Note to users. If you're seeing this message, it means that your browser cannot find this page's style/presentation instructions -- or possibly that you are using a browser that does not support current Web standards. Find out more about why this message is appearing, and what you can do to make your experience of our site the best it can be.

Site Tools

  • AAAS
  • Subscribe
  • Feedback

Site Search

Search Advanced

Science 20 October 1995:
Vol. 270. no. 5235, pp. 404 - 410
DOI: 10.1126/science.270.5235.404
Prev | Table of Contents | Next

Articles

Transfer of Genes to Humans: Early Lessons and Obstacles to Success

Ronald G. Crystal

Enough information has been gained from clinical trials to allow the conclusion that human gene transfer is feasible, can evoke biologic responses that are relevant to human disease, and can provide important insights into human biology. Adverse events have been uncommon and have been related to the gene delivery strategies, not to the genetic material being transferred. Human gene transfer still faces significant hurdles before it becomes an established therapeutic strategy. However, its accomplishments to date are impressive, and the logic of the potential usefulness of this clinical paradigm continues to be compelling.


The author is professor of medicine and chief of the Division of Pulmonary and Critical Care Medicine, The New York Hospital-Cornell Medical Center, 520 East 70th Street, ST505, New York, NY 10021, USA.


THIS ARTICLE HAS BEEN CITED BY OTHER ARTICLES:
Antiviral Antibodies Target Adenovirus to Phagolysosomes and Amplify the Innate Immune Response.
A. K. Zaiss, A. Vilaysane, M. J. Cotter, S. A. Clark, H. C. Meijndert, P. Colarusso, R. M. Yates, V. Petrilli, J. Tschopp, and D. A. Muruve (2009)
J. Immunol. 182, 7058-7068
   Abstract »    Full Text »    PDF »
Efficacy of Therapeutic Angiogenesis by Intramyocardial Injection of pCK-VEGF165 in Pigs.
J.-S. Choi, K.-B. Kim, W. Han, D. S. Kim, J. S. Park, J. J. Lee, and D. S. Lee (2006)
Ann. Thorac. Surg. 82, 679-686
   Abstract »    Full Text »    PDF »
Gene therapy and uterine leiomyoma: a review.
A. Al-Hendy and S. Salama (2006)
Hum. Reprod. Update 12, 385-400
   Abstract »    Full Text »    PDF »
A Novel Method for Viral Gene Delivery in Solid Tumors.
Y. Wang, S. Liu, C.-Y. Li, and F. Yuan (2005)
Cancer Res. 65, 7541-7545
   Abstract »    Full Text »    PDF »
Recipient intramuscular cotransfection of transforming growth factor {beta}1 and interleukin 10 ameliorates acute lung graft rejection.
T. Suda, N. Daddi, T. Tagawa, S. A. Kanaan, B. D. Kozower, J. H. Ritter, and G. A. Patterson (2005)
J. Thorac. Cardiovasc. Surg. 129, 926-931
   Abstract »    Full Text »    PDF »
Lineage Differentiation-Associated Loss of Adenoviral Susceptibility and Coxsackie-Adenovirus Receptor Expression in Human Mesenchymal Stem Cells.
S.-C. Hung, C.-Y. Lu, S.-K. Shyue, H.-C. Liu, and L. L.-T. Ho (2004)
Stem Cells 22, 1321-1329
   Abstract »    Full Text »    PDF »
Therapeutic angiogenesis for critical limb ischemia: design of the hepatocyte growth factor therapeutic angiogenesis clinical trial.
R. J Powell, J. Dormandy, M. Simons, R. Morishita, and B. H Annex (2004)
Vascular Medicine 9, 193-198
   Abstract »    PDF »
Antisense Thymidylate Synthase Electrogene Transfer to Increase Uptake of Radiolabeled Iododeoxyuridine in a Murine Model.
K.-H. Chi, H.-E. Wang, Y.-S. Wang, S.-L. Chou, H.-M. Yu, Y.-H. Tseng, I.-M. Hwang, and W.-Y. Lui (2004)
J. Nucl. Med. 45, 478-484
   Abstract »    Full Text »
Adenovirus-mediated wild-type p53 radiosensitizes human tumor cells by suppressing DNA repair capacity.
