Abstract: The present invention provides an isolated nucleic acid molecule comprising, or consisting of, the nucleic acid sequence of SEQ ID NO:1 or a nucleic acid sequence of at least 400 bp having at least 80% identity to said sequence of SEQ ID NO:1, wherein said isolated nucleic acid molecule specifically leads to the expression in rod photoreceptors of a gene when operatively linked to a nucleic acid sequence coding for said gene.

Abstract: Compositions and methods are disclosed herein for cloning a synthetic or a semi-synthetic donor genome in a heterologous host cell. In one embodiment, the donor genome can be further modified within a host cell. Modified or unmodified genomes can be further isolated from the host cell and transferred to a recipient cell. Methods disclosed herein can be used to alter donor genomes from intractable donor cells in more tractable host cells.

Abstract: Nanoparticles comprising partially deacetylated chitosan and ss or dsRNA partially complementary to, binding to or at least 90% identical to mRNA targets of viruses pathogenic in farmed crustaceans, compositions and farmed aquatic crustaceans comprising the same, and methods for their use in treating or preventing viral infection in aquaculture are provided.

Abstract: Described herein are compositions and methods for modulation of gene expression in the liver including modulation of PCSK9, TTR, SERPINA1, KLKB1 and/or HAO1.

Abstract: The present invention relates to a mouse (Mus musculus) in which expression and site-specific modification of a target protein is temporally and spatially controlled, and a method for producing the same and the use thereof, and more particularly to a transgenic mouse in which expression of a target protein having a modification attached to a specific position is temporally and spatially controlled as a result of incorporation of an unnatural amino acid. In the mouse according to the present invention, in which site-specific modification of a target protein is temporally and spatially controllable, expression of the target protein having the site-specific modification attached thereto is controllable depending on the timing and/or position of introduction of an unnatural amino acid. Thus, the mouse according to the present invention is useful for studies on the in vivo functions of cellular proteins, various human diseases including cancers and neurodegenerative disorders, new drug discovery, and the like.

Abstract: Disclosed herein are recombinant viral vectors comprising a liver specific promotor in operable combination with a heterologous nucleic acid sequence encoding a protein, such as a clotting factor. Methods of treating a subject with a clotting disorder, such as hemophilia A or hemophilia B, are also provided.

Abstract: This invention relates to a method for separating blood cells comprising the steps of: (a) lysing red blood cells of a blood sample and diluting said sample; (b) providing a microfluidic device comprising a spiral-shaped flow channel having at least a first end and a second end, wherein said flow channel has two inlet ports at or near said first end and at least two outlet ports at or near said second end, wherein one of the two inlet ports is located at the inner wall of the spiral-shaped flow channel and the other inlet port is located at the outer wall of the spiral-shaped flow channel and at least one of the outlet ports is connected to a container allowing the storage of blood cells; (c) introducing the sample of step (a) into the inlet port located at the outer wall of the spiral-shaped flow channel and introducing a sheath fluid into the inlet port located at the inner wall of the spiral-shaped flow channel; (d) driving said sample and the sheath fluid through the spiral-shaped flow channel; and (e) re

Abstract: The disclosure in some aspects relates to recombinant adeno-associated viruses having distinct tissue targeting capabilities. In some aspects, the disclosure relates to gene transfer methods using the recombinant adeno-associate viruses. In some aspects, the disclosure relates to isolated AAV capsid proteins and isolated nucleic acids encoding the same.

Abstract: Compositions and methods for reducing susceptibility to infectious disease in bees using RNA interference technology, and more particularly, prevention and treatment of viral infections in honeybees such as Israel acute paralysis virus (IAPV) by feeding of pathogen-specific dsRNA. Further, multiple-pathogen specific dsRNA is disclosed.

Abstract: Provided is a selective method for inducing differentiation from pluripotent stem cells to enterocyte-like cells. Also provided is an excellent enterocyte-like cell expressing drug-metabolizing enzymes and drug transporters. More specifically, provided is an enterocyte-like cell having properties closer to those of primary enterocytes, which are difficult to acquire. The foregoing is achieved by adding an ALK5 inhibitor (SB431542), Wnt3a, and EGF to a culture system of definitive endoderm cells obtained by differentiation induction from pluripotent stem cells and extending a culture time. The foregoing is also achieved by introducing CDX2 gene and/or FOXA2 gene into the pluripotent stem cells or the definitive endoderm cells. The foregoing is also achieved by overlaying a basement membrane matrix on the enterocyte-like cells.

