Abstract: The present disclosure relates generally to cleavable linkers and uses thereof.

Abstract: The present invention provides a method for efficiently obtaining a complex in which a gene (mRNA) and a peptide as a translation product thereof are linked via a peptide receptor molecule, and utilization thereof. In the method of the present invention, at least one 2?-modified nucleoside derivative is introduced into a 5? end side of an antisense strand of a template DNA.

Abstract: The invention provides improved gene therapy vectors, compositions, and methods.

Abstract: The present invention provides a novel class of insecticides for control of disease vector insects, particularly mosquitoes. These insecticides prevent maturation or development of larvae into adult insects using interfering RNA (iRNA). The present invention further includes compositions comprising iRNA and methods of controlling, reducing, or treating an insect infestation with the iRNA or compositions described herein.

Abstract: Disclosed are a pharmaceutical composition for anionic drug delivery, and a preparation method therefor, the pharmaceutical composition for anionic drug delivery containing: an anionic drug as an active ingredient; a cationic compound; an amphiphilic block copolymer; and a polylactate, wherein the anionic drug formed a complex with the cationic lipid, and the complex is encapsulated within a micelle structure formed by the amphiphilic block copolymer and the polylactate.

Abstract: Methods of prohibiting Xist-dependent silencing of X chromosome genes include targeting the required Xist silencing complex components including SHARP, SMRT, HDAC3, SAF-A, LBR, and the respective binding sites of SHARP, LBR, and SAF-A on Xist, thereby prohibiting Xist repression, allowing for reactivation of the silenced X chromosome genes.

Abstract: The present invention relates to recombinant adeno-associated virus (rAAV) delivery of polynucleotides for treating Duchenne Muscular Dystrophy resulting from the duplication of DMD exon 2. The invention provides rAAV products and methods of using the rAAV in the treatment of Duchenne Muscular Dystrophy.

Abstract: The present embodiments provide methods, compounds, and compositions useful for inhibiting YAP1 expression, which may be useful for treating, preventing, or ameliorating a cancer associated with YAP1.

Abstract: An objective of the present invention is to provide a chemically-modified siRNA that has polydeoxyadenylic acid added to the 5? end of the sense strand, and, when complexed with schizophyllan, is high in resistance against RNase and moreover effectively exhibits RNAi activity. To achieve the objective, in the chemically-modified siRNA with polydeoxyadenylic acid added to the 5? end of the sense strand, specific chemical modification is performed for dinucleotide sequences of CA, UA, and UG in the base sequence of the sense strand and dinucleotide sequences of CA, UA, and UG in the base sequence at and after the eighth base from the 5? end of the antisense strand.

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 including those directed to nucleotide sequences for TTR.

Abstract: Disclosed herein are UBAP2L targeting agents and use of such agents for the treatment of a neurodegenerative disorder. Further disclosed herein are methods of monitoring the progression of a neurodegenerative disorder and methods of determining the efficacy of a neurodegenerative disorder therapy.

Abstract: The present invention generally relates to bacterial polypeptide display systems, libraries using these bacterial display systems, and methods of making and using these systems, including methods for improved display of polypeptides on the extracellular surface of bacteria using circularly permuted transmembrane bacterial polypeptides that have been modified to increase resistance to protease degradation and to enhance polypeptide display characteristics.

Abstract: Codon optimized nucleic acid sequences for RDH12 are provided, as well as recombinant viral vectors, such as AAV, expression cassettes, proviral plasmids or other plasmids containing the codon optimized sequence for functional RDH12. Recombinant vectors are provided that express the codon optimized, functional RDH12. Compositions containing these codon optimized sequences are useful in methods for treating, retarding or halting certain blinding diseases resulting from the absence, deficiency or inappropriate expression of RDH12. Other compositions and methods are providing for correcting a non-functional, defective or inadequately expressed native RDH12.

Abstract: Disclosed herein are compositions and methods for reducing expression of C9ORF72 antisense transcript in an animal with C9ORF72 antisense transcript specific inhibitors. Such methods are useful to treat, prevent, or ameliorate neurodegenerative diseases in an individual in need thereof. Such C9ORF72 antisense transcript specific inhibitors include antisense compounds.

Abstract: Provided herein, in some embodiments, are artificial ribosomes that synthesize nonribosomal peptides, polyketides, and fatty acids with full control over peptide sequence. Also provided herein are methods for programmed synthesis of nonribosomal peptides, polyketides, and fatty acids. In particular, provided herein are methods for scalable synthesis of a wide range of antibacterial, antifungal, antiviral, and anticancer compounds.

Abstract: An object of the present invention is to provide ASO that can effectively suppress a formation of RNA foci spontaneously occurring in neurons having SCA36 mutation, without inducing the cleavage of NOP56 pre-mRNA. The present invention provides an oligonucleotide comprising a nucleotide sequence in which one or more nucleotide sequences represented by SEQ ID NO: 2 are consecutively connected and is complementary to pre-mRNA of NOP56 gene, wherein a structure formed by the hybridization of the oligonucleotide to the pre-mRNA is resistant to RNaseH.

Abstract: A method for displaying proteins and peptides is disclosed in which individual proteins or peptides remain associated with the DNA encoding them. Proteins or peptides can be generated by in vitro translation of DNA templates, either free in solution or arrayed on a solid support, such that the proteins or peptides remain immobilized on their DNA templates. In particular, high throughput sequencing can be combined with high throughput functional characterization of encoded proteins and peptides, wherein the identity of each protein or peptide is determined by DNA sequencing, and functional studies are carried out directly on each protein or peptide while immobilized on the DNA template encoding it. The methods of the invention should find numerous applications, for example, in high throughput genetic or pharmacological screening, epitope mapping, and protein engineering and directed evolution.

Abstract: Disclosed herein are compositions and methods for reducing expression of C9ORF72 antisense transcript in an animal with C9ORF72 antisense transcript specific inhibitors. Such methods are useful to treat, prevent, or ameliorate neurodegenerative diseases in an individual in need thereof. Such C9ORF72 antisense transcript specific inhibitors include antisense compounds.

Abstract: The present invention provides methods and compositions for stable genetic modification of cultured mammalian cells. The genetic modifications can be used to produce cultured mammalian cells for therapeutic or diagnostic purposes.

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.