Gene therapy

Buchschacher, G. and Wong-Staal, F. 2001. Approaches to gene therapy for human immunodeficiency virus infection. Human Gene Therapy 12(9), 1013-1019.

Davies, J.C., Geddes, D.M., and Alton, E.W. 2001. Gene therapy for cystic fibrosis. Journal of Gene Medicine 3(5), 409-417.

Demeterco, C. and Levine, F. 2001. Gene therapy for diabetes. Frontiers in Bioscience 6, D175-D191.

Edelstein, M.L., Abedi, M.R., Wixon, J., and Edelstein, R.M. 2004. Gene therapy clinical trials worldwide 1989-2004 - an overview. Journal of Gene Medicine 6(6), 597-602.

Eliyahu, H., Barenholz, Y., and Domb, A.J. 2005. Polymers for DNA delivery. Molecules 10(1), 34-64.

Ferguson, C., Larochelle, A., and Dunbar, C.E. 2005. Hematopoietic stem cell gene therapy: dead or alive? Trends in Biotechnology 23(12), 589-597.

Ferreira, G.N., Monteiro, G.A., Prazeres, D.M., and Cabral, J.M. 2000. Downstream processing of plasmid DNA for gene therapy and DNA vaccine applications. Trends in Biotechnology 18(9), 380-388.

Furlan, R., Butti, E., Pluchino, S., and Martino, G. 2004. Gene therapy for autoimmune diseases. Current Opinion in Molecular Therapeutics 6(5), 525-536.

Gardlik, R., Palffy, R., Hodosy, J., Lukacs, J., Turna, J., and Celec, P. 2005. Vectors and delivery systems in gene therapy. Medical Science Monitor 11(4), RA110-RA121.

Lewin, A. and Hauswirth, W. 2001. Ribozyme gene therapy: applications for molecular medicine. Trends in Molecular Medicine 7(5), 221-228.

Mhashilkar A, Chada S, Roth JA, and Ramesh R. 2001. Gene therapy - therapeutic approaches and implications. Biotechnology Advances 19(4), 279-297.

Ohlfest, J.R., Freese, A.B., and Largaespada, D.A. 2005. Nonviral vectors for cancer gene therapy: prospects for integrating vectors and combination therapies. Current Gene Therapy 5(6), 629-641.

Palmer, D.H., Young, L.S., and Mautner, V. 2006. Cancer gene therapy: clinical trials. Trends in Biotechnology 24(2), 76-82.

Phillips, A. 2001. The challenge of gene therapy and DNA delivery. Journal of Pharmacy and pharmacology. 53(9), 1169-1174.

Pfeifer, A. and Verma, I. 2001. Gene therapy: promises and problems. Annual review of genomics and Human Genetics 2, 177-211.

Robertson, J. and Griffiths, E. 2001. Assuring the quality, safety and efficacy of DNA vaccines. Molecular Biotechnology 17(2), 143-149.

Schatzlein, A. 2001. Non-viral vectors in cancer gene therapy: principles and progress. Anti-Cancer Drugs 12(4), 275-304.

Smith, H. and Klinman, D. 2001. The regulation of DNA vaccines. Current Opinion in Biotechnology 12(3), 299-303.

Verma, I. and Weitzman, M. 2005. Gene therapy: twenty first century medicine. Annual Review of Biochemistry 74, 711-738.

Wu, N. and Ataai, M. 2000. Production of viral vectors for gene therapy applications. Current Opinion in Biotechnology 11(2), 205-208.

Yechoor, V and Chan, L. 2005. Gene therapy progress and prospects: gene therapy for diabetes mellitus. Gene Therapy 12(2), 101-107.

Young, L.S., Searle, P.F., Onion, D., and Mautner, V. 2006. Viral gene therapy strategies: from basic science to clinical application. Journal of Pathology 208(2), 299-318.

Antisense technology

Aboul-Fadt, T. 2005. Antisense oligonucleotides: the state of the art. Current Medicinal Chemistry 12(19), 2193-2214.

Adah, S.A., Bayly, S.F., Cramer, H., Silverman, R.H., and Torrence, P.F. 2001. Chemistry and biochemistry of 2', 5'-oligoadenylate-based antisense strategy. Current Medicinal Chemistry 8(10), 1189-1212.

Akhtar, S., Hughes, M.D., Khan, A., Bibby, M., Hussain, M., Nawaz, Q., Double, J., and Sayyed, P. 2000. The delivery of antisense therapeutics. Advanced Drug Delivery Reviews 44(1), 3-21.

Galderisi, U., Cipollaro, M., and Cascino, A. 2001. Antisense oligonucleotides as drugs for HIV treatment. Expert Opinion on Therapeutic Patents 11(10), 1605-1611.