N. K. Sah, A. Munshi, T. Nishikawa, T. Mukhopadhyay, J. A. Roth, and R. E. Meyn (2003)
Mol. Cancer Ther. 2, 1223-1231
   Abstract »    Full Text »    PDF »
Systemic dissemination of viral vectors during intratumoral injection.
Y. Wang, J. K. Hu, A. Krol, Y.-P. Li, C.-Y. Li, and F. Yuan (2003)
Mol. Cancer Ther. 2, 1233-1242
   Abstract »    Full Text »    PDF »
Adenovirus encoding soluble tumor necrosis factor {alpha} receptor immunoglobulin prolongs gene expression of a cotransfected reporter gene in rat lung.
T. Suda, T. Tagawa, S. A. Kanaan, B. D. Kozower, N. Daddi, T. Mohanakumar, and G. A. Patterson (2003)
J. Thorac. Cardiovasc. Surg. 126, 1155-1161
   Abstract »    Full Text »    PDF »
Molecular Basis of Canavan's Disease: From Human to Mouse.
S. Surendran, K. M. Matalon, S. K. Tyring, and R. Matalon (2003)
J Child Neurol 18, 604-610
   Abstract »    PDF »
Imaging of Vascular Gene Therapy.
X. Yang (2003)
Radiology 228, 36-49
   Abstract »    Full Text »    PDF »
Advanced modular self-inactivating lentiviral expression vectors for multigene interventions in mammalian cells and in vivo transduction.
B. Mitta, M. Rimann, M. U. Ehrengruber, M. Ehrbar, V. Djonov, J. Kelm, and M. Fussenegger (2002)
Nucleic Acids Res. 30, e113
   Abstract »    Full Text »    PDF »
Mechanism of Restriction of Normal and Cystic Fibrosis Transmembrane Conductance Regulator-Deficient Human Tracheal Gland Cells to Adenovirus Infection and Ad-Mediated Gene Transfer.
F. Gaden, L. Franqueville, S. S. Hong, V. Legrand, C. Figarella, and P. Boulanger (2002)
Am. J. Respir. Cell Mol. Biol. 27, 628-640
   Abstract »    Full Text »    PDF »
Gene Therapy of Erectile Dysfunction in the Rat with Penile Neuronal Nitric Oxide Synthase.
T. R. Magee, M. Ferrini, H. J. Garban, D. Vernet, K. Mitani, J. Rajfer, and N. F. Gonzalez-Cadavid (2002)
Biol Reprod 67, 20-28
   Abstract »    Full Text »    PDF »
The Role of Growth Factors in the Repair of Bone : Biology and Clinical Applications.
J. R. Lieberman, A. Daluiski, and T. A. Einhorn (2002)
J. Bone Joint Surg. Am. 84, 1032-1044
   Full Text »    PDF »
Preexisting Immunity to Adenovirus in Rhesus Monkeys Fails To Prevent Vector-Induced Toxicity.
A. N. Varnavski, Y. Zhang, M. Schnell, J. Tazelaar, J.-P. Louboutin, Q.-C. Yu, A. Bagg, G.-p. Gao, and J. M. Wilson (2002)
J. Virol. 76, 5711-5719
   Abstract »    Full Text »    PDF »
Transcriptional Targeting of Lentiviral Vectors by Long Terminal Repeat Enhancer Replacement.
F. Lotti, E. Menguzzato, C. Rossi, L. Naldini, L. Ailles, F. Mavilio, and G. Ferrari (2002)
J. Virol. 76, 3996-4007
   Abstract »    Full Text »    PDF »
Adenovirus-mediated gene transfer to nonparenchymal cells in normal and injured liver.
Q. Yu, L. G. Que, and D. C. Rockey (2002)
Am J Physiol Gastrointest Liver Physiol 282, G565-G572
   Abstract »    Full Text »    PDF »
Activation of p38 and ERK Signaling during Adenovirus Vector Cell Entry Lead to Expression of the C-X-C Chemokine IP-10.
