Science 3 July 1992: Vol. 257. no. 5066, pp. 99 - 103 DOI: 10.1126/science.1352414

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Interfering RNA-mediated purine analog resistance for in vitro and in vivo cell selection.

C. C. Porter and J. DeGregori (2008)
Blood 112, 4466-4474
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Development of gene therapy for blood disorders.

A. W. Nienhuis (2008)
Blood 111, 4431-4444
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Evidence for a Sav1866-like architecture for the human multidrug transporter P-glycoprotein.

J. K. Zolnerciks, C. Wooding, and K. J. Linton (2007)
FASEB J 21, 3937-3948
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Survival of the fittest: in vivo selection and stem cell gene therapy.

T. Neff, B. C. Beard, and H.-P. Kiem (2006)
Blood 107, 1751-1760
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Polyclonal chemoprotection against temozolomide in a large-animal model of drug resistance gene therapy.

T. Neff, B. C. Beard, L. J. Peterson, P. Anandakumar, J. Thompson, and H.-P. Kiem (2005)
Blood 105, 997-1002
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Cloning and Functional Analysis of the Rhesus Macaque ABCG2 Gene: FORCED EXPRESSION CONFERS AN SP PHENOTYPE AMONG HEMATOPOIETIC STEM CELL PROGENY IN VIVO.

T. Ueda, S. Brenner, H. L. Malech, S. M. Langemeijer, S. Perl, M. Kirby, O. A. Phang, A. E. Krouse, R. E. Donahue, E. M. Kang, et al. (2005)
J. Biol. Chem. 280, 991-998
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Transient in vivo selection of transduced peripheral blood cells using antifolate drug selection in rhesus macaques that received transplants with hematopoietic stem cells expressing dihydrofolate reductase vectors.

D. A. Persons, J. A. Allay, A. Bonifacino, T. Lu, B. Agricola, M. E. Metzger, R. E. Donahue, C. E. Dunbar, and B. P. Sorrentino (2004)
Blood 103, 796-803
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Trimetrexate Inhibits Progression of the Murine 32Dp210 Model of Chronic Myeloid Leukemia in Animals Expressing Drug-resistant Dihydrofolate Reductase.

C. L. Sweeney, J. L. Frandsen, C. M. Verfaillie, and R. S. McIvor (2003)
Cancer Res. 63, 1304-1310
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Side effects of retroviral gene transfer into hematopoietic stem cells.

C. Baum, J. Dullmann, Z. Li, B. Fehse, J. Meyer, D. A. Williams, and C. von Kalle (2003)
Blood 101, 2099-2113
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High-level ectopic HOXB4 expression confers a profound in vivo competitive growth advantage on human cord blood CD34+ cells, but impairs lymphomyeloid differentiation.

B. Schiedlmeier, H. Klump, E. Will, G. Arman-Kalcek, Z. Li, Z. Wang, A. Rimek, J. Friel, C. Baum, and W. Ostertag (2003)
Blood 101, 1759-1768
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Topical colchicine selection of keratinocytes transduced with the multidrug resistance gene (MDR1) can sustain and enhance transgene expression in vivo.

W. Pfutzner, A. Terunuma, C. L. Tock, E. K. Snead, T. M. Kolodka, M. M. Gottesman, L. Taichman, and J. C. Vogel (2002)
PNAS 99, 13096-13101
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Pharmacologically regulated in vivo selection in a large animal.

T. Neff, P. A. Horn, V. E. Valli, A. M. Gown, S. Wardwell, B. L. Wood, C. von Kalle, M. Schmidt, L. J. Peterson, J. C. Morris, et al. (2002)
Blood 100, 2026-2031
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Correction of the murine Wiskott-Aldrich syndrome phenotype by hematopoietic stem cell transplantation.

T. S. Strom, X. Li, J. M. Cunningham, and A. W. Nienhuis (2002)
Blood 99, 4626-4628
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In Vivo Selection of Antifolate-Resistant Transgenic Hematopoietic Stem Cells in a Murine Bone Marrow Transplant Model.

C. A. Warlick, M. D. Diers, J. E. Wagner, and R. S. McIvor (2002)
J. Pharmacol. Exp. Ther. 300, 50-56
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Efficient c-kit Receptor-Targeted Gene Transfer to Primary Human CD34-Selected Hematopoietic Stem Cells.

