Ribozyme gene therapy: Applications for molecular medicine

Trends in Molecular Medicine

Volumn 7, Issue 5, 2001, Pages 221-228

  Lewin, Alfred S ^a   Hauswirth, William W ^a  

^a UNIVERSITY OF FLORIDA   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

RIBONUCLEASE P; RIBOZYME;

ALLELE; CANCER; DOWN REGULATION; GENE THERAPY; GENETIC VARIABILITY; IN VITRO STUDY; INTRON; METABOLIC DISORDER; REVIEW; VIRUS INFECTION;

ACQUIRED IMMUNODEFICIENCY SYNDROME; ALLELES; ANIMALS; BASE SEQUENCE; CODON; DEPENDOVIRUS; DNA REPAIR; DOWN-REGULATION; GENE THERAPY; HUMANS; INTRONS; MODELS, BIOLOGICAL; MOLECULAR SEQUENCE DATA; NEOPLASMS; RNA, CATALYTIC; TRANSDUCTION, GENETIC;

HARNESS;

EID: 0034839007     PISSN: 14714914     EISSN: None     Source Type: Journal    
DOI: 10.1016/S1471-4914(01)01965-7     Document Type: Review

References (54)

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4. A general 2-metal-ion mechanism for catalytic RNA
5. The effect of structure in a long target RNA on ribozyme cleavage efficiency
6. Synthesis of 2′-modified nucleotides and their incorporation into hammerhead ribozymes
7. Pharmacokinetics and tissue distribution of a ribozyme directed against hepatitis C virus RNA following subcutaneous or intravenous administration in mice
8. Improved cationic lipid formulations for in vivo gene therapy
9. Immune responses to adenovirus and adeno-associated virus in humans
10. Latent adeno-associated virus infection elicits humoral but not cell-mediated immune responses in a nonhuman primate model
11. Expression of small, therapeutic RNAs in human cell nuclei
12. Factors governing the activity in vivo of ribozymes transcribed by RNA polymerase III
13. Ribozyme rescue of photoreceptor cells in a transgenic rat model of autosomal dominant retinitis pigmentosa
14. Recent advances in inducible gene expression systems
15. The expression cassette determines the functional activity of ribozymes in mammalian cells by controlling their intracellular localization
16. mRNA localization signals can enhance the intracellular effectiveness of hammerhead ribozymes
17. Ribozyme as an approach for growth suppression of human pancreatic cancer
18. A novel allosterically trans-activated ribozyme, the maxizyme, with exceptional specificity in vitro and in vivo
19. Therapeutic efficacy of an adenovirus-mediated anti-H-ras ribozyme in experimental bladder cancer
20. Antitumor and antimetastatic activity of ribozymes targeting the messenger RNA of vascular endothelial growth factor receptors
21. Acute toxicology and pharmacokinetic assessment of a ribozyme (ANGIOZYME) targeting vascular endothelial growth factor receptor mRNA in the cynomolgus monkey
22. Pharmacokinetics of an antiangiogenic ribozyme (Angiozyme) in the mouse
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27. Gene therapy targeting cord blood-derived CD34(+) cells from HIV-exposed infants: Preclinical studies
28. 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
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30. Preclinical characterization of an anti-tat ribozyme for therapeutic application
31. A phase I trial of autologous CD34+ hematopoietic progenitor cells transduced with an anti-HIV ribozyme
32. Clinical aspects of ribozymes as therapeutics in gene therapy
33. + peripheral blood lymphocytes in HIV discordant identical twins
34. Aggregation and motor neuron toxicity of an ALS-linked SOD1 mutant independent from wild-type SOD1
35. Hammerhead ribozymes selectively suppress mutant type I collagen mRNA in osteogenesis imperfecta fibroblasts
36. Ribozyme rescue of photoreceptor cells in P23H transgenic rats: Long-term survival and late-stage therapy
37. A novel SV40-based vector successfully transduces and expresses an α1-antitrypsin ribozyme in a human hepatoma-derived cell line
38. Strategems in vitro for gene therapies directed to dominant mutations
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40. Induction of wild-type p53 activity in human cancer cells by ribozymes that repair mutant p53 transcripts
41. Evaluating group I intron catalytic efficiency in mammalian cells
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44. Group II introns designed to insert into therapeutically relevant DNA target sites in human cells
45. RNase P in research and therapy
46. Requirements for cleavage by a modified RNase P of a small model substrate
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48. Spliceosome-mediated RNA trans-splicing as a tool for gene therapy
49. Collagen 17A1 gene correction using spliceosome mediated RNA trans-splicing (SMaRT™) technology
50. Repair of CFTR mRNA by spliceosome-mediated RNA trans-splicing
51. Human gene targeting by viral vectors
52. In vivo site-directed mutagenesis of the factor IX gene by chimeric RNA/DNA oligonucleotides
53. Gene correction in hematopoietic progenitor cells by homologous recombination
54. Expression of ribozymes in gene transfer systems to modulate target RNA levels