Circular, Hairpin, Circular/Hairpin, Lariat, and Hairpin-Lariat Hammerhead Ribozymes
Ribozymes are RNA molecules that can catalytically cleave nucleic acids, thereby suppressing expression of unwanted gene products such as disease causing proteins. This technology includes various configurations of the hammerhead-class of ribozymes that contain circular, hairpin, circular-hairpin, lariat, and hairpin-lariat ribozyme domain structures. They are catalytically active and provide advantages not present in the wild-type hammerhead configurations. Their production can be accomplished through a variety of techniques, including enzymatic and chemical as well as by fully automated synthesis, thus they are not specific for any particular method of synthesis. The properties of these novel configurations could prove useful as research tools or in therapies involving oligonucleotide-based drug development.Benefits
This invention describes new types of ribozymes that may be useful as either research tools or therapeutic agents. The DNA/RNA market is forecast to generate $1.2 billion by 2010. 45% of the market is set to be generated by ophthalmic therapies. Behind ophthalmology, oncology will dominate the therapeutic focus of the DNA/RNA market, and is set to generate 31.2% of 2010 sales.
Stage of Development
U.S. utility patent 6,307,041 has been issued from the USPTO, and an international application (PCT/US99/06770, publication #WO 99/50277) is in current prosecution.
Additional Info
*Pierce ML and Ruffner DE, Construction of a directed hammerhead ribozyme library: towards the identification of optimal target sites for antisense-mediated gene inhibition, Nucleic Acids Res 26(22): pp 5093-101, (Nov 15, 1998)
*Wang L and Ruffner DE, Novel configurations of the hammerhead ribozyme: increased activity and reduced magnesium ion requirement, Biochem Biophys Res Commun 250(3): pp 711-9 (Sep 29, 1998)
*Wang L and Ruffner DE, An ultraviolet crosslink in the hammerhead ribozyme dependent on 2-thiocytidine or 4-thiouridine substitution, Nucleic Acids Res 25(21): pp 4355-61 (Nov 1, 1997)
Inventor(s): Laixin Wang, Duane Ruffner
Type of Offer: Licensing
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