Publications

Gene Characterization

Friedmann, T., and Brown, D. "Base-Specific Reactions Useful for DNA Sequencing." Nucl. Acids Res. 5, 615-622 (1978)

Sanger, F., Coulson, A., Friedmann, T., Air, G., Barrell, B., Brown, N., Fiddes, J., Hutchison, C., Slocombe, P., and Smith, M. "The Nucleotide Sequence of Bacteriophage fX 174," J. Mol. Biol. 125, 225-246 (1978)

Friedmann, T., Esty, A., La Porte, P., and Deininger, P. "The Nucleotide Sequence and Genome Organization of the Polyoma Early Region: Extensive Nucleotide and Amino Acid Homology with SV40." Cell 17, 714-724 (1979)

Deininger, P., Esty, A., La Porte, P., and Friedmann, T. "Nucleotide Sequence and Genetic Organization of the Polyoma Late Region: Features Common to the Polyoma Early Region and to SV40." Cell 18, 771-779 (1979)

Deininger, P.L., Jolly, D.J., Rubin, C.M., Friedmann, T., and Schmid, C.W. "Base Sequence Studies of 300 Nucleotide Renatured Repeated Human DNA Clones." J. Mol. Biol. 151, 17-33 (1981)

Lin, S., Gaiano, N., Culp, P., Burns, J.C., Friedmann, T., Yee, J.-K., and Hopkins, N. "Integration and Germ Line Transmission of a Pseudotyped Retroviral Vector in Zebrafish." Science 265, 666-669(1994)

Gene Therapy

Friedmann, T., and Roblin, R. "Gene therapy for human genetic disease?" Science 175, 949-955 (1972)

Miller, A.D., Jolly, D.J., Friedmann, T., and Verma, I.M. "A Transmissible Retrovirus Expressing Human Hypoxanthine Phosphoribosyltransferase (HPRT): Gene Transfer into Cells Obtained from Humans Deficient in HPRT." Proc. Natl.Acad. Sci. USA 80, 4709-4713 (1983)

Willis, R.C., Jolly, D.J., Miller, A.D., Plent, M.M., Esty, A.C., Anderson, P.J., Chang, H.C., Jones, O.W., Seegmiller, J.E., and Friedmann, T. "Partial Phenotypic Correction of Human Lesch-Nyhan (HPRT-Deficient) Lymphoblasts with a Transmissible Retroviral Vector." J. Biol. Chem. 259, 7842-7849 (1984)

Miller, A.D., Eckner, R.J., Jolly, D.J., Friedmann, T., and Verma, I.M. "Expression of a Retrovirus Encoding Human HPRT in Mice." Science 225, 630-632 (1984)

Gruber, H., Finley, K., Hershberg, R., Katzman, S., Laikind, P., Friedmann, T., Yee, J.-K., and Jolly, D. "Retroviral Vector-Mediated Gene Transfer into Human Hematopoietic Cells." Science 230, 1057-1061 (1985)

Gage, F., Wolff, J., Rosenberg, M., Xu, L., Yee, J.-K., Shultz, C., and Friedmann, T. "Grafting Genetically Modified Cells to the Brain." Neuroscience 23, 795-807 (1987)

Huang, H.-J. S., Yee, J.-K., Shew, J.-Y., Chen, P.-L., Bookstein, R., Friedmann, T., Lee, E.Y.-H.P., and Lee, W.-H. "Suppression of the Neoplastic Phenotype by Replacement of the Human Retinoblastoma Gene Product in Retinoblastoma and Osteosarcoma Cells." Science 242, 1563-1566 (1988)

Rosenberg, M.B., Friedmann, T., Robertson, R.C., Tuszynski, M., Wolff, J.A., Breakefield, X.O., and Gage, F.H. "Grafting of Genetically Modified Cells to the Damaged Brain: Restorative Effects of NGF Gene Expression." Science 242, 1575-1578 (1988)

Friedmann,T. "Progress Toward Human Gene Therapy." Science 244, 1275-1281 (1989)

Wolff, J.A., Fisher, L.J., Li Xu, Rosenberg, M.B., Friedmann, T., Iuvone, P.M., O'Malley, K.L., Jinnah, H.A., and Gage, F.H. "Grafting Fibroblasts Genetically Modified to Produce L-DOPA in a Rat Model of Parkinson's Disease." Proc. Natl. Acad. Sci. USA 86, 9011-9014 (1989)

Friedmann, T. "Gene Therapy for Cancer Through Restoration of Cancer Suppressor Gene Activity," Cancer 70, 1810-1816 (1991)

Cheng, J., Yee, J.-K., Yeargin, J., Friedmann, T., and Haas, M. "Suppression of Acute Lymphoblastic Leukemia by the Human p53 Gene." Cancer Research 52, 222-226 (1992)

