Abstract: A process and molecular biomarker for platelets stored in blood banks provides for efficient verification of which platelet concentrate bags are in good condition for transfusion by measuring, by real-time polymerase chain reaction, expression levels of hsa-miR-127 and hsa-miR-320a microRNAs as the first process and biomarker predictor of platelet's cell aging during storage and the consequent presence of storage lesions as a quality test of these bags.

Abstract: The present invention relates to a therapeutic compound comprising: an agent that inhibits the activity of at least one gene selected from the group consisting of MAF, MEOX2, SIX2 and homologs thereof having at least 50% identity with said genes and/or an agent that enhances the activity of at least one gene selected from the group consisting of CREB5, E2F1, EGR2, HIC1, IRF7, JUN, MYC, SRF, STAT4, TCF4, FOXS1, GLI1, SOX9 and homologs thereof having at least 50% identity with said gene for use in the treatment of wounds, preferably chronic wounds.

Abstract: This invention is for a new molecular recognition system, where DNA-like molecules comprising segments built from nucleotides that carry only a small which comprises process by which one molecule interacts with a specific second molecule, or by which a portion of a single molecule interacts specifically with another portion of the same molecule. Further, this invention relates to molecular recognition that follows simple rules. Further, this invention relates to molecular systems that are linear biopolymers that are analogs of DNA and RNA, in that they are built from a small set of building blocks that are linked together by phosphate groups, where the building blocks comprise a sugar (ribose, 2?-deoxyribose, or an analog) attached to a heterocycle. The molecular recognition that they display differs from that displayed by DNA and RNA, in that the rules governing molecular recognition break the rules of size complementarity followed in molecular recognition displayed between and within strands of DNA and RNA.

Abstract: This invention provides compounds, compositions and methods for modulating the expression of human GST-? using RNA interference. The RNA interference molecules can be used in methods for preventing or treating diseases such as malignant tumor. A nucleic acid molecule can have a) a polynucleotide sense strand and a polynucleotide antisense strand; b) each strand of the molecule being from 15 to 30 nucleotides in length; c) a contiguous region of from 15 to 30 nucleotides of the antisense strand being complementary to a sequence of an mRNA encoding GST-?; and d) at least a portion of the sense strand can be complementary to at least a portion of the antisense strand, and the molecule has a duplex region of from 15 to 30 nucleotides in length.

Abstract: This invention provides compounds, compositions and methods for modulating the expression of human GST-? using RNA interference. The RNA interference molecules can be used in methods for preventing or treating diseases such as malignant tumor. Provided are a range of siRNA structures, having one or more of nucleotides being modified or chemically-modified. Advantageous structures include siRNAs with 2?-deoxy nucleotides located in the seed region, as well as other nucleotide modifications.

Abstract: The present invention encompasses a class of compounds known as splice modulating oligonucleotides (SMOs) that modulate pre-mRNA splicing, thereby affecting expression and functionality of a specific protein in a cell. The present invention further provides compositions and methods for modulating pre-mRNA splicing using a SMO of the invention to abrogate disease-causing mutations in a protein. Accordingly, the present invention provides compositions and methods of treating a subject at risk of, susceptible to, or having a disease, disorder, or condition associated with aberrant or unwanted target pre-mRNA expression or activity.

Abstract: Provided herein are oligonucleotides complementary to euchromatic regions of target genes that are useful for increasing expression of the target genes; related compositions and methods are also provided.

Abstract: The instant invention provides compositions and methods for inhibiting the JC Virus (JCV), and that can be used, for example, for treating progressive multifocal leukoencephalopathy (PML). Antisense oligonucleotides are provided which are effective in inhibiting JCV replication or multiplication, alone or in a combination. In preferred embodiments, the oligonucleotides contain modifications.

Abstract: The present invention is directed to reversibly inactivated membrane active polymers useful for cellular delivery of compounds. Described are polyconjugate systems that incorporate targeting, anti-opsonization, anti-aggregation, and transfection activities into small biocompatible in vivo delivery conjugates. The use of multiple reversible linkages connecting component parts provides for physiologically responsive activity modulation.

