RNA enzymes (Ribozymes)- gene therapy applications for clinical medicine
Toudjarska I1, Kremensky IM1, Mitev VI2*
*Corresponding Author: Professor Dr. Vanio I Mitev e-mail: mitev@medfac.acad.bg
page: 3

References

1. Kruger K, Grabowski PJ, Zaug AJ, Sands J, Gottschling DE, Cech TR. Self-splicing RNA: autoexcision and autocyclization of the ribosomal RNA intervening sequence of Tetrahymena. Cell 1982;31(1):147-57.

2. Cech and Gestalang, The RNA World: CSHL Press, 1999.

3. Breaker RR. In Vitro Selection of Catalytic Polynucleotides. Chem Rev 1997;97(2):371-390.

4. Puerta-Fernandez E, Romero-Lopez C, Barroso-delJesus A, Berzal-Herranz A. Ribozymes: recent advances in the development of RNA tools. FEMS Microbiol Rev 2003;27(1):75-97.

5. Nissen P, Hansen J, Ban N, Moore PB, Steitz TA. The structural basis of ribosome activity in peptide bond synthesis. Science 2000;289(5481):920-30.

6. Haseloff J, Gerlach WL. Simple RNA enzymes with new and highly specific endoribonuclease activities. Nature 1988;334(6183):585-91.

7. Branch AD, Robertson HD. Efficient trans cleavage and a common structural motif for the ribozymes of the human hepatitis delta agent. Proc Natl Acad Sci U S A 1991;88(22):10163-7.

8. Pestka S, Daugherty BL, Jung V, Hotta K, Pestka RK. Anti-mRNA: specific inhibition of translation of single mRNA molecules. Proc Natl Acad Sci U S A 1984;81(23):7525-8.

9. Izant JG, Weintraub H. Constitutive and conditional suppression of exogenous and endogenous genes by anti-sense RNA. Science 1985;229(4711):345-52.

10. van der Krol AR, Mol JN, Stuitje AR. Modulation of eukaryotic gene expression by complementary RNA or DNA sequences. Biotechniques 1988;6(10):958-76.

11. Phylactou LA, Tsipouras P, Kilpatrick MW. Hammerhead ribozymes targeted to the FBN1 mRNA can discriminate a single base mismatch between ribozyme and target. Biochem Biophys Res Commun 1998;249(3):804-10.

12. Kore AR, Vaish NK, Kutzke U, Eckstein F. Sequence specificity of the hammerhead ribozyme revisited; the NHH rule. Nucleic Acids Res 1998;26(18):4116-20.

13. Beigelman L, McSwiggen JA, Draper KG, Gonzalez C, Jensen K, Karpeisky AM, et al. Chemical modification of hammerhead ribozymes. Catalytic activity and nuclease resistance. J Biol Chem 1995;270(43):25702-8.

14. Weng DE, Usman N. Angiozyme: a novel angiogenesis inhibitor. Curr Oncol Rep 2001;3(2):141-6.

15. Sullenger BA, Cech TR. Tethering ribozymes to a retroviral packaging signal for destruction of viral RNA. Science 1993;262(5139):1566-9.

16. Lee NS, Bertrand E, Rossi J. mRNA localization signals can enhance the intracellular effectiveness of hammerhead ribozymes. Rna 1999;5(9):1200-9.

17. Chirmule N, Propert K, Magosin S, Qian Y, Qian R, Wilson J. Immune responses to adenovirus and adeno-associated virus in humans. Gene Ther 1999;6(9):1574-83.

18. Niidome T, Huang L. Gene therapy progress and prospects: nonviral vectors. Gene Ther 2002;9(24):1647-52.

19. Zhang YA, Nemunaitis J, Scanlon KJ, Tong AW. Anti-tumorigenic effect of a K-ras ribozyme against human lung cancer cell line heterotransplants in nude mice. Gene Ther 2000;7(23):2041-50.

20. Mendoza-Maldonado R, Zentilin L, Fanin R, Giacca M. Purging of chronic myelogenous leukemia cells by retrovirally expressed anti-bcr-abl ribozymes with specific cellular compartmentalization. Cancer Gene Ther 2002;9(1):71-86.

21. Aigner A, Renneberg H, Bojunga J, Apel J, Nelson PS, Czubayko F. Ribozyme-targeting of a secreted FGF-binding protein (FGF-BP) inhibits proliferation of prostate cancer cells in vitro and in vivo. Oncogene 2002;21(37):5733-42.

22. Kobayashi H, Takemura Y, Miyachi H. Novel approaches to reversing anti-cancer drug resistance using gene-specific therapeutics. Hum Cell 2001;14(3):172-84.

23. Wang L, Witherington C, King A, Gerlach WL, Carr A, Penny R, et al. Preclinical characterization of an anti-tat ribozyme for therapeutic application. Hum Gene Ther 1998;9(9):1283-91.

24. Bauer G, Valdez P, Kearns K, Bahner I, Wen SF, Zaia JA, et al. Inhibition of human immunodeficiency virus-1 (HIV-1) replication after transduction of granulocyte colony-stimulating factor-mobilized CD34+ cells from HIV-1-infected donors using retroviral vectors containing anti-HIV-1 genes. Blood 1997;89(7):2259-67.

25. Wong-Staal F, Poeschla EM, Looney DJ. A controlled, Phase 1 clinical trial to evaluate the safety and effects in HIV-1 infected humans of autologous lymphocytes transduced with a ribozyme that cleaves HIV-1 RNA. Hum Gene Ther 1998;9(16):2407-25.

