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Four New RNAi-Related Patent Applications Published By US Patent Office: Apr 16, 2004

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Title: Nucleozymes. Number: 6,713,456. Filed: June, 2, 1995. Lead Inventor: Nassim Usman, Massachusetts Institute of Technology.

The invention comprises “nucleozymes containing ribonucleotides and deoxyribonucleotides, or nucleic acid analogues,” according to the patent’s abstract. “Nucleozymes have catalytic activity and are significantly more resistant to degradation than their all-RNA ribozyme counterparts.”

It adds that the patent also covers “methods for preparing the nucleozymes, along with methods of using nucleozymes, i.e. as therapeutic agents.”


Title: Process for Delivery of Polynucleotides to the Prostate. Number: 20040067907. Filed: Oct. 6, 2003. Lead Inventor: James Hagstrom, Mirus.

The patent application, its abstract states, covers a “system for providing in vivo delivery of polynucleotides to mammalian prostate cells using an intravascular administration route.”

The system involves inserting an “injection solution containing … polynucleotide[s] into the” mammalian vasculature, according to the patent application. The abstract notes that “insertion of the injection solution at an appropriate rate increases the volume of extravascular fluid in the tissue, thereby facilitating delivery of the polynucleotide to the cell.”

The application specifically claims the delivery of an RNAi-inducing polynucleotide into prostate cells of mammals that have benign prostatic hyperplasia and/or prostate cancer.


Title: Model of Transmissible Spongiform Encephalopathic (TSE) Disease. Number: 20040067510. Filed: Oct. 2, 2003 (PCT Filed: Feb. 20, 2002). Inventor: Toby James Gibson, European Molecular Biology Laboratory.

The invention “relates to the mechanism of transmission of spongiform encephalopathics,” the patent application’s abstract states. It “provides simple diagnostic tests for animals that are infected with transmissible spongiform encephalopathic disease.”

The test involves screening for the “presence of mutant non-viral retroelement nucleic acid molecules, wherein the presence of said mutant retroelement nucleic acid molecules is indicative of infection,” the abstract states. “The invention also provides methods for the treatment of TSE disease by administering a therapeutically-effective amount of a compound that is effective to counteract the effect of a non-viral mutant retroelement nucleic acid molecule in inducing TSE disease.”

The application specifically claims a therapeutic compound for TSE disease that is an “RNAi molecule … targeted to the non-viral mutant retroelement nucleic acid molecule.”


Title: Developmental Mutations in Zebrafish. Number: 20040068763. Filed: March 28, 2003. Lead Inventor: Nancy Hopkins, Massachusetts Institute of Technology.

According to the patent application’s abstract, “the invention features novel zebrafish nucleic acid and amino acid molecules, and zebrafish containing mutations in important developmental genes. In addition, the invention features the use of these nucleic acid and amino acid molecules in methods of diagnosing, preventing, and treating a variety of mammalian diseases and developmental disorders,” the abstract states.

“Furthermore, [a] zebrafish mutant for nucleic acid or amino acid molecule of the invention may be used in screens for compounds that modulate the development of an organism as a whole or of specific tissues or organs with an organism,” the abstract adds. “In particular, the … invention features novel nucleic acid sequences involved, e.g. in kidney development and kidney disorders.”

The application claims “a method of treating or preventing a kidney disorder in an organism … [by] contacting said organism with a therapeutically effective amount of a nucleic acid,” specifically a double-stranded RNA molecule,”[that is] sufficient to elicit an alteration in expression of a … nucleic acid sequence in said organism, and wherein said alteration in the level of expression treats or prevents a kidney disorder.”