Chemistry: molecular biology and microbiology – Vector – per se
Patent
1997-01-09
1999-11-30
Elliott, George C.
Chemistry: molecular biology and microbiology
Vector, per se
435 6, 435 9131, 514 44, 536 231, 536 232, 536 241, 536 255, C07H 2102, C07H 2104, A61K 4800
Patent
active
059941243
ABSTRACT:
The invention is drawn to a chimeric ribozyme molecule that comprises a U1 snRNA component and a ribozyme component where the chimeric ribozyme targets an mRNA in the spliceosome in the nuclease of a cell.
REFERENCES:
patent: 5695938 (1997-12-01), Rossi
"Novel Approaches Towards the Nuclear Expression of Ribozyme and Antisense RNA Genes in Transgenic Mice" by Sharp et al., pp. 185-194, 1996.
"Synthesis of Chimeric RNAs between U6 small nuclear RNA and (-)sTRSV and Analysis of Their Cleavage Activities Against the Substrate RNA"by Tani et al.; Nucleic Acids Symposium Series vol. 27; 19-20, 1992.
"Functional Characterization of Ribozymes Expressed Using U1 and T7 Vectors for the Intracellular, Cleavage of ANF mRNA"by De Young et al.; Biochemistry 1994, 33 pp. 12127-12138.
"Self-Cleaving Motifs are Found in Close Proximity to the Sites Utilized for U16 snoRNA Processing"by Prislei et al.; Gene 163 (1995) pp. 221-226.
"Retroviral Vectors Designed for Targeted Expression of RNA Polymerase III-Driven Transcripts: A Comparative Study" by Ilves et al.; Gene. 171 (1996) pp. 203-208.
"Efficient Hammerhead Ribozyme-Mediated Cleavage of the Structured Hepatitis B Virus Encapsidation Signal In Vitro and in Cell Extracts, but not in Intact Cells" by Beck et al.; Nucleic Acids Research, 1995, vol. 23, No. 24, pp. 4954-4962.
Orkin et al "Report and Recommendations of the Panel to Assess the NIH Investment in Research on Gene Therapy", 1995.
Stull et al "Antigene Ribozyme and Aptamer Nucleic Acid Drugs: Progress and Prospects" Pharmaceutical Research vol. 12 No. 4; 465-483, 1995.
Burke, J., "Clearing The Way For Ribozymes" Nature Biotechnology vol. 15: 414, May 1997.
Tani, T., et al "Activity of chimeric RNAs of U6 snRNA and (-) STRSV in the Cleavage of a substrate RNA."NAR vol. 20 No. 12: 2991-2996, 1992.
Michienzi et al U1 Small Nuclear RNA chimeric ribozymes with substrate specifility for the Rev pre-mRNA of human immunodeficiency virus . PNAS vol. 93: 7219-7224, Jul. 1996.
Elliott George C.
McGarry Sean
Universita Degli Studi Di Roma "La Sapienza"
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