Beigelman et al., Beigelman et al., “Chemical Modification of Hammerhead Ribozymes,” J. Biol. Chem. 270:25702-25708 (1995). |
Carter, “Adeno-Associated Virus Vectors,” Curr Opi. Biotech. 3:533-539 (1992). |
Chen et al., “Multitarget-Ribozyme Directed to Cleave at up to Nine Highly Conserved HIV-1 env RNA Regions Inhibits HIV-1 Replication-Potential Effectiveness Against Most Presently Sequenced HIV-1 Isolates,” Nucleic Acids Research 20:4581-4589 (1992). |
Chowrira et al., “In Vitro and in Vivo Comparison of Hammerhead, Hairpin, and Hepatitis Delta Virus Self-Processing Ribozyme Cassettes,” J. Biol. Chem. 269:25856-25864 (1994). |
Chowrira and Burke, “Extensive Phosphorothioate Substitution Yields Highly Active and Nuclease-Resistant Hairpin Ribozymes,” Nucleic Acids Res. 20:2835-2840 (1992). |
Collins and Olive, “Reaction Conditions and Kinetics of Self-Cleavage of a Ribozyme Derived From Neurospora VS RNA,” Biochemistry 32:2795-2799 (1993). |
Dropulic et al., “Functional Characterization of a U5 Ribozyme: Intracellular Suppression of Human Immunodeficiency Virus Type I Expression,” Journal of Virology 66:1432-1441 (1992). |
Duval-Valentin, “Specific inhibition of transcription by triple helix-forming oligonucleotides,” Proc. Natl. Acad. Sci. USA 89:504 (1992). |
Egholm et al., “PNA hybridizes to complementary oligonucleotides obeying the Watson-Crick hydrogen-bonding rules,” Nature 365:566-568 (1993). |
Elroy-Stein and Moss, “Cytoplasmic Expression System Based on Constitutive Synthesis of Bacteriophage T7 RNA Polymerase in Mammalian Cells,” Proc. Natl. Acad. Sci. USA 87:6743-6747 (1990). |
Folkman and Shing, “Angiogenesis,” J. Biol. Chem. 267:10931-10934 (1992). |
Folkman, “What is the Evidence that Tumors are Angiogenesis Dependent?” Journal of the National Cancer Institute 82:4-6 (1990). |
Gao and Huang, “Cytoplasmic Expression of a Reporter Gene by Co-Delivery of T7 RNA Polymerase and T7 Promoter Sequence with Cationic Liposomes,” Nucleic Acids Res. 21:2867-2872 (1993). |
Guerrier-Takada et al., “The RNA Moiety of Ribonuclease P Is the Catalytic Subunit of the Enzyme,” Cell 35:849-857 (1983). |
Hampel et al., “‘Hairpin’ Catalytic RNA Model: Evidence for Helices and Sequence Requirement for Substrate RNA,” Nucleic Acids Research 18:299-304 (1990). |
Hampel and Tritz, “RNA Catalytic Properties of the Minimum a(−)sTRSV Sequence,” Biochemistry 28:4929-4933 (1989). |
Haseloff and Gerlach, “Simple RNA Enzymes with New and Highly Specific Endoribonuclease Activities,” Nature 334:585-591 (1988). |
Hertel et al., “Numbering System for the Hammerhead,” Nucleic Acids Res. 20:3252 (1992). |
Izant and Weintraub, “Constitutive and Conditional Suppression of Exogenous and Endogeneous Genes by Anti-Sense RNA,” Science 229:345-352 (1985). |
Jaeger et al., “Improved Predictions of Secondary Structures for RNA,” Proc. Natl. Acad. Sci. USA 86:7706-7710 (1989). |
Jeffries and Symons, “A Catalytic 13-mer Ribozyme,” Nucleic Acids Research 17:1371-1377 (1989). |
Jellinek et al., “Inhibition of Receptor Binding by High-Affinity RNA Ligands to Vascular Endothelial Growth Factor,” Biochemistry 33:10450-10456 (1994). |
Kashani-Sabet et al., “Reversal of the Malignant Phenotype by an Anti-ras Ribozyme,” Antisense Research & Development 2:3-15 (1992). |
Kim et al., “Inhibition of vascular endothelial growth factor-induced angiogenesis suppresses tumour growth in vivo,” Nature 362:841-844 (1993). |
L'Huillier et al., “Cytoplasmic Delivery of Ribozymes Leads to Efficient Reduction in α-Lactalbumin mRNA Levels in C1271 Mouse,” Embo J. 11:4411-4418 (1992). |
Lieber et al., “Stable High-Level Gene Expression in Mammalian Cells by T7 Phage RNA Polymerase,” Methods Enzymol. 217:47-66 (1993). |
Lisziewicz et al., “Inhibition of Human Immunodeficiency Virus Type 1 Replication by Regulated Expression of a Polymeric Tat Activation Response RNA Decoy as a Strategy for Gene Therapy in AIDS,” Proc. Natl. Acad. Sci. U.S.A. 90:8000-8004 (1993). |
McGarry and Lindquist, “Inhibition of heat shock protein synthesis by heat-inducible antisense RNA,” Proc. Natl. Acad. Sci. USA 83:399-403 (1986). |
Millauer, “High Affintiy VEGF Binding and Developmental Expression Suggest Flk-1 as a Major Regulator of Vasculogenesis and Angiogenesis,” Cell 72:835-846 (1993). |
Milligan and Uhlenbeck, “Synthesis of Small RNAs Using T7 RNA Polymerase,” Methods Enzymol. 180:51-62 (1989). |
Ohkawa et al., “Activities of HIV-RNA Targeted Ribozymes Transcribed From a ‘Shot-Gun’ Type Ribozyme-trimming Plasmid,” Nucleic Acids Symp. Ser. 27:15-16 (1992). |