L. A. Tibbles, J. C. L. Spurrell, G. P. Bowen, Q. Liu, M. Lam, A. K. Zaiss, S. M. Robbins, M. D. Hollenberg, T. J. Wickham, and D. A. Muruve (2002)
J. Virol. 76, 1559-1568
   Abstract »    Full Text »    PDF »
Direct visualization at the single-cell level of electrically mediated gene delivery.
M. Golzio, J. Teissie, and M.-P. Rols (2002)
PNAS
   Abstract »    Full Text »    PDF »
The Role of Natural Killer Cells in Adenovirus-mediated p53 Gene Therapy.
J. L. Carroll, L. L. Nielsen, S. B. Pruett, and J. M. Mathis (2001)
Mol. Cancer Ther. 1, 49-60
   Abstract »    Full Text »    PDF »
Anticancer Effect of a Lentiviral Vector Capable of Expressing HIV-1 Vpr.
S. Pang, M. K. Kang, S. Kung, D. Yu, A. Lee, B. Poon, I. S. Y. Chen, B. Lindemann, and N.-H. Park (2001)
Clin. Cancer Res. 7, 3567-3573
   Abstract »    Full Text »    PDF »
Biology of E1-Deleted Adenovirus Vectors in Nonhuman Primate Muscle.
P. W. Zoltick, N. Chirmule, M. A. Schnell, G.-P. Gao, J. V. Hughes, and J. M. Wilson (2001)
J. Virol. 75, 5222-5229
   Abstract »    Full Text »
Electroporation-Mediated Gene Transfer that Targets Glomeruli.
M. TSUJIE, Y. ISAKA, H. NAKAMURA, E. IMAI, and M. HORI (2001)
J. Am. Soc. Nephrol. 12, 949-954
   Abstract »    Full Text »
Branched co-polymers of histidine and lysine are efficient carriers of plasmids.
Q.-R. Chen, L. Zhang, S. A. Stass, and A. J. Mixson (2001)
Nucleic Acids Res. 29, 1334-1340
   Abstract »    Full Text »    PDF »
Research Opportunities and Advances in Lung Disease.
R. G. Crystal (2001)
JAMA 285, 612-618
   Abstract »    Full Text »    PDF »
An Agrobacterium VirE2 channel for transferred-DNA transport into plant cells.
F. Dumas, M. Duckely, P. Pelczar, P. Van Gelder, and B. Hohn (2001)
PNAS
   Abstract »    Full Text »
Prospects for Re-engineering Salivary Glands.
B.J. Baum (2000)
Advances in Dental Research 14, 84-88
   Abstract »    PDF »
In Vivo Demethylation of a MoMuLV Retroviral Vector Expressing the Herpes Simplex Thymidine Kinase Suicide Gene by 5' Azacytidine.
M. Di Ianni, A. Terenzi, S. Di Florio, G. Venditti, R. Benedetti, A. Santucci, A. Bartoli, K. Fettucciari, P. Marconi, R. Rossi, et al. (2000)
Stem Cells 18, 415-421
   Abstract »    Full Text »
Assessment of efficiency and safety of adenovirus mediated gene transfer into normal and damaged murine livers.
T Nakatani, S Kuriyama, K Tominaga, T Tsujimoto, A Mitoro, M Yamazaki, H Tsujinoue, H Yoshiji, S Nagao, and H Fukui (2000)
Gut 47, 563-570
   Abstract »    Full Text »    PDF »
Percutaneous in vivo gene transfer to the peripheral lungs using plasmid-liposome complexes.
H. Saito, H. Nakamura, S. Kato, S. Inoue, M. Inage, M. Ito, and H. Tomoike (2000)
Am J Physiol Lung Cell Mol Physiol 279, L651-L657
   Abstract »    Full Text »    PDF »
The 17 Nucleotides Downstream from the env Gene Stop Codon Are Important for Murine Leukemia Virus Packaging.
S. Shin Yu, J.-M. Kim, and S. Kim (2000)
J. Virol. 74, 8775-8780
   Abstract »    Full Text »
Dendritic cells modified to express CD40 ligand elicit therapeutic immunity against preexisting murine tumors.
T. Kikuchi, M. A. S. Moore, and R. G. Crystal (2000)
Blood 96, 91-99
   Abstract »    Full Text »    PDF »
Cancer Gene Therapy by Thyroid Hormone-mediated Expression of Toxin Genes.
