Abstract: The subject matters of this invention are a sequence of double-stranded RNA: ATN-RNA, intervention using interference RNA (iRNAi), use of a sequence of double-stranded RNA: ATN-RNA, a method of treating a brain tumor and a method of inhibiting a brain tumor cells which express tenascin, a kit for inhibiting cancer cell which expresses tenascin and a method for a kit preparation in a brain tumor therapy. Malignant gliomas preferentially express a number of surface markers that may be exploited as therapeutic targets, including tenascin-C, an extracellular matrix glycoprotein that is ubiquitously expressed by malignant gliomas and probably contributes to tumor cell adhesion, invasion, migration and proliferation. For tenascin-C inhibition, RNA interference intervention (iRNAi) approach have been applied.

Abstract: The present invention relates to novel methods for modulating the activity of p53 tumor suppressor protein by affecting p53 translational regulation. More specifically, the invention relates to novel methods for modulating p53 mRNA translation in a cell by affecting a function of a p53 5?-untranslated region (5?UTR), including its interaction with proteins such as Ribosomal Protein L26 (RPL26), nucleolin, and p53. The invention also relates to the use of these methods for treating cancer, neurodegenerative disorders and minimizing the negative effects of cellular stresses.

Abstract: Oncogenic Ras-driven cancer is treated with agent by interrupting pathway comprising activation of Akt by oncogenic Ras, activated Akt causing phosphorylation of eNOS at S1177 site of eNOS to provide activated eNOS in cancer cells and activated eNOS causing activation of wildtype Ras by nitrosylation thereof at C118 to provide GTP-bound activated wildtype H and N Ras. L-NAME can be orally administered to interrupt this pathway. Wortmannin can be administered intravenously to interrupt this pathway. Novel siRNAs are disclosed useful to interrupt said pathway.

Abstract: Efficient sequence specific gene silencing is possible through the use of siRNA technology. By selecting particular siRNAs by rational design, one can maximize the generation of an effective gene silencing reagent, as well as methods for silencing genes. Methods, compositions, and kits generated through rational design of siRNAs are disclosed.

Abstract: The present invention provides HIV-derived lentivectors which are multiply modified to create highly safe, efficient, and potent vectors for expressing transgenes for gene therapy. The lentiviral vectors comprise various combinations of an inactive central polypurine tract, a stuffer sequence, which may encode drug susceptibility genes, and a mutated hairpin in the 5? leader sequence that substantially abolishes replication. These elements are provided in conjunction with other features of lentiviral vectors, such as a self-inactivating configuration for biosaftey and promoters such as the EF1? promoter as one example. Additional promoters are also described. The vectors can also comprise additional transcription enhancing elements such as the wood chuck hepatitis virus post-transcriptional regulatory element.

Abstract: The present invention relates to reverse micelle system based on sterols, acylglycerols, phospholipids or sphingolipids and nucleic acids. The reverse micelle system of the invention is able to cross mucosa and cellular membranes. It thus allows vectorization of nucleic acids to target sites. It is advantageously useful in the pharmaceutical and dietetic fields.

Abstract: This invention is directed to mutated Activin A type I receptor proteins (ACVR1) and isolated nucleic acids encoding same. The invention also relates to compositions and methods for siRNA-based regulation of mutated ACVR1 expression in the treatment of Fibrodysplasia Ossificans Progressiva (FOP).

Abstract: Compounds, compositions and methods are provided for modulating the expression and function of small non-coding RNAs. The compositions comprise oligomeric compounds, targeted to small non-coding RNAs. Methods of using these compounds for modulation of small non-coding RNAs as well as downstream targets of these RNAs and for diagnosis and treatment of disease associated with small non-coding RNAs are also provided.

Abstract: A major object of the present invention is to provide a method for producing induced pluripotent stem cells with low tumorigenesis potential and high induction efficiency. The invention provides a method for producing induced pluripotent stem cells comprising the step of introducing one or more nucleic acids that facilitate expression of at least one gene selected from the group consisting of NANOG, SOX2, OCT3/4, KLF4, LIN28, and c-MYC into somatic cells.

Abstract: There is provided an expression cassette comprising a 3?-UTR cDNA library fragment, mammalian cells transfected with the expression cassette, and kits comprising the same. Furthermore, methods for identifying target genes for microRNAs are provided that utilize the expression cassette hereof.