Friedmann, T. "A Brief History of Gene Therapy." Nature Genetics 2, 93-98 (1992)

Friedmann, T. "Gene Therapy - A New Form of Medicine." Trends in Biotechnology 11, 156-159 (1993)

Jinnah, H., and Friedmann, T. "Gene Therapy for Disorders of the Central Nervous System." Trends in Biotechnology 11, 192-197 (1993)

Friedmann, T. "Gene Therapy - an Immature Genie but Certainly Out of the Bottle." Nature Medicine 2, 144-147 (1996)

Friedmann, T. "Overcoming the Obstacles to Gene Therapy." Scientific American 276, 95-101 (1997)

Friedmann, T. "The Road Toward Human Gene Therapy - A 25 Year Perspective." Annals of Medicine 29, 575-577 (1997)

Friedmann, T. "The New Era of Human Gene Therapy Research: A Balance of unequivoval Benefit and Unequivocal Harm." Mol.Ther.8(1), 5-7(2003)

Lesch Nyhan Disease

Jolly, D.J., Esty, A.C., Bernard, H.U., and Friedmann, T. "Isolation of a Genomic Clone Encoding Human Hypoxanthine Guanine Phosphoribosyl Transferase." Proc. Natl. Acad. Sci. USA 79, 5038-5041 (1982)

Jolly, D.J., Okayama, H., Berg, P., Esty, A.C., Filpula, D., Bohlen, P., Johnson, G.G., Shively, J.E., Hunkapillar, T., and Friedmann, T. "Isolation and Characterization of a Full Length, Expressible cDNA for Human Hypoxanthine Guanine Phosphoribosyl Transferase." Proc. Natl. Acad. Sci. USA, 80, 477-481 (1983)

Jinnah, H.A., Gage, F.H., and Friedmann, T. "Animal Models of Lesch-Nyhan Syndrome." Brain Res. Bulletin 25, 467-475 (1990)

Jinnah, H.A., Gage, F.H., and Friedmann, T. "Amphetamine-Induced Behavioral Phenotype in HPRT-Deficient Mice." Behav. Neuroscience 105, 1004-1012 (1991)

Jinnah, H.A., Langlais, P.J., and Friedmann, T., "Functional Analysis of Brain Dopamine Systems in a Genetic Mouse Model of Lesch-Nyhan Syndrome." J.Pharmacol.Exper.Therap.263, 506-607 (1992)

Jinnah, H.A., Page, R., and Friedmann, T. "Brain Purines in a Genetics Mouse Model of Lesch-Nyhan Disease." J. Neurochemistry 60, 2036-2045 (1993)

Jinnah, H.A., Wojcik, B.E., Hunt, M., Narang, N., Lee, K.Y., Goldstein, M., Wamsley, J.K., Langlais, P.J., and Friedmann, T. "Dopamine Deficiency in a Genetic Mouse Model of Lesch-Nyhan Disease." The Journal of Neuroscience 14, 1164-1175 (1994)

Smith, D.W., and Friedmann, T. "Characterization of the Dopamine Defect in Primary Cultures of Dopaminergic Neurons from HPRT Knockout Mice." Molec.Ther. 1, 486-491 (2000)

Jinnah, H.A., and Friedmann, T. "Lesch-Nyhan Disease and its Variants." In The. Metabolic and Molecular Bases of Inherited Disease, Scriver, Beaudet, Sly and Valle (eds), McGraw Hill, pp. 2537-2570 (2000)

Visser, J., Smith, D., Moy, S., Breese, G.R., Friedmann, T., Rothstein, J., and Jinnah, H. "Oxidative Stress and Dopamine Deficiency in a Genetic Mouse Model of Lesch-Nyhan Disease." Dev.Brain Res.133, 127-139 (2002)

Virus Characterization and Vector Development

Friedmann, T. "In Vitro Reassembly of Shell-Like Particles from Disrupted Polyoma Virus." Proc. Natl. Acad. Sci. USA 68, 2574-2578 (1971)

Friedmann, T., and David, D. "Structural Roles of Polyoma Proteins." J.Virology 10, 776-782 (1972)

Emi, N., Friedmann, T., and Yee, J.-K., "Pseudotype Formation of Murine Leukemia Virus with the G Protein of Vesicular Stomatitis Virus," J. Virol. 65, 1202-1207 (1991)

Burns, J.C., Friedmann, T., Driever, W., Burrascano, M., and Yee, J.-K. "VSV-G Pseudotyped Retroviral Vectors: Concentration to Very High Titer and Efficient Gene Transfer into Mammalian and Non-Mammalian Cells." Proc.Nat'l. Acad. Sci. USA 90, 8033-8037 (1993)

Yee, J.-K., Miyanohara, A., LaPorte, P., Bouic, K., Burns, J.C., and Friedmann, T. "A General Method for the Generation of High Titer, Pantropic Retroviral Vectors: Highly Efficient Infection of Primary Hepatocytes." Proc.Nat'l.Acad.Sci.USA 91, 9564-9568 (1994)

