Title: Delivery Vectors for Short Interfering RNA, microRNA, and Antisense RNA
Filed: March 9, 2005
Lead Inventor: Ulrich Thomann
"This invention relates to compositions and methods for transcription and expression of nucleic acids into organisms," the patent application's abstract states. "In particular, the invention comprises a tRNA vector system to deliver and express short interfering nucleic acid, small interfering and microRNA, and antisense into an organism with high efficiency. The compositions further provide expression of nucleic acids to perform as therapeutic compounds in organisms."
Title: Identification of Toxic Nucleotide Sequences
Filed: Jan. 21, 2005
Lead Inventor: Yuriy Fedorov, Dharmacon
According to its abstract, the patent application relates to "toxic nucleic acid sequences and methods for identifying, using, and screening libraries for them, including in silico screening.
"Compositions of the invention comprise unimolecular and double stranded polynucleotides comprising at least one toxicity region," the abstract notes. "Toxic sequences of the invention include A/G UUU A/G/U, G/C AAA G/C, and/or GCCA, NUUU, wherein N is any nucleotide, or complements thereof. The invention also provides a method of inducing a toxic response in a cell, comprising introducing into the cell a unimolecular or double stranded polynucleotide comprising at least one toxicity region comprising a sequence selected from the group consisting of A/G UUU A/G/U, G/C AAA G/C, GAAT, and GCCA, NUUU, wherein N is any nucleotide, or a complement of any of the foregoing, wherein said unimolecular or double-stranded polynucleotide is at least 5 base pairs long and is comprises a sense and antisense region that are at least substantially complementary," the abstract states.
Title: RNA Interference-mediated Inhibition of Vascular Cell Adhesion Molecule Gene Expression Using Short Interfering Nucleic Acid
Filed: Aug. 16, 2004
Lead Inventor: Ivan Richards, Sirna Therapeutics
"This invention relates to compounds, compositions, and methods useful for modulating vascular cell adhesion molecule gene expression using short interfering nucleic acid molecules," the patent application's abstract states. "This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of vascular cell adhesion molecule gene expression and/or activity by RNA interference using small nucleic acid molecules. In particular, the instant invention features small nucleic acid molecules, such as short interfering nucleic acid, short interfering RNA, double-stranded RNA, microRNA, and short hairpin RNA molecules and methods used to modulate the expression of vascular cell adhesion molecule genes, such as vascular cell adhesion molecule-1 (VCAM-1)."
Title: Targeted Delivery of RNA Interference Molecules
Filed: Feb. 22, 2005
Lead Inventor: James Watson, Genesis Research and Development
"Compositions for the treatment and/or prevention of IgE-mediated disorders in a mammal by means of RNA interference" are covered under the patent application, its abstract states, "together with methods for the use of such compounds. The inventive compositions comprise a binding agent that specifically binds to a target internalizable antigen that is expressed on the surface of a target cell of interest and a genetic construct that is capable of expressing a small interfering nucleic acid molecule that suppresses expression of a target gene within the target cell, whereby, after binding to the target antigen, the binding agent and genetic construct are internalized into the cell, and the genetic construct released."
Title: Delivery of Genes Encoding Short Hairpin RNA Using Receptor-Specific Nanocontainers
Filed: March 12, 2004
Lead Inventor: William Pardridge, University of California, Los Angeles
According to the patent application's abstract, the invention covers "receptor-specific nanocontainers … used to deliver a gene that encodes short hairpin RNA to cells having a given receptor. Once inside the cell, the gene expresses short hairpin RNA that includes a nucleotide sequence that is antisense to at least a portion of an oncogene, such as human epidermal growth factor receptor (EGFR) mRNA, or other disease-causing nucleotide sequence. The short hairpin RNA is converted, in the cellular cytoplasm, into short RNA duplexes that are effective in deactivating (knocking down) the oncogenic or disease causing gene," the abstract states.