Abstract: The invention provides a nucleic acid molecule having a binding affinity to a rodent-derived IgG antibody, which can be prepared easier than an antibody and has a binding affinity equivalent or superior to that of an antibody, a binder using the nucleic acid molecule, a detection reagent, and a detection kit. The nucleic acid molecule of the invention has a binding affinity to a rodent-derived IgG antibody and has a dissociation constant of 1 ?M or less. The binder for a rodent-derived IgG antibody of the present invention includes the nucleic acid molecule of the present invention. The detection reagent for detecting a rodent-derived IgG antibody of the invention includes the binder for a rodent-derived IgG antibody of the invention. The detection kit for detecting a rodent-derived IgG antibody of the invention includes the detection reagent for detecting a rodent-derived IgG antibody of the invention.

Abstract: Compositions and methods effective for modulating Hepatitis C viral infection are provided.

Abstract: The present invention provides a pharmaceutical composition for treating cancer, comprising at least one selected from deoxyribonucleic acids (DNA) for encoding small interfering RNA (siRNA) which complementarily binds to the base sequence of the transcript (mRNA transcript) of the FLJ25416 gene, represented by sequence number 3, sequence number 5, and sequence number 7 to inhibit the intracellular expression of the FLJ25416 gene, antisense RNA which inhibits expression of the FLJ25416 gene, and short hairpin RNA (shRNA) which inhibits expression of the FLJ25416 gene. As the siRNA, which is complementary to the base sequence of the transcript (mRNA transcript) of the FLJ25416 gene, the antisense RNA, and the shRNA, according to the present invention, inhibit expression of the FLJ25416 gene which is known to be expressed in cancer cells, and thus kill cancer cells, the composition of the present invention can be used as a novel anti-cancer agent.

Abstract: The instant application relates to methods and reagents for modulating the Hedgehog signaling pathway using RNA interference technology (RNAi). The application provides potential targets of the Hedgehog RNAi, methods to identify additional Hedgehog signaling pathway components, methods to inhibit Hedgehog signaling targets using siRNA, and their uses in the treatment of a number of disease conditions.

Abstract: The present invention relates to novel chimeric oligomeric compounds having a plurality of alternating regions having either RNA like having northern or 3?-endo conformational geometry (3?-endo regions) or DNA like having southern or C2?-endo/O4?-endo conformational geometry. The oligomeric compounds of the present invention have shown reduction in mRNA levels in multiple in vitro and in vivo assay systems and are useful, for example, for investigative and therapeutic purposes.

Abstract: Disclosed are methods for treating cancer by administering an effective amount of a modified Herpes simplex virus.

Abstract: The present invention provides a method of improving a therapeutic response to a cancer treatment, in a subject, the method comprising administering an effective amount of an agent that enhances the expression of microRNA-140-5p or an agent that mimics the effects of microRNA-140-5p. Further provided is a method of treating a cancer in a subject in need of such treatment comprising the step of administering an effective amount of a microRNA-140-5p or an agent that enhances the expression of microRNA-140-5p.

Abstract: The present invention relates to compositions and methods for inhibiting loss of a retinal ganglion cell in a subject, comprising non-invasively applying to the surface of the eye of the subject an ophthalmic composition comprising a therapeutically effective amount of at 5 least one siRNA which down regulates expression of a target gene associated with loss of the retinal ganglion cell, thereby inhibiting loss of the retinal ganglion cell in the subject. The methods of the invention also relate to the use of chemically modified siRNA compounds possessing structural motifs which down-regulate the expression of human genes expressed in retinal tissue in the mammalian eye.

Abstract: The present invention concerns a nucleotide aptamer having the sequence: 5?-AUGAUCAAUCGCCUCAAUUCGACAGGAGGCUCAC-3?(SEQ ID NO: 1) for use in the treatment and/or prevention and/or diagnosis of an Axl receptor tyrosine kinase induced disorder and a pharmaceutical composition comprising the same. The invention also relates to a method for the diagnosis of an Axl receptor tyrosine kinase induced disorder in a patient from which a sample is obtained and related diagnostic kit.

Abstract: The invention provides pharmaceutical compositions comprising short single stranded oligonucleotides, of length of between 8 and 26 nucleobases which are complementary to human microRNAs selected from the group consisting of miR19b, miR21, miR122a, miR155 and miR375. The short oligonucleotides are particularly effective at alleviating miRNA repression in vivo. It is found that the incorporation of high affinity nucleotide analogues into the oligonucleotides results in highly effective anti-microRNA molecules which appear to function via the formation of almost irreversible duplexes with the miRNA target, rather than RNA cleavage based mechanisms, such as mechanisms associated with RNaseH or RISC.