V. Martín, M. L. Cortés, Pablo de Felipe, A. Farsetti, N. B. Calcaterra, and M. Izquierdo (2000)
Cancer Res. 60, 3218-3224
   Abstract »    Full Text »
Adenovirus Vector-Induced Expression of the C-X-C Chemokine IP-10 Is Mediated through Capsid-Dependent Activation of NF-kappa B.
S. L. Borgland, G. P. Bowen, N. C. W. Wong, T. A. Libermann, and D. A. Muruve (2000)
J. Virol. 74, 3941-3947
   Abstract »    Full Text »
Development of lentiviral vectors for gene therapy for human diseases.
G. L. Buchschacher Jr and F. Wong-Staal (2000)
Blood 95, 2499-2504
   Abstract »    Full Text »    PDF »
Readministration of Adenovirus Vector in Nonhuman Primate Lungs by Blockade of CD40-CD40 Ligand Interactions.
N. Chirmule, S. E. Raper, L. Burkly, D. Thomas, J. Tazelaar, J. V. Hughes, and J. M. Wilson (2000)
J. Virol. 74, 3345-3352
   Abstract »    Full Text »
Toward a More Accurate Quantitation of the Activity of Recombinant Retroviruses: Alternatives to Titer and Multiplicity of Infection.
S. Andreadis, T. Lavery, H. E. Davis, J. M. Le Doux, M. L. Yarmush, and J. R. Morgan (2000)
J. Virol. 74, 1258-1266
   Abstract »    Full Text »
IN VIVO ADENOVIRUS-MEDIATED ENDOTHELIAL NITRIC OXIDE SYNTHASE GENE TRANSFER AMELIORATES LUNG ALLOGRAFT ISCHEMIA-REPERFUSION INJURY.
T. Suda, B. N. Mora, F. D’Ovidio, J. A. Cooper, M. Hiratsuka, W. Zhang, T. Mohanakumar, and G. A. Patterson (2000)
J. Thorac. Cardiovasc. Surg. 119, 297-304
   Abstract »    Full Text »    PDF »
Interleukin-7/B7.1-encoding Adenoviruses Induce Rejection of Transplanted but not Nontransplanted Tumors.
G. Willimsky and T. Blankenstein (2000)
Cancer Res. 60, 685-692
   Abstract »    Full Text »
Involvement of Rb family proteins, focal adhesion proteins and protein synthesis in senescent morphogenesis induced by hydrogen peroxide.
Q. Chen, V. Tu, J Catania, M Burton, O Toussaint, and T Dilley (2000)
J. Cell Sci. 113, 4087-4097
   Abstract »    PDF »
Highly efficient electro-gene therapy of solid tumor by using an expression plasmid for the herpes simplex virus thymidine kinase gene.
T. Goto, T. Nishi, T. Tamura, S. B. Dev, H. Takeshima, M. Kochi, K. Yoshizato, J.-i. Kuratsu, T. Sakata, G. A. Hofmann, et al. (2000)
PNAS 97, 354-359
   Abstract »    Full Text »    PDF »
Focal angiogen therapy using intramyocardial delivery of an adenovirus vector coding for vascular endothelial growth factor 121.
L. Y. Lee, S. R. Patel, N. R. Hackett, C. A. Mack, D. R. Polce, T. El-Sawy, R. Hachamovitch, P. Zanzonico, T. A. Sanborn, M. Parikh, et al. (2000)
Ann. Thorac. Surg. 69, 14-23
   Abstract »    Full Text »    PDF »
Induction of Endogenous Genes following Infection of Human Endothelial Cells with an E1- E4+ Adenovirus Gene Transfer Vector.
R. Ramalingam, S. Rafii, S. Worgall, N. R. Hackett, and R. G. Crystal (1999)
J. Virol. 73, 10183-10190
   Abstract »    Full Text »
Correction of X-Linked Immunodeficient Mice by Competitive Reconstitution With Limiting Numbers of Normal Bone Marrow Cells.
J. Rohrer and M. E. Conley (1999)
Blood 94, 3358-3365
   Abstract »    Full Text »    PDF »
Replication-Defective Bovine Adenovirus Type 3 as an Expression Vector.