Abstract: Disclosed herein is a method for inhibiting expression of a gene of a subject comprising administering (1) a composition comprising R-(L)a-(G)b; wherein R is an oligonucleotide selected from the group consisting of DNA, RNA, siRNA, and microRNA; L is a linker and each occurrence of L is independently selected from Table 3; G is a targeting ligand and each occurrence of G is independently selected from Table 4; each of a and b is independently 0, 1, 2, 3 or 4; and (2) a composition comprising (P)c-(L)d-(G)e; wherein P is a peptide and each occurrence of P is independently selected from Table 2; L is a linker and each occurrence of L is independently selected from Table 3; G is a targeting ligand and each occurrence of G is independently selected from Table 4; d is 0, 1, 2, 3, 4, 5 or 6; and each of c and e is independently 1, 2, 3, 4, 5 or 6. Compositions in (1) and (2) can be co-administered or sequentially administered.

Abstract: The present embodiments provide methods, compounds, and compositions for treating, preventing, ameliorating a disease associated with excess growth hormone using antisense compounds oligonucleotides targeted to growth hormone receptor (GHR).

Abstract: The present invention relates to methods of treating a condition associated with mTORC1 hyperactivation or TSC2-deficient cancer, the method comprising administering to a subject having the cancer a pharmaceutically-effective amount of a poly(ADP-ribose) polymerase 1 (PARP1) inhibitor. In some embodiments, the condition associated with mTORC1 hyperactivation is tuberous sclerosis complex (TSC). In some embodiments, the condition associated with mTORC1 hyperactivation is lymphangioleiomyomatosis (LAM). In some embodiments, the condition associated with mTORC1 hyperactivation is TSC2-deficient cancer.

Abstract: Provided is a drug that allows highly-efficient skipping of exon 51 in the human dystrophin gene. The present invention provides an antisense oligomer which enables exon 51 in the human dystrophin gene to be skipped.

Abstract: The present invention is related to a nucleic acid molecule binding to SDF-1, whereby the nucleic acid molecule influences migration of cells.

Abstract: This invention relates, e.g., to a method for enhancing exon skipping in a pre-mRNA of interest, comprising contacting the pre-mRNA with an effective amount of a compound such as, for example, Perphenazine, Flupentixol DiHCl, Zuclopenthixol or Corynanthine HCl, or a compound which shares a similar 2-D structure and activity level with one of these compounds, or a pharmaceutically acceptable salt, hydrate, solvate, or isomer of the compound, and, optionally, with an antisense oligonucleotide that is specific for a splicing sequence in the pre-mRNA Methods for treating Duchenne muscular dystrophy (DMD) are disclosed.

Abstract: An object of the present invention is to provide lipid particles which have low cytotoxicity, can stably hold nucleic acid molecules outside cells, and can promptly release nucleic acids in cytoplasm after escaping from endosome, and a nucleic acid delivery carrier. According to the present invention, there are provided lipid particles containing a compound represented by the following General Formula (1), sterol, at least one lipid selected from the group consisting of a neutral lipid and a lipid having a polyethylene glycol chain, and nucleic acids, and a nucleic acid delivery carrier. In the formula, R1 and R2 are the same as or different from each other, and are alkyl groups having 10 to 22 carbon atoms.

Abstract: The present invention provides synthetic RNA aptamers that bind RDX. In various embodiments, the synthetic RNA aptamers may include one or more aptamers selected from the group consisting of SEQ ID 1-12. The synthetic RNA aptamers that bind RDX provide an inexpensive, in situ method for testing for RDX, which may be used for both soil and water samples.

Abstract: Compositions for modulating the expression of a protein in a target cell comprising at least one RNA molecule which comprises at least one modification 5 conferring stability to the RNA, as well as related methods, are disclosed.

Abstract: The present invention relates to antisense oligonucleotides that modulate the expression of and/or function of a Lipid transport and metabolism gene, in particular, by targeting natural antisense polynucleotide of a Lipid transport and metabolism gene. The invention also relates to the identification of these antisense oligonucleotides and their use in treating diseases and disorders associated with the expression of a Lipid transport and metabolism genes.

Abstract: The invention relates to siRNA molecules and their use in methods and pharmaceutical compositions for inhibiting the expression of the PDK1 gene. The invention also relates to the use of said siRNAs molecules in the treatment and/or prevention of an eye condition characterized by increased expression and/or activity of PDK1 gene, preferably said eye condition is conjunctivitis and/or an ocular allergy such as seasonal allergic conjunctivitis, perennial allergic conjunctivitis, vernal keratoconjunctivitis, atopic keratoconjunctivitis, and giant papillary conjunctivitis.