Allosterically controllable maxizymes for molecular gene therapy

Current Opinion in Molecular Therapeutics

Volumn 4, Issue 2, 2002, Pages 154-165

  Iyo, Mayu ^a   Kawasaki, Hiroaki ^a   Taira, Kazunari ^a  

^a NATIONAL INSTITUTE OF ADVANCED INDUSTRIAL SCIENCE AND TECHNOLOGY AIST   (Japan)

Author keywords

Allosteric ribozyme; BCR ABL; Gene therapy; Hammerhead ribozyme; Leukemia; Maxizyme

Indexed keywords

BCR ABL PROTEIN; DIHYDROLIPOAMIDE SUCCINYLTRANSFERASE; MAXIZYME; MESSENGER RNA; NUCLEIC ACID; RETINOIC ACID RECEPTOR ALPHA; RIBOZYME; UNCLASSIFIED DRUG;

ALLOSTERISM; ANIMAL EXPERIMENT; ANIMAL MODEL; ANTINEOPLASTIC ACTIVITY; CHIMERA; CONTROLLED STUDY; DRUG DESIGN; DRUG EFFICACY; DRUG SPECIFICITY; DRUG STRUCTURE; ENZYME REGULATION; GENE TARGETING; GENE TECHNOLOGY; GENE THERAPY; IN VITRO STUDY; IN VIVO STUDY; NONHUMAN; REVIEW; RNA CLEAVAGE; RNA SEQUENCE; ANIMAL; ENZYME SPECIFICITY; METABOLISM; MOUSE;

ALLOSTERIC REGULATION; ANIMAL; GENE THERAPY; MICE; RNA, CATALYTIC; SUBSTRATE SPECIFICITY; ANIMALS;

EID: 0035997961     PISSN: 14648431     EISSN: None     Source Type: Journal    
DOI: None     Document Type: Review

References (103)