P. S. Reddy, N. Idamakanti, Y. Chen, T. Whale, L. A. Babiuk, M. Mehtali, and S. K. Tikoo (1999)
J. Virol. 73, 9137-9144
   Abstract »    Full Text »    PDF »
An Inducible Human Immunodeficiency Virus Type 1 (HIV-1) Vector Which Effectively Suppresses HIV-1 Replication.
D. S. An, K. Morizono, Q.-X. Li, S. H. Mao, S. Lu, and I. S. Y. Chen (1999)
J. Virol. 73, 7671-7677
   Abstract »    Full Text »
Redirected infection of directly biotinylated recombinant adenovirus vectors through cell surface receptors and antigens.
J. S. Smith, J. R. Keller, N. C. Lohrey, C. S. McCauslin, M. Ortiz, K. Cowan, and S. E. Spence (1999)
PNAS 96, 8855-8860
   Abstract »    Full Text »    PDF »
Ovine Adenovirus Vectors Overcome Preexisting Humoral Immunity against Human Adenoviruses In Vivo.
C. Hofmann, P. Löser, G. Cichon, W. Arnold, G. W. Both, and M. Strauss (1999)
J. Virol. 73, 6930-6936
   Abstract »    Full Text »
Paradoxical Effects of Adenovirus-Mediated Blockade of TNF Activity in Murine Collagen-Induced Arthritis.
E. Quattrocchi, M. Walmsley, K. Browne, R. O. Williams, L. Marinova-Mutafchieva, W. Buurman, D. M. Butler, and M. Feldmann (1999)
J. Immunol. 163, 1000-1009
   Abstract »    Full Text »    PDF »
Intracellular Accumulation of Secreted Proteoglycans Inhibits Cationic Lipid-mediated Gene Transfer. CO-TRANSFER OF GLYCOSAMINOGLYCANS TO THE NUCLEUS.
M. Belting and P. Petersson (1999)
J. Biol. Chem. 274, 19375-19382
   Abstract »    Full Text »    PDF »
Fiber Swap between Adenovirus Subgroups B and C Alters Intracellular Trafficking of Adenovirus Gene Transfer Vectors.
N. Miyazawa, P. L. Leopold, N. R. Hackett, B. Ferris, S. Worgall, E. Falck-Pedersen, and R. G. Crystal (1999)
J. Virol. 73, 6056-6065
   Abstract »    Full Text »
Frequency and Stability of Chromosomal Integration of Adenovirus Vectors.
A. Harui, S. Suzuki, S. Kochanek, and K. Mitani (1999)
J. Virol. 73, 6141-6146
   Abstract »    Full Text »
The Effect of Regional Gene Therapy with Bone Morphogenetic Protein-2-Producing Bone-Marrow Cells on the Repair of Segmental Femoral Defects in Rats.
J. R. LIEBERMAN, A. DALUISKI, S. STEVENSON, L. JOLLA, L. WU, P. McALLISTER, Y. P. LEE, J. M. KABO, G. A.M. FINERMAN, A. J. BERK, et al. (1999)
J. Bone Joint Surg. Am. 81, 905-17
   Abstract »    Full Text »
Repeated Administration of Adenoviral Vectors in Lungs of Human CD4 Transgenic Mice Treated with a Nondepleting CD4 Antibody.
N. Chirmule, A. Truneh, S. E. Haecker, J. Tazelaar, G.-p. Gao, S. E. Raper, J. V. Hughes, and J. M. Wilson (1999)
J. Immunol. 163, 448-455
   Abstract »    Full Text »    PDF »
Passive entry of a DNA molecule into a small pore.
P.-G. de Gennes (1999)
PNAS 96, 7262-7264
   Abstract »    Full Text »    PDF »
Engineering Viral Vectors to Subvert the Airway Defense Response.
D. C. Look and S. L. Brody (1999)
Am. J. Respir. Cell Mol. Biol. 20, 1103-1106
   Full Text »
Transcriptional Targeting of Retroviral Vectors to the Erythroblastic Progeny of Transduced Hematopoietic Stem Cells.
A. Grande, B. Piovani, A. Aiuti, S. Ottolenghi, F. Mavilio, and G. Ferrari (1999)
Blood 93, 3276-3285
   Abstract »    Full Text »    PDF »
Enhanced Efficiency of Lactosylated Poly-L-Lysine-Mediated Gene Transfer into Cystic Fibrosis Airway Epithelial Cells.