Abstract: The present disclosure provides recombinant adeno-associated virus (rAAV) virions comprising a variant AAV capsid protein, e.g., an AAV capsid protein derived from an ancestral AAV capsid protein amino acid sequence. An rAAV virion of the present disclosure can exhibit greater infectivity of a target cell. The present disclosure also provides methods of delivering a gene product to a target cell in an individual by administering to the individual an rAAV of the present disclosure. The present disclosure also provides methods of generating rAAV virions that have a variant AAV capsid protein derived from an ancestral AAV capsid protein amino acid sequence.

Abstract: Genetically modified rodents such as mice and rats, and methods and compositions for making and using the same, are provided. The rodents comprise a humanization of at least one endogenous rodent Tmprss gene, such as an endogenous rodent Tmprss2, Tmprss4, or Tmprss11d gene.

Abstract: Disclosed herein are altered adherent stromal cells and methods of producing and utilizing same.

Abstract: The present disclosure provides recombinant nucleic acids comprising one or more polynucleotides encoding a cystic fibrosis transmembrane conductance regulator (CFTR) polypeptide (e.g., a human CFTR polypeptide); viruses comprising the recombinant nucleic acids; compositions and formulations comprising the recombinant nucleic acids and/or viruses; methods of their use (e.g., for the treatment of a chronic lung disease, such as cystic fibrosis); and articles of manufacture or kits thereof.

Abstract: Disclosed herein is a polynucleotide construct comprising one or more nuclease recognition sequences upstream and downstream of a Gene editing multi-site that comprises a plurality of nuclease recognition sequences. The plurality of nuclease recognition sequences facilitate insertion of one or more exogenous donor genes into the host cell.

Abstract: Disclosed is a method for preparing a cell growth scaffold having a structural memory feature, comprising a step of preparing a micro-fibrous or flocculent acellular tissue matrix material; a step of preparing an acidification-treated hydrogel-like acellular tissue matrix particles; proportionally mixing the micro-fibrous or flocculent acellular tissue matrix material with the acidification-treated hydrogel-like acellular tissue matrix particles, followed by injection-molding, freezing treatment, radiation treatment, and ultimately preparing a porous cell growth scaffold that can be stored at room temperature. The prepared cell growth scaffold is a porous cell growth scaffold that has no chemical crosslinking, and has a biological activity, a stable three-dimensional structure and a structural memory feature.

Abstract: This invention pertains to the field of ovarian aging and premature ovarian failure. Specifically, we herein disclose methods for treating these conditions using endothelial progenitor cells alone or in combination with mesenchymal stem cells. Furthermore, these cell-based therapies can be utilized in combination with estrogen and progesterone receptor ligands to enhance the therapeutic activity of said cells for restoring or maintaining ovarian function and female fertility.

Abstract: Viral vectors comprising engineered hOTC DNA and RNA sequences are provided which when delivered to a subject in need thereof are useful for treating hyperammonemia, ornithine transcarbamylase transcarbamylasc deficiency and symptoms associated therewith. Also provided are methods of using hOTC for treatment of liver fibrosis and/or cirrhosis in OTCD patients by administering hOTC.

Abstract: The present invention provides a composition containing a single-stranded nucleic acid molecule consisting of a nucleotide sequence shown by 5?-AGCAGAGUACACACAGCAUAUACC-P-GGUAUAUGCUGUGUGUACUCUGCUUC-P-G-3? (SEQ ID NO: 1) (in the sequence, P is a proline derivative linker represented by (I) in the DESCRIPTION) and a buffer, and having the following features: (a) being in the form of a solution at ambient temperature; and (b) a content of the nucleic acid molecule after storage at 25° C., relative humidity 60% for 4 weeks, of not less than 80% relative to the content at the time of start of the storage.

Abstract: Provided herein are genetic constructs comprising genetic perturbation cassettes and methods of using such to assess the timing and order of gene expression.