1. Biological catalysis by RNA
2. A synthetic peptide ligase
3. Training ribozymes to switch
4. Nucleic acids molecular switches
5. Oligonucleotide aptamers that recognize small molecules
6. In vitro selection of nucleic acids and proteins: What are we learning?
7. Deoxyribozymes: New players in the ancient game of biocatalysis
8. In vitro selection of functional nucleic acids
9. Ribozyme-catalyzed tRNA aminoacylation
10. Ribozymes and their medical implications
11. Therapeutic Applications of Ribozymes
12. Intracellular Ribozyme Applications: Principles and Protocols
13. Ribozyme Biochemistry and Biotechnology
14. The hydrolysis of RNA: From theoretical calculations to the hammerhead ribozyme-mediated cleavage of RNA
15. Distinct roles of the co-activators p300 and CBP in retinoic-acid-induced F9-cell differentiation
16. Ribozyme-mediated repair of sickle β-globin mRNAs in erythro-cyto precursors
17. Effective inhibition of influenza virus production in cultured cells by external guide sequences and ribonuclease P
18. A hairpin ribozyme inhibits expression of diverse strains of human immunodeficiency virus type 1
19. Factors governing the activity in vivo of ribozymes transcribed by RNA polymerase III
20. RNA enzymes (ribozymes) as antiviral therapeutic agents
21. Catalytic RNA
22. Expression of chimeric tRNA-driven antisense transcripts renders NIH 3T3 cells highly resistant to Moloney murine leukemia virus replication
23. Can hammerhead ribozymes be efficient tools for inactivating gene function?
24. The expression cassette determines the functional activity of ribozymes in mammalian cells by controlling their intracellular localization
25. Recognition of engineered tRNAs with an extended 3′ end by exportin-t (Xpo-t) and transport of tRNA-attached ribozymes to the cytoplasm in somatic cells
26. Significantly higher activity of a cytoplasmic hammerhead ribozyme than a corresponding nuclear counterpart: Engineered tRNAs with an extended 3′ end can be exported efficiency and specifically to the cytoplasm in mammalian cells
27. RNA-protein hybrid ribozymes that efficiently cleave any mRNA independently of the structure of the target RNA
28. Identification of genes that function in the TNFα-mediated pathway to apoptosis by analysis of randomized hybrid-ribozyme libraries
29. Identification of genes by hybrid-ribozyme that couple cleavage activity with the unwinding activity of an endogenous RNA helicase
30. Ribozyme-mediated inactivation of mutant K-ras oncogene in a colon cancer cell line
31. Generation of a ribozyme-adenoviral vector against K-ras mutant human lung cancer cells
32. Anti-K-ras ribozyme induces growth inhibition and increased chemosensitivity in human colon cancer cells
33. Adenovirus-mediated anti-K-ras ribozyme induces apoptosis and growth suppression of human pancreatic carcinoma
34. Inhibition of cell proliferation in HCC-9204 hepatoma cells by a c-myc-specific ribozyme
35. Adenovirus-mediated ribozyme targeting of HER-2/neu inhibits in vivo growth of breast cancer cells
36. Induction of apoptosis in oral cancer cells by an anti-BCL-2 ribozyme delivered by an adenovirus vector
37. Retrovirus-mediated transfer of anti-MDR1 hammerhead ribozymes into multidrug-resistant human leukemia cells: Screening for effective target sites
38. Retrovirus-mediated transfer of anti-MDR1 ribozymes fully restores chemosensitivity of P-glycoprotein-expressing human lymphoma cells
39. The 5′-end of hTERT mRNA is a good target for hammerhead ribozyme to suppress telomerase activity
40. Inhibition of telomerase activity by a hammerhead ribozyme targeting the RNA component of telomerase in human melanoma cells
41. Acute toxicology and pharmacokinetic assessment of a ribozyme (ANGIOZYME) targeting vascular endothelial growth factor receptor mRNA in the cynomolgus monkey
42. Pharmacokinetics of an antiangiogenic ribozyme (Angiozyme) in the mouse
43. Inhibition of human immunodeficiency virus type 1 expression by a hairpin ribozyme
44. Ribozyme therapy for HIV infection
45. Elimination of hepatitis C virus RNA in infected human hepatocytes by adenovirus-mediated expression of ribozymes
46. Combinatorial screening and intracellular antiviral activity of hairpin ribozymes directed against hepatitis B virus
47. Gene therapy targeting cord blood-derived CD34(+) cells from HIV-exposed infants: Preclinical studies
48. Resistance to human immunodeficiency virus type 1 infection conferred by transduction of human peripheral blood lymphocytes with ribozyme, antisense, or polymeric trans-activation response element constructs
49. Preclinical characterization of an anti-tat ribozyme for therapeutic application
50. A phase I trial of autologous CD34+ hematopoietic progenitor cells transduced with an anti-HIV ribozyme
51. Clinical aspects of ribozymes as therapeutics in gene therapy
52. A marker study of therapeutically transduced CD4+ peripheral blood lymphocytes in HIV discordant identical twins
53. +-mediated reactions catalyzed by a hammerhead ribozyme
54. The structure, function and application of the hammerhead ribozyme
55. Ribozymes: The hammerhead swings into action
56. The hairpin ribozyme: Structure, assembly and catalysis
57. Nucleic acid enzymes
58. Structure, folding and catalysis of the small nucleolytic ribozymes
59. RNA structure, metal ions, and catalysis
60. Ribozyme structure and function
61. Differences among mechanisms of ribozyme-catalyzed reactions
62. Kinetic characterization of intramolecular and intermolecular hammerhead RNAs with stem II deletions
63. Hammerhead ribozymes: Importance of stem-loop II activity
64. Minimal sequence requirements for ribozyme activity
65. Hammerhead ribozymes containing non-nucleoside linkers are active RNA catalysts
66. Hammerhead minizymes with high cleavage activity: A dimeric structure as the active conformation of minizymes
67. Formation of a catalytically active dimer by tRNA(Val)- Driven short ribozymes
68. A novel allosterically trans-activated ribozyme, the maxizyme, with exceptional specificity in vitro and in vivo
69. Val-heterodimeric maxizymes with high potential as gene-inactivating agents: Simultaneous cleavage at two sites in HIV-1 tat mRNA in cultured cells
70. Allosterically controlled single-chained maxizymes with extremely high and specific activity
71. Maxizymes, novel allosterically controllable ribozymes can be designed to cleave various substrates
72. Characterization of several kinds of dimer minizyme: Simultaneous cleavage at two sites in HIV-1 tat mRNA by dimer minizymes
73. Chemical and enzymatic probing of effector-mediated changes in the conformation of a maxizyme
74. Allosterically controllable ribozymes with biosensor functions
75. Allosterically controllable maxizymes cleave mRNA with high efficiency and specificity
76. Working at the cutting edge: The creation of allosteric ribozymes
77. Relationships between the activities in vitro and in vivo of various kinds of ribozyme and their intracellular localization in mammalian cells
78. Cleavage of specific sites of RNA by designed ribozymes
79. Sequence requirements of the hammerhead RNA self-cleavage reaction
80. Extended target-site specificity for a hammerhead ribozyme
81. Comparative analysis of cleavage rates after systematic permutation of the NUX consensus target motif for hammerhead ribozymes
82. Generality of the NUX rule: Kinetic analysis of the results of systematic mutations in the trinucleotide at the cleavage site of hammerhead ribozymes
83. Sequence specificity of the hammerhead ribozyme revisited; the NHH rule
84. A new consistent chromosomal abnormality in chronic myelogenous leukemia identified by quinacrine fluorescence and Giemsa staining
85. Translocation of c-ab1 oncogene correlates with the presence of a Philadelphia chromosome in chronic myelocytic leukemia
86. Localization of the c-ab1 oncogene adjacent to a translocation break point in chronic myelocytic leukaemia
87. Philadelphia chromosomal breakpoints are clustered within a limited region, bcr, on chromosome 22
88. Alternative splicing of RNAs transcribed from the human ABL gene and from the BCR-ABL fused gene
89. An alteration of the human c-ABL protein in K562 leukemia cells unmasks associated tyrosine kinase activity
90. Treatment of hematology and bone marrow transcription, City of Hope National Medical Center, Duarte, California
91. Untreated donor marrow transplantation therapy for chronic mylogenous leukemia: Initial experience of the National Marrow Donor Program
92. BCR-ABL protein expression in peripheral blood cells of chronic myelogenous leukemia patients undergoing therapy
93. Induction of chronic myelogenous leukemia in mice by the P210bcr/abl gene of the Philadelphia chromosome
94. Initiation of deregulated growth of multipotent progenitor cells by bcr-abl in vitro
95. Comparison of the specificities and catalytic activities of hammerhead ribozymes and DNA enzymes with respect to the cleavage of BCR-ABL chimeric L6 (b2a2) mRNA
96. Oncogene inactivation in an mouse model: Tissue invasion by leukaemic cells is stalled by loading them with a designer ribozyme
97. Allosterically controllable maxizyme-mediated suppression of progression of leukemia in mice
98. The PML-RARα fusion mRNA generated by the t(15;17) translocation in acute promyelocytic leukemia encodes a functionally altered RAR
99. Fusion protein of the retinoic acid receptor-α recruit histone deacetylase in promyelocytic leukemia
100. Isolation, characterization and structural organization of the gene and pseudogene for the dihydrolipoamide succinyltransferase component of the human 2-oxoglutarate dehydrogenase complex
101. A polypeptide derived from mitochondrial dihydrolipoamide succinyltransferase is located on the plasma membrane in skeletal muscle