W. J. W. Kollen, F. M. Schembri, G. J. Gerwig, J. F. G. Vliegenthart, M. C. Glick, and T. F. Scanlin (1999)
Am. J. Respir. Cell Mol. Biol. 20, 1081-1086
   Abstract »    Full Text »
Gene transfer into rat heart-derived endothelial cells.
M. Hein, M. Ernst, F. Moller, and D. Regensburger (1999)
Eur. J. Cardiothorac. Surg. 13, 460-466
   Abstract »    Full Text »    PDF »
Molecular advances in cardiac and cardiovascular disease.
A. Basile-Borgia, J. H Abel, and H. Mahloogi (1999)
Perfusion 14, 89-99
   Abstract »    PDF »
Modification of the Genetic Program of Human Alveolar Macrophages by Adenovirus Vectors In Vitro Is Feasible but Inefficient, Limited in Part by the Low Level of Expression of the Coxsackie/Adenovirus Receptor.
R. J. Kaner, S. Worgall, P. L. Leopold, E. Stolze, E. Milano, C. Hidaka, R. Ramalingam, N. R. Hackett, R. Singh, J. Bergelson, et al. (1999)
Am. J. Respir. Cell Mol. Biol. 20, 361-370
   Abstract »    Full Text »
Fiberless Recombinant Adenoviruses: Virus Maturation and Infectivity in the Absence of Fiber.
V. Legrand, D. Spehner, Y. Schlesinger, N. Settelen, A. Pavirani, and M. Mehtali (1999)
J. Virol. 73, 907-919
   Abstract »    Full Text »
The endocrine secretion of mammalian digestive enzymes by exocrine glands.
L. Isenman, C. Liebow, and S. Rothman (1999)
Am J Physiol Endocrinol Metab 276, E223-E232
   Abstract »    Full Text »    PDF »
Selective Expansion of Alveolar Macrophages In Vivo by Adenovirus-Mediated Transfer of the Murine Granulocyte-Macrophage Colony-Stimulating Factor cDNA.
S. Worgall, R. Singh, P. L. Leopold, R. J. Kaner, N. R. Hackett, N. Topf, M. A.S. Moore, and R. G. Crystal (1999)
Blood 93, 655-666
   Abstract »    Full Text »    PDF »
In Vivo Gene Transfer To Salivary Glands.
B.J. Baum and B.C. O'Connell (1999)
Critical Reviews in Oral Biology & Medicine 10, 276-283
   Abstract »    Full Text »    PDF »
Gene transfer in the kidney.
V. R. Kelley and V. P. Sukhatme (1999)
Am J Physiol Renal Physiol 276, F1-F9
   Abstract »    Full Text »    PDF »
Is Gene Therapy for Hypertension Possible?.
M. I. Phillips (1999)
Hypertension 33, 8-13
   Full Text »    PDF »
Correction of murine galactosialidosis by bone marrow-derived macrophages overexpressing human protective protein/cathepsin A under control of the colony-stimulating factor-1 receptor promoter.
C. N. Hahn, M. del Pilar Martin, X.-Y. Zhou, L. W. Mann, and A. d'Azzo (1998)
PNAS 95, 14880-14885
   Abstract »    Full Text »    PDF »
Long-Term Contribution to the Myeloid Compartment by Lineage-Committed Stem Cells.
C.-C. Chen, A. Rivera, N. Ron, N. Sutkowski, J. P. Dougherty, and Y. Ron (1998)
Blood 92, 3210-3217
   Abstract »    Full Text »    PDF »
Development of a Self-Inactivating Lentivirus Vector.
H. Miyoshi, U. Blomer, M. Takahashi, F. H. Gage, and I. M. Verma (1998)
J. Virol. 72, 8150-8157
   Abstract »    Full Text »    PDF »
Liposome-mediated transfection of fetal lung epithelial cells: DNA degradation and enhanced superoxide toxicity.
