Generation of Ribozyme Libraries
Antisense technology provides a platform for drug discovery as well as clinical therapeutic applications. Antisense agents (such as DNA, RNA, and ribozymes) bind to complementary regions on host nucleotides and thereby silence the expression of target genes. Given that target genes and their RNA transcripts can be comprised of thousands of nucleotide bases, and given that not all those nucleotides are equally accessible to antisense inhibition, it remains a challenge to design optimal molecules that interact with those target sequences. This technology discloses simple steps to generate antisense libraries directed against the accessible sites of RNA target transcripts and how such directed libraries can be used to make ribozymes against those target sites.
Companies interested in antisense technology need a competitive edge in the design and optimization of nucleic acid-based therapeutics. In developing ribozyme therapeutics, many companies employ either a trial and error approach or use simple thermodynamic modeling software, both of which are prone to error. This technology reduces the complexity of the molecular engineering process, thereby allowing company to rapidly develop an optimal ribozyme for a particular need. Such an approach can be used by a company either to improve development of its own products, or to rapidly meet the synthesis demands of contracting partners.
Stage of Development
The U.S. patent 6,586,180 has been issued, foreign counterparts (under PCT/US99/06742) are in prosecution, and continuing applications have been filed with publication numbers US 2003/0119041 A1, US 2003/0108939 A1, and US 2004/0077082 A1. Extensive post-patent development has gone into this technology to demonstrate its clinical feasibility. This technology is available for licensing under either exclusive or non-exclusive terms.
*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):5093-5101(Nov 15, 1998).
*Wang L, et. al. Progress in the delivery of therapeutic oligonucleotides: organ/cellular distribution and targeted delivery of oligonucleotides in vivo, Antisense Nucleic Acid Drug Dev. 13(3):169-189 (2003).
Zhidong Chen, Ramesh Prakash, Richard (Dick) Koehn, Michael Pierce, Duane Ruffner
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