Q. Zhong, P. Oliver, W. Huang, D. Good, V. La Russa, Z. Zhang, J. R. Cork, R. W. Veith, C. Theodossiou, J. K. Kolls, et al. (2001)
J. Virol. 75, 10393-10400
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The effect of multidrug-resistance 1 gene versus neo transduction on ex vivo and in vivo expansion of rhesus macaque hematopoietic repopulating cells.

S. E. Sellers, J. F. Tisdale, B. A. Agricola, M. E. Metzger, R. E. Donahue, C. E. Dunbar, and B. P. Sorrentino (2001)
Blood 97, 1888-1891
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Methotrexate Accumulates to Similar Levels in Animals Transplanted with Normal versus Drug-resistant Transgenic Marrow.

L. R. Belur, D. Boelk-Galvan, M. D. Diers, R. S. McIvor, and C. L. Zimmerman (2001)
Cancer Res. 61, 1522-1526
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Gene Transfer into Nonhuman Primate Hematopoietic Stem Cells: Implications for Gene Therapy.

Y. Hanazono, K. Terao, and K. Ozawa (2001)
Stem Cells 19, 12-23
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Direct Reversal of DNA Damage by Mutant Methyltransferase Protein Protects Mice against Dose-intensified Chemotherapy and Leads to in Vivo Selection of Hematopoietic Stem Cells.

S. Ragg, M. Xu-Welliver, J. Bailey, M. D’Souza, R. Cooper, S. Chandra, R. Seshadri, A. E. Pegg, and D. A. Williams (2000)
Cancer Res. 60, 5187-5195
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Enforced P-glycoprotein pump function in murine bone marrow cells results in expansion of side population stem cells in vitro and repopulating cells in vivo.

K. D. Bunting, S. Zhou, T. Lu, and B. P. Sorrentino (2000)
Blood 96, 902-909
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Hematopoietic stem cells with controllable tEpoR transgenes have a competitive advantage in bone marrow transplantation.

S. Kirby, W. Walton, and O. Smithies (2000)
Blood 95, 3710-3715
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Limiting numbers of G156A O6-methylguanine-DNA methyltransferase-transduced marrow progenitors repopulate nonmyeloablated mice after drug selection.

B. M. Davis, O. N. Koc, and S. L. Gerson (2000)
Blood 95, 3078-3084
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Retroviral transfer of the hENT2 nucleoside transporter cDNA confers broad-spectrum antifolate resistance in murine bone marrow cells.

D. H. Patel, J. A. Allay, J. A. Belt, and B. P. Sorrentino (2000)
Blood 95, 2356-2363
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Quantitative assessment of retroviral transfer of the human multidrug resistance 1 gene to human mobilized peripheral blood progenitor cells engrafted in nonobese diabetic/severe combined immunodeficient mice.

B. Schiedlmeier, K. Kuhlcke, H. G. Eckert, C. Baum, W. J. Zeller, and S. Fruehauf (2000)
Blood 95, 1237-1248
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Drug-Resistant Variants of Escherichia coli Thymidylate Synthase: Effects of Substitutions at Pro-254.

C. Fantz, D. Shaw, W. Jennings, A. Forsthoefel, M. Kitchens, J. Phan, W. Minor, L. Lebioda, F. G. Berger, and H. T. Spencer (2000)
Mol. Pharmacol. 57, 359-366
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Green Fluorescent Protein as a Selectable Marker of Retrovirally Transduced Hematopoietic Progenitors.

A. Kume, M. Hashiyama, T. Suda, and K. Ozawa (1999)
Stem Cells 17, 226-232
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Clinical Trials Involving Multidrug Resistance Transcription Units in Retroviral Vectors.

A. B. Deisseroth (1999)
Clin. Cancer Res. 5, 1607-1609
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Paclitaxel Chemotherapy after Autologous Stem-Cell Transplantation and Engraftment of Hematopoietic Cells Transduced with a Retrovirus Containing the Multidrug Resistance Complementary DNA (MDR1) in Metastatic Breast Cancer Patients.