A. K. Tanswell, O. Staub, R. Iles, R. Belcastro, J. Cabacungan, L. Sedlackova, B. Steer, Y. Wen, J. Hu, and H. O'Brodovich (1998)
Am J Physiol Lung Cell Mol Physiol 275, L452-L460
   Abstract »    Full Text »    PDF »
Nucleic Acid Therapeutics: State of the Art and Future Prospects.
A. M. Gewirtz, D. L. Sokol, and M. Z. Ratajczak (1998)
Blood 92, 712-736
   Full Text »    PDF »
Intermittent, Repetitive Corticosteroid-Induced Upregulation of Platelet Levels After Adenovirus-Mediated Transfer to the Liver of a Chimeric Glucocorticoid-Responsive Promoter Controlling the Thrombopoietin cDNA.
K. Narumi, M. Suzuki, W. Song, M. A.S. Moore, and R. G. Crystal (1998)
Blood 92, 822-833
   Abstract »    Full Text »    PDF »
Sustained Gene Expression in Retrovirally Transduced, Engrafting Human Hematopoietic Stem Cells and Their Lympho-Myeloid Progeny.
L. Cheng, C. Du, C. Lavau, S. Chen, J. Tong, B. P. Chen, R. Scollay, R. G. Hawley, and B. Hill (1998)
Blood 92, 83-92
   Abstract »    Full Text »    PDF »
High-Efficiency Gene Transfer into Normal and Adenosine Deaminase-Deficient T Lymphocytes Is Mediated by Transduction on Recombinant Fibronectin Fragments.
K. E. Pollok, H. Hanenberg, T. W. Noblitt, W. L. Schroeder, I. Kato, D. Emanuel, and D. A. Williams (1998)
J. Virol. 72, 4882-4892
   Abstract »    Full Text »    PDF »
Transduction of Dendritic Cells by DNA Viral Vectors Directs the Immune Response to Transgene Products in Muscle Fibers.
K. Jooss, Y. Yang, K. J. Fisher, and J. M. Wilson (1998)
J. Virol. 72, 4212-4223
   Abstract »    Full Text »    PDF »
High-Efficiency Gene Transfer Into Ex Vivo Expanded Human Hematopoietic Progenitors and Precursor Cells by Adenovirus Vectors.
B. M. Frey, N. R. Hackett, J. M. Bergelson, R. Finberg, R. G. Crystal, M. A.S. Moore, and S. Rafii (1998)
Blood 91, 2781-2792
   Abstract »    Full Text »    PDF »
Research on Cystic Fibrosis . A Journey from the Heart House.
M. J. WELSH and B. W. RAMSEY (1998)
Am. J. Respir. Crit. Care Med. 157, S148-S154
   Full Text »    PDF »
Polyethylenimine but Not Cationic Lipids Promotes Transgene Delivery to the Nucleus in Mammalian Cells.
H. Pollard, J.-S. Remy, G. Loussouarn, S. Demolombe, J.-P. Behr, and D. Escande (1998)
J. Biol. Chem. 273, 7507-7511
   Abstract »    Full Text »    PDF »
The Emerging Technology and Application of Preimplantation Genetic Diagnosis.
R. J. Tasca and M. E. McClure (1998)
J. Law Med. Ethics 26, 7-16
   PDF »
Adenovirally Mediated Gene Transfer of Functional Human Tissue-type Plasminogen Activator to Murine Lungs.
W. L. Simmons, K. E. Rivera, D. T. Curiel, W. F. Williams, and M. A. Olman (1998)
Am. J. Respir. Cell Mol. Biol. 18, 307-314
   Abstract »    Full Text »
Biologic bypass with the use of adenovirus-mediated gene transfer of the complementary deoxyribonucleic acid for vascular endothelial growth factor 121 improves myocardial perfusion and function in the ischemic porcine heart.
C. A. Mack, S. R. Patel, E. A. Schwarz, P. Zanzonico, R. T. Hahn, A. Ilercil, R. B. Devereux, S. J. Goldsmith, T. F. Christian, T. A. Sanborn, et al. (1998)
J. Thorac. Cardiovasc. Surg. 115, 168-177
   Abstract »    Full Text »    PDF »
Development of an epithelium-specific expression cassette with human DNA regulatory elements for transgene expression in lung airways.
