Chemistry: molecular biology and microbiology – Process of mutation – cell fusion – or genetic modification – Introduction of a polynucleotide molecule into or...
Reexamination Certificate
2005-02-01
2005-02-01
Falk, Anne-Marie (Department: 1636)
Chemistry: molecular biology and microbiology
Process of mutation, cell fusion, or genetic modification
Introduction of a polynucleotide molecule into or...
C435S235100, C435S325000
Reexamination Certificate
active
06849454
ABSTRACT:
The present invention pertains to a method for efficiently introducing exogenous genes into stem cells, particularly human stem cells. The method optionally includes the steps of inducing the proliferation of target cells by pre-stimulation with cytokines and/or growth factors, followed by incubating these cells with RD114-pseudotyped vector particles. In a specific embodiment, the vector particles are retronectin-immobilized or ultracentrifugation-concentrated retroviral vector particles pseudotyped with the feline endogenous retrovirus (RD114) envelope protein. The present invention further discloses a method for somatic gene therapy, which can be used for various therapeutic applications and involves introducing a gene of interest contained within the retroviral genome into human repopulating stem cells followed by introducing these cells into a human host. Finally, the present invention discloses a method for monitoring the efficiency of the stem cell-mediated gene transfer based on detecting the presence of the genes (or the expression products) of the retroviral vector in various stem cell-derived lineages of the host.
REFERENCES:
patent: 4861719 (1989-08-01), Miller
patent: 5667998 (1997-09-01), Dougherty et al.
patent: 5910434 (1999-06-01), Rigg et al.
patent: 5952225 (1999-09-01), Pensiero et al.
patent: 6017761 (2000-01-01), Rigg et al.
patent: WO 9604934 (1996-02-01), None
patent: PCTGB96/02061 (1996-08-01), None
patent: WO 9915684 (1999-04-01), None
patent: WO 9932646 (1999-07-01), None
Hennemann et al. Optimization of retroviral-miediated gene transfer to hmuan NOD/SCID mouse repopulating cord blood cells through a systematic analysis of protocol variables. Experimental Hematology, 1999. vol. 27, 817425.*
Onodera et al., Development of improved adenosine deaminase retroviral vectors, 1998, Journal of Virology, pp. 1769-1774.*
Hanenberg et al., Colocalization of retrovirus and target cells on specific fibronectin fragments increase genetic transduction of mammalian cells, 1996, Nature Medicine, vol. 2, pp. 876-882.*
Moritz et al., Fibronectin improves transduction of reconstituting hematopoietic stem cells by retroviral vectors: Evidence of direct viral binding to chymotryptic carboxy-terminal fragments, 1996, Blood, vol. 88, pp. 855-862.*
Porter et al., Comparison of efficiency of infection of human gene therapy target cells via four different retroviral receptors, 1996, Human Gene Therapy, vol. 9, pp. 913-919.*
Uchida et al., HIV, but not murine leukemia virus, vectors mediate high efficiency gene transfer into freshly isolated G0/G1 human hematopoietic stem cells, 1998, Proc. Natl. Acad. Sci. USA, vol. 95, pp. 11939-11944.*
Rebel et al., One-day ex vivo culture allows effective gene transfer into human nonobese diabetic/severe combined immune-deficient repopulating cells using high-titer vesicular stomatitis G protein pseudotyped retrovirus, 1999, Blood, vol. 93, pp. 2217-222.*
Moritz et al., Fibronectin improves transduction of reconstituting hematopoietic stem cells by retroviral vectors: Eveidence of direct viral binding to chymotryptic carboxy-terminal fragments, 1996, Blood, vol. 88, pp. 855-862.*
Uchida et al., HIV, but not murine leukemia virus, vectors mediate high efficiency gene transfer into freshly isolated G0/G1 human hematopoietic stem cells, 1998, Proc. Natl. Acad. Sci. USA, vol. 95, pp. 11939-11944.*
Porter et al., Comparison of efficiency of infection of human gene therapy target cells via four different retroviral receptors, 1996, Human Gene Therapy, vol. 7, pp. 913-919.*
Kelly. P. et al.: “Efficient transducticn of CD34+ and CD38− human haematopoietic cells with SClD repopulating cells (SRC) potential with an oncoretroviral virus (RD114) enevlope protein” Blood, vol. 94, No.10 Part 1 Supl.1, Nov. 15, 1999, p. 611a; Abs. 2718, XP002190046.
Hanenberg, H et al.: “Optimization of Fibronectin-Assisted Retroviral Gene Transfer Into Human CD34 + Hematopoietic Cells” Human Gene Therapy, vol. 8, No. 18, Dec. 10, 1997, pp. 2193-2206, XP000867308.
Kelly, P. et al.: “Highly efficient gene transfer into cord blood nonobese diabetic/serve combined immunodeficiency repopulating cells by oncoretroviral vector particles psuedotyped with the feline endogenous retrovirus (RD114) envelope protein” Blood, vol. 96, No. 4 Aug. 15, 2000, pp. 1206-1214, XP002190047.