K. H. Cowan, J. A. Moscow, H. Huang, J. A. Zujewski, J. O'Shaughnessy, B. Sorrentino, K. Hines, C. Carter, E. Schneider, G. Cusack, et al. (1999)
Clin. Cancer Res. 5, 1619-1628
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Engraftment of MDR1 and NeoR Gene-Transduced Hematopoietic Cells After Breast Cancer Chemotherapy.

J. A. Moscow, H. Huang, C. Carter, K. Hines, J. Zujewski, G. Cusack, C. Chow, D. Venzon, B. Sorrentino, Y. Chiang, et al. (1999)
Blood 94, 52-61
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The Common Marmoset as a Target Preclinical Primate Model for Cytokine and Gene Therapy Studies.

H. Hibino, K. Tani, K. Ikebuchi, M.-S. Wu, H. Sugiyama, Y. Nakazaki, T. Tanabe, S. Takahashi, A. Tojo, S. Suzuki, et al. (1999)
Blood 93, 2839-2848
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Simultaneous Protection of G156A Methylguanine DNA Methyltransferase Gene-transduced Hematopoietic Progenitors and Sensitization of Tumor Cells Using O6-Benzylguanine and Temozolomide.

J. S. Reese, B. M. Davis, L. Liu, and S. L. Gerson (1999)
Clin. Cancer Res. 5, 163-169
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Transduction of Murine Bone Marrow Cells With an MDR1 Vector Enables Ex Vivo Stem Cell Expansion, but These Expanded Grafts Cause a Myeloproliferative Syndrome in Transplanted Mice.

K. D. Bunting, J. Galipeau, D. Topham, E. Benaim, and B. P. Sorrentino (1998)
Blood 92, 2269-2279
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Retroviral Marking of Canine Bone Marrow: Long-Term, High-Level Expression of Human Interleukin-2 Receptor Common Gamma Chain in Canine Lymphocytes.

T. Whitwam, M. E. Haskins, P. S. Henthorn, J. N. Kraszewski, S. E. Kleiman, N. E. Seidel, D. M. Bodine, and J. M. Puck (1998)
Blood 92, 1565-1575
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Targeted expansion of genetically modified bone marrow cells.

L. Jin, N. Siritanaratkul, D. W. Emery, R. E. Richard, K. Kaushansky, T. Papayannopoulou, and C. A. Blau (1998)
PNAS 95, 8093-8097
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Genetic induction of immune tolerance to human clotting factor VIII in a mouse model for hemophilia A.

G. L. Evans and R. A. Morgan (1998)
PNAS 95, 5734-5739
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Isolation and Characterization of Thymitaq (AG337) and 5-Fluoro-2-deoxyuridylate-resistant Mutants of Human Thymidylate Synthase from Ethyl Methanesulfonate-exposed Human Sarcoma HT1080 Cells.

Y. Tong, X. Liu-Chen, E. A. Ercikan-Abali, G. M. Capiaux, S.-C. Zhao, D. Banerjee, and J. R. Bertino (1998)
J. Biol. Chem. 273, 11611-11618
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Biology of Human Umbilical Cord Blood-Derived Hematopoietic Stem/Progenitor Cells.

H. Mayani and P. M. Lansdorp (1998)
Stem Cells 16, 153-165
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Identification of Human and Mouse Hematopoietic Stem Cell Populations Expressing High Levels of mRNA Encoding Retrovirus Receptors.

D. Orlic, L. J. Girard, S. M. Anderson, L. C. Pyle, M. C. Yoder, H. E. Broxmeyer, and D. M. Bodine (1998)
Blood 91, 3247-3254
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Efficient Retroviral-Mediated Gene Transfer to Human Cord Blood Stem Cells With In Vivo Repopulating Potential.

E. Conneally, C.J. Eaves, and R.K. Humphries (1998)
Blood 91, 3487-3493
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Stimulating Cell Proliferation Through the Pharmacologic Activation of c-kit.

L. Jin, H. Asano, and C. Anthony Blau (1998)
Blood 91, 890-897
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Development of a Novel Selective Amplifier Gene for Controllable Expansion of Transduced Hematopoietic Cells.

K. Ito, Y. Ueda, M. Kokubun, M. Urabe, T. Inaba, H. Mano, H. Hamada, T. Kitamura, H. Mizoguchi, T. Sakata, et al. (1997)
Blood 90, 3884-3892
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Long-Term Transfer and Expression of the Human beta -Globin Gene in a Mouse Transplant Model.