Y.-H. Chow, H. O'Brodovich, J. Plumb, Y. Wen, K.-J. Sohn, Z. Lu, F. Zhang, G. L. Lukacs, A. K. Tanswell, C.-C. Hui, et al. (1997)
PNAS 94, 14695-14700
   Abstract »    Full Text »    PDF »
Transfection by Cationic Liposomes Using Simultaneous Single Cell Measurements of Plasmid Delivery and Transgene Expression.
W.-C. Tseng, Frederick. R. Haselton, and T. D. Giorgio (1997)
J. Biol. Chem. 272, 25641-25647
   Abstract »    Full Text »    PDF »
Gene therapy for collateral vessel development.
G. Melillo, M. Scoccianti, I. Kovesdi, J. Safi Jr, T. Riccioni, and M. C Capogrossi (1997)
Cardiovasc Res 35, 480-489
   Abstract »    Full Text »    PDF »
Glucose-Stimulated Genes and Prospects of Gene Therapy for Type I Diabetes.
D. Mitanchez, B. Doiron, R. Chen, and A. Kahn (1997)
Endocr. Rev. 18, 520-540
   Abstract »    Full Text »    PDF »
Ex Vivo Adenoviral-Mediated Gene Transfer to Lung Isografts During Cold Preservation.
C. H. R. Boasquevisque, B. N. Mora, R. A. Schmid, T. C. Lee, I. Nagahiro, J. D. Cooper, and G. A. Patterson (1997)
Ann. Thorac. Surg. 63, 1556-1560
   Abstract »    Full Text »
Manipulating Angiogenesis: From Basic Science to the Bedside.
M. S. Pepper (1997)
Arterioscler. Thromb. Vasc. Biol. 17, 605-619
   Abstract »    Full Text »
High-Level Globin Gene Expression Mediated by a Recombinant Adeno-Associated Virus Genome That Contains the 3' gamma  Globin Gene Regulatory Element and Integrates as Tandem Copies in Erythroid Cells.
P. W. Hargrove, E. F. Vanin, G. J. Kurtzman, and A. W. Nienhuis (1997)
Blood 89, 2167-2175
   Abstract »    Full Text »    PDF »
Gene transfer by guanidinium-cholesterol cationic lipids into airway epithelial cells in vitro and in vivo.
N. Oudrhiri, J.-P. Vigneron, M. Peuchmaur, T. Leclerc, J.-M. Lehn, and P. Lehn (1997)
PNAS 94, 1651-1656
   Abstract »    Full Text »    PDF »
Structure of DNA-Cationic Liposome Complexes: DNA Intercalation in Multilamellar Membranes in Distinct Interhelical Packing Regimes.
J. O. Rädler, I. Koltover, T. Salditt, and C. R. Safinya (1997)
Science 275, 810-814
   Abstract »    Full Text »
Topical Review: Gene Therapy for Neurologic Disease: Benchtop Discoveries to Bedside Applications. 2. The Bedside.
B. L. Maria, C. D. Medina, K. B.N. Hoang, and M. Ian Phillips (1997)
J Child Neurol 12, 77-84
   Abstract »    PDF »
Topical Review: Gene Therapy for Neurologic Disease: Benchtop Discoveries to Bedside Applications. 1. The Bench.
B. L. Maria, C. D. Medina, K. B.N. Hoang, and M. I. Phillips (1997)
J Child Neurol 12, 1-12
   Abstract »    PDF »
Transduction of nondividing cells using pseudotyped defective high-titer HIV type 1 particles.
J. Reiser, G. Harmison, S. Kluepfel-Stahl, R. O. Brady, S. Karlsson, and M. Schubert (1996)
PNAS 93, 15266-15271
   Abstract »    Full Text »    PDF »
Direct In Vivo Gene Transfer to Canine Myocardium Using a Replication-Deficient Adenovirus Vector.
C. J. Magovern, C. A. Mack, J. Zhang, R. T. Hahn, W. Ko, O. W. Isom, R. G. Crystal, and T. K. Rosengart (1996)
Ann. Thorac. Surg. 62, 425-433
   Abstract »    Full Text »
Somatic gene therapy.
J. Dorin (1996)
BMJ 312, 323-324
   Full Text »


To Advertise     Find Products

Science. ISSN 0036-8075 (print), 1095-9203 (online)