Hughes, M.D., Hussain, M., Nawaz, Q., Sayyed, P., and Akhtar, S. 2001. The cellular delivery of antisense oligonucleotides and ribozymes. Drug Discovery Today 6(6), 303-315.

Jason, T.L.H., Koropatnick, J., and Berg, R.W. 2004. Toxicology of antisense therapeutics. Toxicology and Applied Pharmacology 201(1), 66-83.

Lebedeva, I. and Stein, C. 2001. Antisense oligonucleotides: promise and reality. Annual Review of Pharmacology and Toxicology 41, 403-419.

Rubenstein, M., Tsui, P., and Guinan, P. 2004. A review of antisense oligonucleotides in the treatment of human disease. Drugs of the Future 29(9), 893-909.

Stein, C.A., Benimetskaya, L., and Mani, S. 2005. Antisense strategies for oncogene inactivation. Seminars in Oncology 32(6), 563-572.

Vidal L., Blagden, S., Attard, G., and de Bono, J. 2005. Making sense of antisense. European Journal of Cancer 41(18), 2812-2818.

RNA interference

Amarzguioui, M., Rossi, J.J., and Kim, D. 2005. Approaches for chemically synthesized siRNA and vector-mediated RNAi. FEBS Letters 579(26), 5974-5981.

Bagasra, O. 2005. RNAi as an antiviral therapy. Expert Opinion on Biological Therapy 5(11), 1463-1474.

Campbell, T. and Choy, F. 2005. RNA interference: past, present and future. Current Issues in Molecular Biology 7, 1-6.

Caplen, N. 2003. RNAi as a gene therapy approach. Expert Opinion on Biological Therapy 3(4), 575-568.

Chowdhury, D. and Novina, C. 2005. RNAi and RNA-based regulation of immune system function. Advances in Immunology 88(88), 267-292.

Pai, S.I., Lin, Y.Y., Macaes, B., Meneshian, A., Hung, C.F., and Wu, T.C. 2006. Prospect of RNA interference therapy for cancer. Gene Therapy 13(6), 464-477.

Uprichard, S. 2005. The therapeutic potential of RNA interference. FEBS Letters 579(26), 5996-6007.


Alvarez-Salas, L.M., Benitez-Hess, M.L., and DiPaolo, J.A. 2003. Advances in the development of ribozymes and antisense oligodeoxynucleotides as antiviral agents for human papillomaviruses. Antiviral Therapy 8(4), 265-278.

Bagheri, S and Kashani-Sabet, M. 2004. Ribozymes in the age of molecular therapeutics. Current Molecular Medicine 4(5), 489-506.

Lilley, D. 2005. Structure, folding and mechanisms of ribozymes. Current Opinion in Structural Biology 15(3), 313-323.

Kahn, A. and Lai, S. 2003. Ribozymes: a modern tool in medicine. Journal of Biomedical Science 10(5), 457-467.

Kashani-Sabet, M. 2004. Non-viral delivery of ribozymes for cancer gene therapy. Expert Opinion on Biological Therapy 4(11), 1749-1755.

Sioud, M. and Iversen, P. 2005. Ribozymes, DNAzymes and small interfering RNAs as therapeutics. Current Drug Targets 6(6), 647-653.


Hoppe-Seyler, F., Crnkovic-Mertens, I., Tomai, E., and Butz, K. 2004. Peptide aptamers: specific inhibitors of protein function. Current Molecular Medicine 4(5), 529-583.

Nimjee, S.M., Rusconi, CP., and Sullenger, B.A. 2005. Aptamers: an emerging class of therapeutics. Annual Review of Medicine 56, 555-583.

Proske, D., Blank, M., Buhmann, R., and Resch, A. 2005. Aptamers - basic research, drug development, and clinical applications. Applied Microbiology and Biotechnology 69(4), 367-374.

Yan, A.C., Bell, K.M., Breeden, M.M., and Ellington, A.D. 2005. Aptamers: prospects in therapeutics and biomedicine. Frontiers in Bioscience 10, 1802-1827.

You, KM., Lee, S.H., Im, A., and Lee, S.B. 2003. Aptamers as functional nucleic acids: in vitro selection and biotechnological applications. Biotechnology and Bioprocess Engineering 8(2), 64-75.

Stem cells

Hoffman, L. and Carpenter, M. 2005. Characterization and culture of human embryonic stem cells. Nature Biotechnology 23(6), 699-708.

Mayhall, E., Paffett-Lugassy, N., and Zon, L. 2004. The clinical potential of stem cells. Current Opinion in Cell Biology 16, 713-720.

Wagers, A. and Weissman, I. 2004. Plasticity of adult stem cells. Cell 116, 639-648.

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