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Gene functional analysis and discovery using randomized or target-specific ribozyme gene vector libraries

a technology of ribozyme and functional analysis, applied in the field of gene functional analysis and discovery using randomized or target-specific ribozyme gene vector libraries, can solve the problem of unexpectedly high expression level of ribozyme gene products

Inactive Publication Date: 2004-05-20
IMMUSOL
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  • Summary
  • Abstract
  • Description
  • Claims
  • Application Information

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Benefits of technology

[0009] In the present invention, a hairpin ribozyme gene library having a randomized target recognition sequence, packaged in a vector which is suitable for high level transduction and expression in a wide variety of cells, is used to identify, isolate, and characterize unknown genes and gene products responsible for detectable phenotypic traits, and to characterize unknown phenotypic effects of known genes. In a preferred embodiment, the vector is an adeno-associated virus vector (AAV). The ribozyme gene is preferably operably linked to a transcriptional promoter that allows for optimal inhibition of target RNA expression in vivo, such as a pol III promoter. The result is an unexpectedly high level of expression of the ribozyme gene products. This efficient expression in turn makes possible the in vivo or in vitro selection of ribozyme genes that are active in vivo, even when the target site is not known.

Problems solved by technology

The result is an unexpectedly high level of expression of the ribozyme gene products.

Method used

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  • Gene functional analysis and discovery using randomized or target-specific ribozyme gene vector libraries
  • Gene functional analysis and discovery using randomized or target-specific ribozyme gene vector libraries
  • Gene functional analysis and discovery using randomized or target-specific ribozyme gene vector libraries

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Embodiment Construction

[0056] 1. Definitions

[0057] Unless defined otherwise, all technical and scientific terms used herein have the same meaning as commonly understood by one of ordinary skill in the art to which this invention belongs. Singleton et al. (1994) DICTIONARY OF MICROBIOLOGY AND MOLECULAR BIOLOGY, 2d ed., John Wiley and Sons (New York); Walker (ed.) (1988) THE CAMBRIDGE DICTIONARY OF SCIENCE AND TECHNOLOGY, Press Syndicate of the University of Cambridge, NY; and Hale and Marham (1991) THE HARPER COLLINS DICTIONARY OF BIOLOGY, Harper Perennial, NY, provide one of skill with a general dictionary of many of the terms used in this invention. Paul (1993) FUNDAMENTAL IMMUNOLOGY, Third Edition Raven Press, New York, N.Y. and the references cited therein provide one of skill with a general overview of the ordinary meaning of many of the virally or immunologically related terms herein. Although any methods and materials similar or equivalent to those described herein can be used in the practice or tes...

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Abstract

The present invention provides a hairpin ribozyme library having a randomized recognition sequence, packaged in a vector and operably linked to a promoter suitable for high level expression in a wide variety of cells. The invention comprises using the library in a variety of selection protocols for identifying, isolating and characterizing known or unknown target RNAs, to reveal the phenotypic effects of such cleavage, and to identify the gene products that produce those phenotypic effects.

Description

A. BACKGROUND OF THE INVENTION[0001] 1. Field of the Invention[0002] This invention relates generally to methods for using hairpin ribozymes to inactivate target RNA molecules. The present invention specifically provides methods for identifying, isolating, and characterizing unknown genes and gene products responsible for detectable phenotypic traits or for characterizing unknown phenotypic effects of known genes, and methods of inactivating target RNAs. Compared to other known ribozymes, the hairpin ribozyme has been discovered to be uniquely effective as a randomized antisense tool.[0003] 2. Related Art[0004] A ribozyme is an RNA molecule that catalytically cleaves other RNA molecules. Different kinds of ribozymes have been described, including group I ribozymes, hammerhead ribozymes, hairpin ribozymes, RNAse P, and axhead ribozymes. See Castanotto et al. (1994) Advances in Pharmacology 25:289-317 for a general review of the properties of different ribozymes.[0005] The general fea...

Claims

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Application Information

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Patent Type & Authority Applications(United States)
IPC IPC(8): C12N9/00C12N15/10C12N15/113C12Q1/68C12N15/09C12Q1/6811C12Q1/6897
CPCA01K2217/05C12N15/1048C12N15/113C12Q1/6897C12N2799/025C12Q1/6811C12N2310/122
Inventor BARBER, JACK R.WELCH, PETERYEI, SOONPINTRITZ, RICHARD
Owner IMMUSOL
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