Hanawa, H. et al.: “Improved transduction of human primitive hematopoietic cells with a lentiviral vector pseudotyped with the envelope protein of endogenous feline leukemia virus (RD114)” Blood, vol. 96, No. 11 Part.1, Nov. 16, 2000, p. 524a, XP002190048.
An Improved Method For Generating Retroviral Producer Clones For Vectors Lacking A Selectable Marker Gene, Derek A. Persons et al., Blood Cells, Mole-cules & Diseases (1998) Vol. 24, pp. 167-182.
High-Titer Packaging Celis Producing Recombinant Retroviruses Resistant to Human Serum, Cosset, Françis-Loic Cosset et al., Journal of Virology, Dec. 1995, vol. 69, No. 12, pp. 7430-7436.
A Stable Human-Derived Packaging Cell Line For Production Of High Titer Retrovirus/-Vesicular Stomatitis Viru G Pseudotypes, Daniel S. Ory, Proc. Natl. Acad. Sci. USA, Oct. 1996, Vol. 93, pp. 11400-11406.
The RD114/Simian Type D. Retrovirus Receptor Is A Neutral Amino Acid Transporter, John E. J. Rasko et al., Pro. Natl. Acad. Sci, USA, Mar. 1999, Vol. 96, pp. 2129-2134.
Envelope-Binding Domain In The Cationic Amino Acid Transporter Determines The Host Range Of Ecotropic Murine Retroviruses- Lorraine M. Aibritton et al., Journal of Virology, Apr. 1993 p2091-2096 vol. 67, No. 4; © 1993 American Society of Microbiology.
Improved Transfer of the Leukocyte Integrin CD18 Subunit Into Hematopoietic Cell Lines by Using Retroviral Vectors Having a Gibbon Ape Leukemia Virus Envelope—Thomas R. Bauer Jr. et al., Blood, Vol. 86 No. 6, Sep. 15, 1995; pp 2379-2387; © 1995 The American Society of Hematology.
Restoration of Lymphocyte Function In Janus Kinase 3-Deficient Mice By Retroviral-Mediated Gene Transfer—Kevin D. Bunting et al., Nature Medicine, vol. 4, No. 1, Jan. 1998.
Lymphocytes As Cellular Vehicles For Gene Therapy In Mouse And Man—Kenneth Culver et al., Pro. Natl. Acad. Sci. USA, vol. 88, No. 8, pp. 3155-3159, Apr. 15, 1991 Medical Sciences.
Efficient Transduction of Human Lymphocytes and CD34+Cells Via Human Immunodefi-ciency Virus-Based Gene Transfer Vectors—Janet Douglas et al., Human Gene Therapy Vol. 10, No. 6, pp935-945; Apr. 10, 1999.
Retrovirally Marked CD34-Enriched Peripheral Blood and Bone Marrow Cells Contribute To Long-Term Engraftment After Autologous Transplantation—Cynthia E. Dunbar et al., Blood, Vol. 85, No. 11, pp 3048-3057, Jun. 1, 1995.
Human Cord Blood CD34+CD38−Cell Transduction Via Lentivirus-Based Gene Transfer Vectors—Jay T. Evans et al., Human Gene Therapy, Vol. 10, No. 9, pp1479-l489, Jun. 10, 1999.
Extended Long-Term Culture Reveals A Highly Quiescent And Primitive Human Hemato-poietic ProgenitorPopulation—Quin-Lin Hao et al., Blood, vol. 88, No. 9, pp3306-3313, Nov. 1, 1996.
Optimization Of Retroviral-Mediated Gene Transfer To Human NOD/SCID Mouse Repopulat-ing Cord Blood Cells Through A Systematic Analysis Of Protocol Variables—Burkhard Hennemann et al., Experimental Hematology vol. 27, pp. 817-825, Jan. 1999, © 1999 International Society For Experimental Hematology.
Human Gene Transfer: Characterization of Human Tumor-Infiltrating Lymphocytes As Vehicles For Retroviral-Mediated Gene Transfer In Man—Attan Kasid et al., Proc. Natl. Acad. Sci. USA, vol. 87, No. 1, pp. 473-477, Jan. 1990.
Efficient Transduction By An Amphotropic Retrovirus Vector Is Dependent On High-Level Expression Of The Cell Surface Virus Receptor—Peter Kurre et al., Journal of Virology, vol. 73, No. 1, pp. 495-500, Jan. 1999.
Retrovirus-Mediated Gene Transfer Into Human CD34+38LowPrimitive Cells Capable Of Reconstituting Long-Term Cultures In Vitro and Nonobese Diabetic-Severe Combined Immunodeficiency Mice ln Vivo&#x
Kelly Patrick F.
Vanin Elio F.
Darby & Darby
Falk Anne-Marie
Qian Celine
St. Jude Children's Research Hospital
LandOfFree
Highly efficient gene transfer into human repopulating stem... does not yet have a rating. At this time, there are no reviews or comments for this patent.
If you have personal experience with Highly efficient gene transfer into human repopulating stem..., we encourage you to share that experience with our LandOfFree.com community. Your opinion is very important and Highly efficient gene transfer into human repopulating stem... will most certainly appreciate the feedback.
Profile ID: LFUS-PAI-O-3504106