Abstract: The present disclosure provides AAV capsid proteins comprising a modification in the amino acid sequence and virus vectors comprising the modified AAV capsid protein. The disclosure also provides methods of administering the virus vectors and virus capsids of the disclosure to a cell or to a subject in vivo.

Abstract: The invention provides a nucleic acid construct comprising a sequence that encodes a human telomerase reverse transcriptase (hTERT) protein which is devoid of telomerase catalytic activity and of a nucleolar localization signal. The construct is useful triggering an immune response in a subject, against cells that overexpress telomerase, preferably dysplasia cells or tumor cells.

Abstract: The present disclosure provides AAV capsid proteins comprising a modification in the amino acid sequence and virus vectors comprising the modified AAV capsid protein. The disclosure also provides methods of administering the virus vectors and virus capsids of the disclosure to a cell or to a subject in vivo.

Abstract: Methods and compositions for the treatment of a corneal dystrophy in a subject in need thereof are provided. In one aspect, the method includes the step of obtaining a plurality of stem cells comprising a nucleic acid mutation in a corneal dystrophy target nucleic acid from the subject and manipulating the nucleic acid mutation in one or more stem cells of the plurality of stem cells to correct the nucleic acid mutation, thereby forming one or more manipulated stem cells. The manipulated stem cells are isolated and then transplanted into the subject. In some embodiments, the nucleic acid mutation is manipulated using CRISPR system.

Abstract: Provided are linkers suitable for preparing a conjugate of a nucleic acid and a peptide as a translation product thereof in a reconstituted cell-free translation system in genotype-phenotype mapping (display methods), said linkers comprising a single-stranded structure region having a side chain base pairing with the base at the 3?-end of an mRNA at one end and a peptidyl acceptor region containing an amino acid attached to an oligo RNA consisting of a nucleotide sequence of ACCA via an ester bond at the other end, characterized in that the ester bond is formed by using an artificial RNA catalyst. Also provided are display methods using [mRNA]-[linker]-[peptide] conjugates assembled via such linkers.

Abstract: Disclosed is a composition for diagnosing, preventing or treating drug addiction based on synaptotagmin binding cytoplasmic RNA interacting protein (SYNCRIP). SYNCRIP or a gene encoding the protein can be used to diagnose drug addiction. In addition, a therapeutic agent for drug addiction can be discovered by measuring the expression of SYNCRIP. Furthermore, an agent expressing SYNCRIP or promoting the activity of SYNCRIP can be used to prevent or treat drug addiction.

Abstract: Methods of characterizing an analyte using a detector such as a nanopore and an enzyme are provided. One aspect features methods for characterizing a double-stranded polynucleotide using a detector, e.g., without using a hairpin connecting a template and a complement of the double-stranded polynucleotide. Another aspect features methods for characterizing an analyte using a tag-modified nanopore with increased sensitivity and/or higher throughput. Compositions and systems including, e.g., adaptors for attachment to double-stranded polynucleotides and tag-modified nanopores, which can be used in the methods are also provided.

Abstract: The present invention relates to polypeptide expression systems and methods of using the same.

Abstract: A method of site-specific modification of an endogenous target DNA of a eukaryotic cell is provided. The method includes contacting the endogenous target DNA having an intended modification site with (i) a gene editing system configured to introduce a double strand break in the endogenous target DNA at or near the intended modification site, and (ii) a donor DNA repair template comprising a plurality of tandem repeat sequences. In the method, each of the plurality of tandem repeat sequences comprises an exogenous donor DNA sequence flanked by a donor 5? flanking sequence and a donor 3? flanking sequence. The donor 5? flanking sequence and the donor 3? flanking sequence are homologous to a continuous DNA sequence on either side of the intended modification site in the endogenous target DNA.

Abstract: The present disclosure provides methods for detecting a single-stranded target RNA. The present disclosure provides methods of cleaving a precursor C2c2 guide RNA array into two or more C2c2 guide RNAs. The present disclosure provides a kit for detecting a target RNA in a sample.

Abstract: The disclosure is directed to an expression vector or a combination of at least two expression vectors for producing a polypeptide of interest, the vector or vectors comprising a polynucleotide encoding a mutated folate receptor as a selectable marker. The disclosure also relates to host cells, selection methods and methods for producing polypeptides with high yield.

Abstract: The present invention provides mammalian cell expression vectors that impart to mammalian host cells an ability to produce high levels of foreign gene-derived proteins. A ubiquitously acting chromatin opening element (UCOE) is introduced into an expression vector that has a plasmid DNA integrated into a transcriptional hot spot on the chromosome of a dihydrofolate reductase gene-deficient host cell so that it allows for selection of strains that grow in hypoxanthine-thymidine (hereinafter denoted as HT)-free medium, whereby transformants will produce a protein of interest in increased amounts.