The invention provides cells and animals, as well as methods of producing cells and animals, that express at least one interfering RNA molecule to regulate the expression of a specific gene or family of genes. The invention further provides novel iRNA molecules, as well as DNA templates for producing iRNA molecules.

The present invention relates to agents that modulate/regulate the activity of the protein TMEM230 for use in the therapeutic treatment of pathologies in which therapeutic regulation of angiogenesis is advisable or necessary.

The present invention pertains to a non-human genetically modified animal with increased susceptibility to infection with a human virus. The invention suggests to genetically impair the expression of newly identified viral infection repression factors CD302, