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Five RNAi-Related Patent Applications Published by the US Patent Office: Dec 3, 2004

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Title: Inhibition of Hepatitis B Virus Replication by RNA Interference. Number: 20040235775. Filed: May 19, 2004. Lead Inventor: Hsiang-Fu Kung, University of Hong Kong.

“The … invention relates to the inhibition of hepatitis B virus replication by RNA molecules … [that] are double-stranded ribonucleic acid molecules,” according to the patent application’s abstract. “Specifically, the invention relates to small interfering RNAs which are double-stranded RNAs that direct the sequence-specific degradation of messenger RNA in mammalian cells.”

The abstract adds that “the invention relates to development of a new anti-HBV therapy by inhibition of hepatitis B virus replication using stably-expressed short hairpin RNAs [that] degrade HBV pregenomic RNA and message RNAs.”


Title: Genetic Suppression and Replacement. Number: 20040234999. Filed: Aug. 29, 2003. Lead Inventor: Gwenyth Jane Farrar, Trinity College Dublin.

“Methods and agents for suppressing expression of a mutant allele of a gene and providing a replacement nucleic acid are provided” under the patent application, its abstract states. “The methods of the invention provide suppression effectors such as, for example, antisense nucleic acids, ribozymes, or RNAi, that bind to the gene or its RNA. The invention further provides for the introduction of a replacement nucleic acid with modified sequences such that the replacement nucleic acid is protected from suppression by the suppression effector,” the abstract notes.

“The replacement nucleic acid is modified at degenerate wobble positions in the target region of the suppression effector and thereby is not suppressed by the suppression effector,” the abstract adds. “In addition, by altering wobble positions, the replacement nucleic acid can still encode a wild type gene product. The invention has the advantage that the same suppression strategy could be used to suppress, in principle, many mutations in a gene. Also disclosed is a transgenic mouse that expresses human rhodopsin (modified replacement gene) and a transgenic mouse that expresses a suppression effector targeting rhodopsin. Also disclosed [is] intraocular administration of siRNA.”


Title: Methods of Inhibiting Gene Expression by RNA Interference. Number: 20040234504. Filed: Dec. 18, 2003. Lead Inventor: Inder Verma, Salk Institute for Biological Sciences.

“The invention provides a lentiviral vector capable of inhibiting the expression of at least one target gene,” the patent application’s abstract states.

“A lentiviral vector of the invention encompasses a first nucleic acid sequence derived from a target gene transcript and a second nucleic acid sequence corresponding to the reverse complement of said first nucleic acid sequence,” it adds. “A lentiviral vector of the invention capable of inhibiting the expression of a target gene is useful in therapeutic applications to inactivate disease-associated transcripts and thereby reduce the severity of inherited metabolic, infectious or malignant conditions. Methods for inhibiting one or more target genes in a cell, as well as methods for producing a non-human mammal in which the expression of one or more target genes is inhibited, also are provided by the present invention.”


Title: Methods of Inhibiting Expression of a Target Gene in Mammalian Cells. Number: 20040235764. Filed: Oct. 3, 2003. PCT Filed: Feb. 27, 2002. Lead Inventor: Eric Billy, Novartis.

The patent application’s abstract states that the invention comprises “a method … for inhibiting gene expression in a mammalian cell based on dsRNA, as well as construct useful for carrying out the invention and resulting cells and transgenic mammals.”


Title: Silencing of Gene Expression by siRNA. Number: 20040235171. Filed: June 22, 2004. PCT Filed: July 17, 2002. Inventor: Ann Josephine Milner, University of York.

“The ... invention relates to a method of selective post-transcriptional silencing in a mammalian cell of the expression of an exogenous gene of viral origin,” the patent application’s abstract states. “The method involves comprises introducing an siRNA construct into a mammalian cell where the siRNA construct is homologous to a part of the mRNA sequence of the exogenous gene. The invention also comprises an siRNA construct with a nucleotide sequence which is homologous to a part of the mRNA sequence of an exogenous gene of viral origin.”