H. Raftopoulos, M. Ward, P. Leboulch, and A. Bank (1997)
Blood 90, 3414-3422
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Sensitization of Hematopoietic Stem and Progenitor Cells to Trimetrexate Using Nucleoside Transport Inhibitors.

J. A. Allay, H. T. Spencer, S. L. Wilkinson, J. A. Belt, R. L. Blakley, and B. P. Sorrentino (1997)
Blood 90, 3546-3554
 |  Abstract »  |  Full Text »  |  PDF »

Retroviral-Mediated Transfer of the Green Fluorescent Protein Gene Into Murine Hematopoietic Cells Facilitates Scoring and Selection of Transduced Progenitors In Vitro and Identification of Genetically Modified Cells In Vivo.

D. A. Persons, J. A. Allay, E. R. Allay, R. J. Smeyne, R. A. Ashmun, B. P. Sorrentino, and A. W. Nienhuis (1997)
Blood 90, 1777-1786
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Cytokine-Facilitated Transduction Leads to Low-Level Engraftment in Nonablated Hosts.

E. L.W. Kittler, S. O. Peters, R. B. Crittenden, M. E. Debatis, H. S. Ramshaw, F. M. Stewart, and P. J. Quesenberry (1997)
Blood 90, 865-872
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A proliferation switch for genetically modified cells.

C. A. Blau, K. R. Peterson, J. G. Drachman, and D. M. Spencer (1997)
PNAS 94, 3076-3081
 |  Abstract »  |  Full Text »  |  PDF »

In Vivo Drug-Selectable Genes: A New Concept in Gene Therapy.

T. Licht, F. Herrmann, M.M. Gottesman, and I. Pastan (1997)
Stem Cells 15, 104-111
 |  Abstract »  |  Full Text »

Cytokine Prestimulation as a Gene Therapy Strategy: Implications for Using the MDR1 Gene as a Dominant Selectable Marker.

C. A. Blau, T. Neff, and T. Papayannopoulou (1997)
Blood 89, 146-154
 |  Abstract »  |  Full Text »  |  PDF »

Retroviral transduction of a mutant methylguanine DNA methyltransferase gene into human CD34 cells confers resistance to O6-benzylguanine plus 1,3-bis(2-chloroethyl)-1-nitrosourea.

J. S. Reese, O. N. Koc, K. M. Lee, L. Liu, J. A. Allay, W. P. Phillips Jr., and S. L. Gerson (1996)
PNAS 93, 14088-14093
 |  Abstract »  |  Full Text »  |  PDF »

Peripheral blood progenitor cell transplantation: a replacement for marrow auto- or allografts.

M Korbling and R Champlin (1996)
Stem Cells 14, 185-195
 |  Abstract »

The Molecular Basis of Leukemia.

M. J. Cline (1994)
N. Engl. J. Med. 330, 328-336
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Exploiting Multidrug Resistance to Treat Cancer.

M.M. Gottesman, S.V. Ambudkar, B. Ni, J.M. Aran, Y. Sugimoto, C.O. Cardarelli, and I. Pastan (1994)
Cold Spring Harb Symp Quant Biol 59, 677-683
 |  Abstract »  |  PDF »

The basic science of gene therapy.

R. Mulligan (1993)
Science 260, 926-932
 |  Abstract »  |  PDF »

Drug selection with paclitaxel restores expression of linked IL-2 receptor gamma -chain and multidrug resistance (MDR1) transgenes in canine bone marrow.

T. Licht, M. Haskins, P. Henthorn, S. E. Kleiman, D. M. Bodine, T. Whitwam, J. M. Puck, M. M. Gottesman, and J. R. Melniczek (2002)
PNAS 99, 3123-3128
 |  Abstract »  |  Full Text »  |  PDF »

Preselective gene therapy for Fabry disease.

G. Qin, T. Takenaka, K. Telsch, L. Kelley, T. Howard, T. Levade, R. Deans, B. H. Howard, H. L. Malech, R. O. Brady, et al. (2001)
PNAS 98, 3428-3433
 |  Abstract »  |  Full Text »  |  PDF »

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