Title: Frog Prince, a Transposon Vector for Gene Transfer in Vertebrates
Filed: May 26, 2005 PCT Filed: May 30, 2003
Lead Inventor: Csaba Miskey, Max Delbruck Center for Molecular Medicine
"The present invention relates to a transposon-based DNA integration system comprising (a) a transposon which is devoid of a polynucleotide encoding a functional transposase and which comprises a polynucleotide of interest, wherein the transposon comprises inverted repeats having a degree of identity with the repeats within [a specific sequence] and its inverted repeat, respectively, of at least 90%; and (b) a transposase having at its N-terminus a DNA binding domain comprising the sequences of [specific sequences]; or (c) a polynucleotide encoding the transposase of (b)," the patent application's abstract states. "The present invention further relates to a method of transferring a polynucleotide of interest into cells of a vertebrate comprising the step of introducing the transposon-based DNA integration system of the invention into said cells. In addition, the invention relates to a method of effecting RNAi comprising (a) stably introducing a transposon comprising an expression cassette expressing a short interfering RNA and a selectable marker gene as part of the transposon-based DNA integration system of the invention into a cell; (b) selecting for cells expressing the selectable marker; and (c) assessing whether the transcription/translation of the desired gene is effected by RNAi. A further embodiment of the invention is a method of gene trapping genes comprising the steps of (a) introducing the transposon-based DNA integration system of the invention into a cell; and (b) assessing for the expression of a selectable marker wherein expression of a selectable marker is indicative of integration of the transposon into a transcibed gene of the cell."
Title: RNA Interference-Mediated Inhibition of MAP Kinase Gene Expression Using Short Interfering Nucleic Acid
Filed: Aug. 20, 2004
Lead Inventor: James McSwiggen, Sirna Therapeutics
"This invention relates to compounds, compositions, and methods useful for modulating mitogen activated protein kinase (MAP kinase) 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 MAP kinase gene expression and/or activity by RNA interference using small nucleic acid molecules. In particular, the … 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 MAP kinase genes, such as Jun amino-terminal kinase (e.g., JNK-1, JNK-2), p38 (MAPK 14), ERK (e.g., ERK-1, ERK-2) and/or c-Jun."
Title: Double-Stranded RNA Structures and Constructs, and Methods for Generating and Using the Same
Filed: Jan. 31, 2005 PCT Filed: July 31, 2003
Lead Inventor: Catherine Pachuk, Nucleonics
"The present invention relates to novel double-stranded RNA structures and dsRNA expression constructs, methods for generating them, and methods of utilizing them for silencing genes," the patent application's abstract states. "Desirably, these methods specifically inhibit the expression of one or more target genes in a cell or animal (e.g., a mammal such as a human) without inducing toxicity. These methods can be used to prevent or treat a disease or infection by silencing a gene associated with the disease or infection. The invention also provides methods for identifying nucleic acid sequences that modulate a detectable phenotype, such as the function of a cell, the expression of a gene, or the biological activity of a target polypeptide," the abstract adds.
Title: Methods and Compositions for Delivering siRNA in Mammalian Cells
Filed: April 26, 2004
Lead Inventor: Gilles Divita, Centre National de la Recherche Scientifique
According to the patent application's abstract, the invention relates to a "complex comprising a peptide carrier of SEQ ID NO:1 GALFLGFLGAAGSTMGAWSQPKR.sub.1KRKVR and an appropriate siRNA, wherein R represents any amino acid residue and more preferably K or S, R is null or represents one of the following groups: cysteamide, cysteine, thiol, amide, linear or ramified C-C alkyl optionally substituted, primary or secondary amine, osidic derivative, lipid, phospholipid or cholesterol and said siRNA is selected to silence a target mRNA."
Title: Methods and Compositions for Inhibiting the Function of Polynucleotide Sequences
Filed: Oct. 29, 2004
Lead Inventor: Chandrasekhar Satishchandran, Nucleonics
The patent application, its abstract states, covers "a therapeutic composition for inhibiting the function of a target polynucleotide sequence in a mammalian cell [that] includes an agent that provides to a mammalian cell an at least partially double-stranded RNA molecule comprising a polynucleotide sequence of at least about 200 nucleotides in length, said polynucleotide sequence being substantially homologous to a target polynucleotide sequence. This RNA molecule desirably does not produce a functional protein. 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 therefrom, or alternatively, can be capable of generating the desired RNA molecule in vivo after delivery to the mammalian cell," the abstract notes. "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: Compositions and Methods for Topical Delivery of Oligonucleotides
Filed: April 21, 2005
Lead Inventor: Sujatha Dokka, Isis Pharmaceuticals
The invention, the patent application's abstract states, "relates to compositions and methods which enhance the delivery of nucleic acids and other nucleosidic moieties via topical routes of administration. The invention relates to the use of an aqueous solution to preferentially deliver nucleic acids preferentially to hair follicles. The invention relates to a method of inhibiting hair growth comprising administration of a nucleic acid preferentially to a hair follicle."