Ojwang et al., “Inhibition of Human Immunodeficiency Virus Type 1 Expression by a Hairpin Ribozyme,” Proc. Natl. Acad. Sci. USA 89:10802-10806 (1992). |
Perreault et al., “Mixed Deoxyribo- and Ribo-Oligonucleotides with Catalytic Activity,” Nature 344:565-567 (1990). |
Perrotta and Been, “Cleavage of Oligoribonucleotides by a Ribozyme Derived from the Hepatitis δ Virus RNA Sequence,” Biochemistry 31:16-21 (1992). |
Pieken et al., “Kinetic Characterization of Ribonuclease-Resistant 2′-Modified Hammerhead Ribozymes,” Science 253:314-317 (1991). |
Plate, “Vascular endothelial growth factor is potential tumor angiogenesis factor in human gilomas in vivo,” Nature 359:845-848 (1992). |
Rossi et al, “Ribozymes as Anti-HIV-1 Therapeutic Agents: Principles, Applications, and Problems,” Aids Research and Human Retroviruses 8:183-189 (1992). |
Sarver et al., “Ribozymes as Potential Anti-HIV-1 Therapeutic Agents” Science 247:1222-1225 (1990). |
Saville and Collins, “A Site-Specific Self-Cleavage Reaction Performed by a Novel RNA In Neurospora Mitochondria,” Cell 61:685-696 (1990). |
Saville and Collins, “RNA-Mediated Ligation of Self-Cleavage Products of a Neurospora Mitochondrial Plasmid Transcript,” Proc. Natl. Acad. Sci. USA 88:8826-8830 (1991). |
Scanlon et al., “Ribozyme-Mediated Cleavage of c-fos mRNA Reduces Gene Expression of DNA Synthesis Enzymes and Metallothionein,” Proc. Natl. Acad. Sci. USA 88:10591-10595 (1991). |
Scaringe et al., “Chemical synthesis of biologically active oligoribonucleotides using β-cyanoethyl protected ribonucleoside phosphoramidites,” Nucl. Acids Res. 18:5433-5441 (1990). |
Stein and Cheng, “Antisense Oligonucleotides as Therapeutic Agenst—Is the Bullet Really Magical?” Science 261:1004-1288 (1993). |
Taira et al., “Construction of a novel RNA-transcript-trimming plasmid which can be used both in vitro in place of run-off and (G)-free transcriptions and in vivo as multi-sequences transcription vectors,” Nucleic Acids Research 19:5125-5130 (1991). |
Thompson et al., “Improved accumulation and activity of ribozymes expressed from a tRNA-based RNA polymerase III promoter,” Nucleic Acids Research 23:2259-2268 (1995). |
Torrence et al., “Targeting RNA for degradation with a (2′-5′) oligoadenylate-antisense chimera,” Proc. Natl. Acad. Sci. USA 90:1300-1304 (1993). |
Uhlenbeck, “A Small Catalytic Oligoribonucleotide,” Nature 328:596-600 (1987). |
Usman et al.,“Automated Chemical Synthesis of Long Oligoribonucleotides Using 2′-O-Silylated Ribonucleoside 3′-O-Phosphoramidites on a Controlled-Pore Glass Support: Synthesis of a 43-Nucleotide Sequence Similar to the 3′-Half Molecule of an Escherichia coli Formylmethoionine tRNA,” J. Am. Chem. Soc. 109:7845-7854 (1987). |
Usman and Cedergren, “Exploiting the chemical synthesis of RNA,” TIBS 17:334-339 (1992). |
Usman et al., “Chemical modification of hammerhead ribozymes: activity and nuclease resistance,” Nucleic Acids Symposium Series 31:163-164 (1994). |
Ventura et al., “Activation of HIV-Specific Ribozyme Activity by Self-Cleavage,” Nucleic Acids Research 21:3249-3255 (1993). |
Weerasinghe et al., “Resistance to Human Immunodeficiency Virus Type 1 (HIV-1) Infection in Human CD4+ Lymphocyte-Derived Cell Lines Conferred by Using Retroviral Vectors Expressing an HIV-1 RNA-Specific Ribozyme,” Journal of Virology 65:5531-5534 (1994). |
Wincott et al., “Synthesis, deprotection, analysis and purification of RNA and ribozymes,” Nucleic Acids Research 23:2677-2684 (1995). |
Yu et al., “A Hairpin Ribozyme Inhibits Expression of Diverse Strains of Human Immunodeficiency Virus Type 1,” Proc. Natl. Acad. Sci. USA 90:6340-6344 (1993). |
Zabner et al., “Adenovirus-Mediated Gene Transfer Transiently Corrects the Chloride Transport Defect in Nasal Epithelia of Patients with Cystic Fibrosis,” Cell 75:207-216 (1993). |
Zhou et al., “Synthesis of Functional mRNA in Mammalian Cells by Bacteriophage T3 RNA Polymerase,” Mol. Cell. Biol. 10:4529-4537 (1990). |
Andrea D. Branch, A good antisense molecule is hard to find, TIBS, 47-48, Feb. 1998.* |
Eugene Uhlmann and Anusch Peyman, Antisense Oligonucleotides: A new Therapeutic Principle, Chemical Reviews, pp. 545-546, Jun. 1990.* |
Shibuya, M. et al., Nucleotide sequence and expression of a novel human receptor-type tyrosine kinase gene (flt) closely related to the fms family, Oncogene, 5, pp. 519-524, 1990.* |
Stanley T. Crooke, Basic Principles of Antisense Therapeutics, Springer-Verlag, NY, p. 3, Jul. 1998. |