Chen, S.-T., Iida, A., Guo, L ., Friedmann, T., and Yee, J.K. "Generation of Packaging Cell Lines for VSV-G Pseudotyped Retroviral Vectors Using a Modified Tetracycline Inducible System." Proc.Nat.Acad.Sci.USA 93, 10057-10062 (1996)

Abe, A., Miyanohara, A., and Friedmann, T. "Enhanced Gene Transfer with Fusogenic Liposomes Containing Vesicular Stomatitis Virus G Glycoprotein." J. Virology 72, 6159-6163 (1998)

Abe, A., Chen, S.-T., Miyanohara, A., and Friedmann, T. "In Vitro Cell-Free Conversion of Noninfectious Moloney Retrovirus Particles to an Infectious Form by the Addition of the Vesicular Stomatitis Virus Surrogate Envelop G Protein." J. Virology 72, 6356-6361 (1998)

Okimoto, T., Friedmann, T., and Miyanohara, A. "VSV-G envelope glycoprotein forms complexes with plasmid DNA and MLV retrovirus-like particles in cell-free conditions and enhances DNA transfection." Molec.Ther.4:232-238 (2001)

Guibinga, G.H., Miyanohara, A., Esko, J.D., and Friedmann, T. "Cell Surface Heparan Sulfate is a Receptor for Attachment of Envelope Protein-Free Retrovirus-Like Particles (VLP) and VSV-G Pseudotyped MLV-Derived Retrovirus Vectors to Target Cells." Molec.Ther.5, 538-546 (2002)

Guibinga, GH, Hall, FL, Gordon, EM, Ruoslahti, E, and Friedmann, T. "Ligand-Modified Vesicular Stomatitis Virus Variants Display a Temperature-Sensitive Intracellular Trafficking and Virus Assembly Phenotype." Molec.Ther. 9(1), 76-84 (2004)

Guibinga, G.H., and Friedmann, T. "Baculovirus GP64-Pseudotyped HIV-Based Lentivirus Vectors are Stabilized Against Complement Inactivation by Codisplay of Decay Accelerating Factor (DAF) or of a GP64-DAF Fusion Protein." Molec.Ther. 11 (4), 645-651(2005)

Policy

Friedmann, T. "Prenatal Diagnosis of Genetic Disease." Scientific American 225, 34 (1971)

Friedmann, T. "Scientific and Public Policy Towards Gene Therapy." Bioessays 3, 40-41 (1985)

Friedmann, T. "Some Ethical Implications of Human Gene Therapy." In Genetics, Ethics and Human Values, Proceedings of XXIVth CIOMS Conference, Tokyo and Inuyama, Japan (1990)

Friedmann, T. "Molecular Medicine." In The Genetic Revolution: Scientific Prospects and Public Perceptions, B.D. Davis, ed., Johns Hopkins Press, Baltimore, 132-151 (1991)

Friedmann, T. "Ethical Duties of Scientists, Their Institutions and the Guild of Science." J. NIH Research 4, 19-22 (1992)

Friedmann, T. "The Promise And Overpromise Of Human Gene Therapy." Gene Therapy. Editorial Volume 1, Number 4, July (1994)

Friedmann, T. "Rigor in Gene Therapy Studies." Gene Therapy 2, 1-2 (1995)

Friedmann, T. "Changing Roles for Academia and Industry in Genetics and Gene Therapy." Mol.Ther. 1, 9-11 (2000)

Friedmann, T. "Principles for Human Gene Therapy Studies." Science 287, 2163-2165 (2000)

Friedmann, T., Noguchi, P., and Mickelson, C. "Evolution of Review and Regulation in Gene Therapy." Current Opinions in Biotechnology 12, 304-311(2001)

Friedmann, T., and Koss, J.O. "Gene Transfer and Athletics - An Impending Problem." Molec. Ther. 3, 819-820 (2001)

"Lessons for the Stem Cell Discourse from the Gene Therapy Experience." Perspectives in Biol. and Med 48(4), 585-591 (2005)

Books (selected)

Friedmann, T. "Gene Therapy - Fact and Fiction" in Biology's New Approaches to Disease.Cold Spring Harbor Laboratory, pp 91-96 (1983)

Friedmann, T. (ed.) Therapy for Genetic Disease, Oxford University Press, Oxford (1991)

Friedmann, T. The Development of Human Gene Therapy, Cold Spring Harbor Press (1999)

T. Friedmann and A. Schneider. Genetic Doping in Sport: The Science of Genetically Modified Athletes. Elsevier Academic Press, San Diego (2006).

T. Friedmann and J. Rossi. Gene Transfer - A Laboratory Manual. Cold Spring Harbor Press, New York, N.Y., (2006)