., 62: 191-217; Karilsson, S. (1991) Blood, 78: 2481-2492). For example, different viral vectors have been used for hematopoietic stem cells, each having their own advantage (Anderson, W. F. (1992), mentioned above). The insertion of a retroviral vector into the host genome ensures its replication in the host cell. This insertion requires an active proliferative state (Anderson, W. F. (1992), mentioned above; Varmus, H. E., Padgett, T., Heasley, S., Simon, G., & Bishop, J. M. (1977), Cell., 11: 307-319; Nolta, J. A., & Kohn, D. B. (1990) Hum. Gene Ther., 1: 257-268; Miller, D. G., Adam, M. A., & Miller, A. D. (1990) Mol. Cell. Biol., 10: 4239-4242) which does not exist in the compartment of the hematopoietic stem cell which is in a quiescent state (Lajtha, L. G., & Schofield, R. (1974) Differenciation, 2: 313-320), or in other types of stem cells and somatic cells such as the hepatic cells. Using antisense oligonucleotides against inhibitor gene, in particular TGF-.beta.-1, has shown that the early progenitors of human bone marrow can be released from their quiescent state by blockage of an autocrine TGF-.beta. (Hatzfeld, J., Li, M.-L., Brown, E. L., Sookdeo, H., Levesque, J.-P., O'Toole, T., Gurney, C., Clark, S. C., and Hatzfeld, A. (1991), J. Exp. Med., 174: 925-929).
Moreover, where transplants of hematopoietic tissue are concerned, in the past, the capacity for transplanting samples of bone marrow has been analysed by estimating the quantity of CFU-GM (colony-forming unit--granulocyte/macrophage: granulo-monocytic progenitor) cells. However, these cells which probably play an important part in cell generation for a short period following the transplantation of a graft may not reflect the quantity of more primitive cells, and in particular of hematopoietic stem cells which are important in long-term hematopoiesis. The stem cells involved in long-term transplants represent a small sub-population of cells which may possibly have the phenotype CD34+CD38- (compartment of the hematopoietic stem cell: cells rich in membrane antigen CD34 and poor in maturation membrane antigen CD38).
Recently, human cord blood has proved to be sufficient for reconstituting hematopoiesis after transplantation in children (Gluckman, E., Broxmeyer, H. E., Auerbach, A. D., Friedman, H. S., Douglas, G. W., Devergie, A., Esperou, H., Thierry, D., Socie, G., Lehn, P., Cooper, S., English, D., Kurtzberg, J., Bard, J., & Boyse, E. A. (1989), N. Eng. J. Med., 3: 1174-1178; Broxmeyer, H. E., Douglas, G. W., Hangoe, G., Cooper, S., Bard, J., English, D., Arny, M., Thomas, L., & Boyse, E. A. (1989) Proc. Natl. Acad. Sci. USA, 86: 3828-3832; Broxmeyer, H. E., Hangoe, G., & Cooper, S. (1992) Bone Marrow Transplant., 9: 7-10). In vitro, the data from various laboratories (Broxmeyer, H. E., Hangoe, G., Cooper, S., Ribeiro, R. C., Graves, V., Yoder, M., Wagner, J., Vadhan-Raj, S., Benninger, L., Rubinstein, P., & Broun, E. R. (1992) Proc. Natl. Acad. Sci. USA, 89: 4109-4113; Lu, L., Xiao, M., Shen, R.-N., Grisby, S., & Broxmeyer, H. E. (1993) Blood, 81: 41-48; Hows, J. M., Bradley, B. A., Marsh, J. C. W., Luft, T., Coutinho, L., Testa, N. G., & Dexter, T. M. (1992) Lancet, 340: 73-76; Cardoso, A. A., Li, M. L., Batard, P., Hatzfeld, A., Brown, E. L., Levesque, J.-P., Sookdeo, H., Panterne, B., Sansilvestri, P., Clark, S. C., & Hatzfeld, J. (1993) Proc. Natl. Acad. Sci. USA, 90: 8707-8711, suggesting that cord blood has a greater capacity for generating progenitors in culture than that of bon
REFERENCES:
By A. Hatzfeld et al., "Increased Stable Retroviral Gene Transfer in Early Hematopoietic Progenitors Released from Quienscence", Human Gene Therapy, vol. 7, No. 2, Jan. 20, 1996, pp. 207-213.
