Abstract: Congenital abnormalities of the kidney or the urinary tract (CAKUT) are the most common cause of pediatric kidney failure. These disorders are highly heterogenous, and their etiology is poorly understood. Dual serine/threonine and tyrosine protein kinase (DSTYK) mutations were detected in 2.2% of patients with congenital abnormalities of the kidney and urinary tract, suggesting that DSTYK is a major determinant of human urinary development, downstream of fibroblast growth factor (FGF) signaling. Methods and kits are provided for identifying and treating subjects at greater risk of developing CAKUT based on the presence of DSTYK mutations. Techniques include obtaining a biological sample from a subject and determining if the biological sample indicates a mutation of a gene for DSTYK. If it is determined that the biological sample indicates the mutation of the gene for DSTYK, then it is determined that the subject has or is at risk of developing CAKUT.

Abstract: The invention in some aspects relates to methods and compositions for assessing the effectiveness of miRNA inhibitors. In other aspects of the invention, methods and compositions for treating cholesterol related disorders are provided. In one aspect of the invention, miRNA inhibitors against miR-122 and rAAV-based compositions comprising the same are provided.

Abstract: What is described is a pharmaceutical composition for treating a fibrotic disease comprising a drug carrier, which comprises a lipid and a retinoid, and a double-stranded nucleic acid molecule, which comprises an antisense sequence to mRNA encoding human hsp47.

Abstract: What is described is a method for treating a fibrotic disease by administering a pharmaceutical composition comprising a drug carrier, which comprises a lipid and a retinoid, and a double-stranded nucleic acid molecule, which comprises an antisense sequence to mRNA encoding human hsp47.

Abstract: The invention relates to novel aptamers, in particular aptamers which are capable of binding to EGFR. The invention also relates to cancer cell binding complexes comprising said aptamers and the use of said cancer cell binding complexes in the treatment of cancer.

Abstract: The present invention relates to ocular administration of sd-rxRNA and rxRNAori molecules.

Abstract: The application discloses methods and compositions for the inhibition of the alternative complement pathway. The methods and compositions involve the use of aptamers for inhibiting complement Factor D. The application further provides anti-Factor D aptamers for the treatment of dry age-related macular degeneration, geographic atrophy, wet age-related macular degeneration or Stargardt disease.

Abstract: A kit for altering allogeneic human cells for a human recipient where the kit includes a set of lentivirus vectors where each of the lentivirus vectors expresses a sequence targeting a consensus conserved nucleic acid sequence, which when expressed in cells, functions as a negative modulator for nucleic acid encoding a domain having a mismatch in an HLA protein and where the set of lentivirus vectors includes individual lentivirus vectors that correspond to individual HLA mismatches for a set of HLA mismatches that consist of HLA Class I mismatches and at least one HLA Class II mismatch and where the kit is for treatment of human cells by an appropriate subset of the set of lentivirus vectors based at least in part on a determined subset of the set of HLA mismatches between a human donor and a human recipient or between human cells and a human recipient.

Abstract: The invention relates to si RNA molecules and their use in methods and pharmaceutical compositions for inhibiting the expression of the ORAI1 gene. The invention also relates to the use of said si RNAs molecules in the treatment and/or prevention of an eye condition characterized by increased expression and/or activity of ORAI1 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.

Abstract: Provided are a method and use of microRNA MiR-2911 in regulating an ebola virus. Particularly provided are a method and use of isolated microRNA MiR-2911 in regulating an ebola virus protein gene.

Abstract: Summary Problem The purpose of the present invention is to provide: a chiral nucleic acid adjuvant having anti-tumor activity; and an anti-tumor agent. Solution The present invention relates to an adjuvant for anti-tumor agent, wherein the adjuvant comprises oligonucleotides which comprise two to four CpG motif each represented by 5?-X1CpGX2-3? and has a length of 14 to 32 nucleotides, wherein a nucleic acid at 3? end side of at least two CpG motifs is connected by phosphorothioate linkage, wherein each nucleic acid at 3? end and 5? end of the oligonucleotide is S type nucleic acid connected by phosphorothioate linkage, and wherein the oligonucleotide comprises at least one nucleic acid without phosphorothioate modification. The present invention relates to an anti-tumor agent containing its adjuvant.