26. Macejak DG, Jensen KL, Pavco PA, Phipps KM, Heinz BA, Colacino JM, et al. Enhanced antiviral effect in cell culture of type 1 interferon and ribozymes targeting HCV RNA. J Viral Hepat 2001;8(6):400-5.

27. Lee PA, Blatt LM, Blanchard KS, Bouhana KS, Pavco PA, Bellon L, et al. Pharmacokinetics and tissue distribution of a ribozyme directed against hepatitis C virus RNA following subcutaneous or intravenous administration in mice. Hepatology 2000;32(3):640-6.

28. Morrissey DV, Lee PA, Johnson DA, Overly SL, McSwiggen JA, Beigelman L, et al. Characterization of nuclease-resistant ribozymes directed against hepatitis B virus RNA. J Viral Hepat 2002;9(6):411-8.

29. Dawson PA, Marini JC. Hammerhead ribozymes selectively suppress mutant type I collagen mRNA in osteogenesis imperfecta fibroblasts. Nucleic Acids Res 2000;28(20):4013-20.

30. Toudjarska I, Kilpatrick MW, Niu J, Wenstrup RJ, Tsipouras P. Delivery of a hammerhead ribozyme specifically downregulates mutant type I collagen mRNA in a murine model of osteogenesis imperfecta. Antisense Nucleic Acid Drug Dev 2001;11(5):341-6.

31. Millington-Ward S, O'Neill B, Kiang AS, Humphries P, Kenna PF, Farrar GJ. A mutation-independent therapeutic strategem for osteogenesis imperfecta. Antisense Nucleic Acid Drug Dev 1999;9(6):537-42.

32. Lewin AS, Drenser KA, Hauswirth WW, Nishikawa S, Yasumura D, Flannery JG, et al. Ribozyme rescue of photoreceptor cells in a transgenic rat model of autosomal dominant retinitis pigmentosa. Nat Med 1998;4(8):967-71.

33. Suzuki Y, Funato T, Munakata Y, Sato K, Hirabayashi Y, Ishii T, et al. Chemically modified ribozyme to V gene inhibits anti-DNA production and the formation of immune deposits caused by lupus lymphocytes. J Immunol 2000;165(10):5900-5.

34. Takahashi M, Funato T, Suzuki Y, Fujii H, Ishii KK, Kaku M, et al. Chemically modified ribozyme targeting TNF-alpha mRNA regulates TNF-alpha and IL-6 synthesis in synovial fibroblasts of patients with rheumatoid arthritis. J Clin Immunol 2002;22(4):228-36.

35. Enjoji M, Wang F, Nakamuta M, Chan L, Teng BB. Hammerhead ribozyme as a therapeutic agent for hyperlipidemia: production of truncated apolipoprotein B and hypolipidemic effects in a dyslipidemia murine model. Hum Gene Ther 2000;11(17):2415-30.

 




Number 26
Number 26 VOL. 26(2), 2023 All in one
Number 26
VOL. 26(2), 2023
Number 26
VOL. 26, 2023 Supplement
Number 26
VOL. 26(1), 2023
Number 25
VOL. 25(2), 2022
Number 25
VOL. 25 (1), 2022
Number 24
VOL. 24(2), 2021
Number 24
VOL. 24(1), 2021
Number 23
VOL. 23(2), 2020
Number 22
VOL. 22(2), 2019
Number 22
VOL. 22(1), 2019
Number 22
VOL. 22, 2019 Supplement
Number 21
VOL. 21(2), 2018
Number 21
VOL. 21 (1), 2018
Number 21
VOL. 21, 2018 Supplement
Number 20
VOL. 20 (2), 2017
Number 20
VOL. 20 (1), 2017
Number 19
VOL. 19 (2), 2016
Number 19
VOL. 19 (1), 2016
Number 18
VOL. 18 (2), 2015
Number 18
VOL. 18 (1), 2015
Number 17
VOL. 17 (2), 2014
Number 17
VOL. 17 (1), 2014
Number 16
VOL. 16 (2), 2013
Number 16
VOL. 16 (1), 2013
Number 15
VOL. 15 (2), 2012
Number 15
VOL. 15, 2012 Supplement
Number 15
Vol. 15 (1), 2012
Number 14
14 - Vol. 14 (2), 2011
Number 14
The 9th Balkan Congress of Medical Genetics
Number 14
14 - Vol. 14 (1), 2011
Number 13
Vol. 13 (2), 2010
Number 13
Vol.13 (1), 2010
Number 12
Vol.12 (2), 2009
Number 12
Vol.12 (1), 2009
Number 11
Vol.11 (2),2008
Number 11
Vol.11 (1),2008
Number 10
Vol.10 (2), 2007
Number 10
10 (1),2007
Number 9
1&2, 2006
Number 9
3&4, 2006
Number 8
1&2, 2005
Number 8
3&4, 2004
Number 7
1&2, 2004
Number 6
3&4, 2003
Number 6
1&2, 2003
Number 5
3&4, 2002
Number 5
1&2, 2002
Number 4
Vol.3 (4), 2000
Number 4
Vol.2 (4), 1999
Number 4
Vol.1 (4), 1998
Number 4
3&4, 2001
Number 4
1&2, 2001
Number 3
Vol.3 (3), 2000
Number 3
Vol.2 (3), 1999
Number 3
Vol.1 (3), 1998
Number 2
Vol.3(2), 2000
Number 2
Vol.1 (2), 1998
Number 2
Vol.2 (2), 1999
Number 1
Vol.3 (1), 2000
Number 1
Vol.2 (1), 1999
Number 1
Vol.1 (1), 1998

 

 


 About the journal ::: Editorial ::: Subscription ::: Information for authors ::: Contact
 Copyright © Balkan Journal of Medical Genetics 2006