By P. Sansilvestri et al., "Early CD34.sup.high Cells Can be Separated into KIT.sup.high Cells in Which Transforming Growth Factor-.beta. (TGF-.beta.) Downmodulates c-kit and KIT.sup.low Cells in Which Anti-TGF-.beta. Upmodulates c-kit", Blood, vol. 86, No. 5, Sep. 1, 1995, pp. 1729-1735.
By Olivier Danos et al., "Safe and efficient generation of recombinant retroviruses with amphotropic and ecotropic host ranges", Proceedings of the National Academy of Sciences, vol. 85, No. 17, Sep. 1988, pp. 6460-6464.
By R. K. Strair et al., "Retroviral mediated gene transfer into bone marrow progenitor cells: use of beta-galactosi-dase as a selectable marker", Nucleic Acids Research, vol. 18, No. 16, Aug. 25, 1990, pp. 4759-4762.
By N. Ferry et al., "Retroviral-mediated gene transfer into hepatocytes in vivo" Proceedings of the National Academy of Sciences, vol. 88, No. 19, Oct. 1, 1991, pp. 8377-8381.
J.M. Hows et al., "Growth of human umbilical-cord blood in longterm haemopoetic cultures", The Lancet, vo. 340, No. 8810, Jul. 4, 1992, pp. 73-76.
By J. Hatzfeld et al., "Release of Early Human Hematopoetic Progenitors from Quiescence by Antisense Transforming Growth Factor .beta.1 or Rb Oligonucleotides", The Journal of Experimental Medicine, vol. 174, Oct. 1991, 925-929.
By J. Hatzfeld et al., "Antisense oligonucleotides for inhibitors and tumor suppressor genes reveal the hemato-poietic potential of quiescent progenitors", The Negative Regulation of Hematopoiesis, vol. 229, 1993, pp. 283-289.
By L. Lu et al., "High Efficience Retroviral Mediated Gene Transduction into Single Isolated Immature and Replatable CD34.sup.3+ Hematopoietic Stem/Progenitor Cells from Human Umbilical Cord Blood", The Journal of Experimental Medi-cine, vol. 178, No. 6, Dec. 1, 1993, pp. 2089-2096.
By T. Moritz et al., "Human Cord Blood Cells as Targets for Gene Transfer: Potential Use in Genetic Therapies of Severe Combined Immunodeficiency Disease", The Journal of Experimental Medicine, vol. 178, Aug. 1993, pp. 529-536.
By J. Hatzfeld et al., "Purification and Release from Quiescence of Umbilical Cord Blood Early Progenitors Reveal Their Potential to Engraft Adults", Blood Cells, 1994, pp. 430-435.
By Ma-Lin Li et al., "Additive Effects of Steel Factor and Antisense TGF-.beta.1 Oligodeoxynucleotide on CD34.sup.+ Hematopoi-etic Progenitor Cells", Leukemia, vol. 8, No. 3, Mar. 1994, pp. 441-445.
BY A.A. Cardoso et al., "Release from quiescence of CD34.sup.+ CD38.sup.- human umbilical cord blood cells reveals their potentially to engraft adults", Proceedings of the Na-tional Academy of Sciences, vol. 90, No. 18, Sep. 15, 1993, pp. 8707-8711.
By A. Hatzfeld et al., "Release from Quiescence of the Human Hematopoietic Stem Cell Compartment", British Jour-nal of Haematology, vol. 87, Supplement 1, 1994, p. 97.
By A. Hatzfeld et al., Release from Quiescence of the Human Hematopoietic Stem Cell Compartment: Application in Early Progenitor Amplification and High Efficiency Gene Transfer, Blood -The Journal of The American Society of Hematology, vol. 84, No. 10, Suppl. 1, Nov. 15, 1994, p. 126a.
By A. Hatzfeld et al., "Large Scale Gene Transfer in Early Human Hematopoietic Progenitors", Journal of Cellular Biochemistry, 1993, p. 249 .
Hatzfeld Jacques
Klein Antoinette
Centre National de la Recherche Scientifique
Saunders David
VanderVegt F. Pierre
LandOfFree
Method for gene transfer into cells activated from a quiescent s does not yet have a rating. At this time, there are no reviews or comments for this patent.
If you have personal experience with Method for gene transfer into cells activated from a quiescent s, we encourage you to share that experience with our LandOfFree.com community. Your opinion is very important and Method for gene transfer into cells activated from a quiescent s will most certainly appreciate the feedback.
Profile ID: LFUS-PAI-O-702496