Abstract: Problem The purpose of the present invention is to provide: a chiral nucleic acid adjuvant having immunity-inducing activity and an immunity-inducing activator. Solution The present invention relates to an adjuvant which comprises oligonucleotides which comprise two to four sequences each represented by 5?-X1CpG X2-3? and has a length of 14 to 32 nucleotides, wherein a nucleic acid at 3? end side of at least two CpG motifs is connected by phosphorothioate linkage, wherein each nucleic acids at 3? end and 5? end of the oligonucleotide is S type nucleic acids connected by phosphorothioate linkage, and wherein the oligonucleotide comprises at least one nucleic acid without phosphorothioate modification. The present invention relates to an immunity-inducible activator comprising the adjuvant.

Abstract: The present invention provides nucleic acid inhibitors of MYH7B and compositions thereof. The present invention also provides methods of treating or preventing a cardiac disorder such as cardiac hypertrophy, myocardial infarction, or heart failure in a subject by administering to the subject an inhibitor of MYH7B. The present invention further provides methods of modulating the activity or expression of ?-MHC in cardiac cells of a subject by administering to the subject an inhibitor of MYH7B.

Abstract: An siRNA for specifically targeting a PMP22 mutant gene and a pharmaceutical composition for preventing or treating Charcot Marie Tooth disease, which includes the same, are provided. According to the present invention, it is confirmed that selective suppression of a PMP22 mutant allele by a non-viral delivery system of siRNA may improve demyelinating neuropathic symptoms of CMT in vivo, enhance a motor ability and increase a volume of muscle. Therefore, the siRNA may be used in a useful method for treating various dominantly inherited peripheral neuropathies including CMT.

Abstract: Disclosed herein are methods of treating and diagnosing muscular dystrophy. In some examples, the methods include treating muscular dystrophy by administering to the subject a therapeutically effective amount of an agent that alters the expression of at least one miR gene product, such as miRNA-124 and/or miRNA-29 thereby treating muscular dystrophy. In one particular example, the method of treatment includes administering an agent that decreases the expression or activity of miRNA-124. In another embodiment, the method of treatment includes administering a composition that includes one or more agents to decrease the expression and/or activity of miRNA-124 and one or more agents to alter the activity of miRNA-29 (increase or decrease). Also disclosed are methods of enhancing muscle regeneration, repair, or maintenance in a subject and methods of enhancing ?7?1 integrin expression. Methods of prospectively preventing or reducing muscle injury or damage in a subject are also disclosed.

Abstract: An active agent capable of reducing quinone reductase 2 activity, for use in improvement of cognition in a subject is provided. Such an active agent may be a nucleic acid molecule that reduces the gene expression level of quinone reductase 2 or an inhibitor of quinone reductase 2 activity. A vector comprising a nucleic acid molecule that reduces the gene expression level of quinone reductase 2 and a pharmaceutical composition comprising an active agent capable of reducing quinone reductase 2 activity or said vector are provided as well.

Abstract: Provided is an adjuvant composition comprising a nucleic acid comprising a double-stranded RNA bound to a single-stranded DNA, the double-stranded RNA consisting of a nucleotide sequence of SEQ ID NO: 1 and its complementary sequence, the single-stranded DNA consisting of a nucleotide sequence of SEQ ID NO: 2. Also provided is a vaccine composition comprising the adjuvant composition and an antigen or an antigen component. The nucleic acid contained in the adjuvant composition is a chemically synthesizable nucleic acid having potent adjuvant activity and high safety.

Abstract: Antisense sequences, including duplex RNAi compositions, which possess improved properties over those taught in the prior art are disclosed. The invention provides optimized antisense oligomer compositions and method for making and using the both in in vitro systems and therapeutically. The invention also provides methods of making and using the improved antisense oligomer compositions.

Abstract: Certain embodiments are directed to methods of identifying neuroblastoma differentiation-inducing compounds or agent and their use in treating neuroblastoma.

Abstract: Disclosed herein are methods for treating/and or preventing diabetes using a specific inhibitor of SMAD7 expression or function. Also disclosed are methods of promoting organ and/or cell, e.g., pancreatic islet cell, survival after transplantation using a specific inhibitor of SMAD7 expression or function.