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Seven RNAi-Related Patent Applications Published by the US Patent Office


Title: Compositions and Methods for Tissue-Specific or Inducible Inhibition of Gene Expression. Number: 20040198967. Filed: Sept. 4, 2003. Lead Inventor: Danny Allen, College of the Holy and Undivided Trinity of Queen Elizabeth.

The patent application, its abstract states, covers “methods and reagents ... for the tissue specific, cell specific and/or inducible expression of RNAi. The invention may be used to downregulate the expression of endogenous or exogenous RNAs targeted by the RNAi in cells, animals and/or plants,” the abstract notes.

Title: Endosomolytic Polymers. Number: 20040198687. Filed: April 1, 2004. Lead Inventor: David Rozema, Mirus.

According to the patent application’s abstract, the invention comprises “pH-sensitive endosomolytic polymers, delivery particles containing pH-sensitive endosomolytic polymers. The described particles are capable of delivering polynucleotides to cells from the peripheral circulation with subsequent release from endosomes.”

The abstract adds that “the endosomolytic polymers are inactive outside the cell but disrupt membranes upon exposure to an acidified endosomal compartment.”

Title: RNA Interference-Mediated Inhibition of Placental Growth Factor Gene Expression Using Short-Interfering Nucleic Acid. Number: 20040198682. Filed: Oct. 10, 2003. Lead Inventor: James McSwiggen, Sirna Therapeutics.

“The … invention concerns methods and reagents useful in modulating placental growth factor … gene expression in a variety of applications, including use in therapeutic, diagnostic, target validation, and genomic discovery applications,” the patent application’s abstract states. “Specifically, the invention relates to small nucleic acid molecules, such as short interfering nucleic acid, short interfering RNA, double-stranded RNA, micro-RNA, and short hairpin RNA molecules capable of mediating RNA interference against PGF gene expression and/or activity. The small nucleic acid molecules are useful in the diagnosis and treatment of cancer, proliferative diseases, and any other disease or condition that responds to modulation of PGF expression or activity.”

Title: Stabilized Polynucleotides for Use in RNA Interference. Number: 20040198640. Filed: April 2, 2003. Lead Inventor: Devin Leake, Dharmacon.

The patent application, its abstract states, covers “methods and compositions for performing RNA interference comprising a wide variety of stabilized polynucleotides suitable for use in serum-containing media and for in vivo applications, such as therapeutic applications. These polynucleotides permit effective and efficient applications of RNA interference to applications such as diagnostics and therapeutics through the use of one or more modifications including orthoesters, terminal conjugates, modified linkages and 2’modified nucleotides.”

Title: Methods and Compositions for Inhibiting the Function of Polynucleotide Sequences. Number: 20040198690. Filed: April 30, 2004. Lead Inventor: C. Satishchandran, Wyeth (Nucleonics).

The invention comprises “a therapeutic composition for inhibiting the function of a target polynucleotide sequence in a mammalian cell, [which] includes an agent that provides to [the] cell an at least partially double-stranded RNA molecule comprising a polynucleotide sequence of at least about 200 nucleotides in length [with] said polynucleotide sequence being substantially homologous to a target polynucleotide sequence,” the patent application’s abstract states.

“This RNA molecule desirably does not produce a functional protein,” the abstract adds. “The agents useful in the composition can be RNA molecules made by enzymatic synthetic methods or chemical synthetic methods in vitro, or made in recombinant cultures of microorganisms and isolated. … Alternatively, [the agents] can be capable of generating the desired RNA molecule in vivo after delivery to the mammalian cell. In methods of treatment of prophylaxis of virus infections, other pathogenic infections or certain cancers, these compositions are administered in amounts effective to reduce or inhibit the function of the target polynucleotide sequence, which can be of pathogenic origin or produced in response to a tumor or other cancer, among other sources.”

Title: Methods and Means for Improving Retroviral Integration. Number: 20040197913. Filed: Sept. 8, 2003. Lead Inventor: Mark James O’Connor, KuDos Pharmaceuticals.

The patent application, its abstract states, covers “retroviral integration … promoted in mammalian cells by inhibition of RAD52 DNA-binding activity, e.g. using RNAi.

“This is useful in gene therapy, especially ex vivo,” the abstract notes. “Retroviral integration is inhibited by increasing mammalian RAD52 DNA-binding activity, useful in inhibiting retroviruses.”

Title: Methods for Using Primers That Encode One Strand of a Double-Stranded Promoter. Number: 20040197802. Filed: Nov. 21, 2003. Lead Inventor: Gary Dahl, Epicentre Technologies.

“The … invention provides methods, compositions and kits for using an RNA polymerase for making transcription products corresponding to a target sequence by obtaining circular single-stranded DNA transcription substrates using a promoter primer that encodes one strand of a double-stranded promoter,” the patent application’s abstract states. “The invention has broad applicability for research, diagnostic and therapeutic applications, such as preparing cDNA corresponding to mRNA, making sense or anti-sense probes, detecting gene- or organism-specific sequences, or making RNAi.”


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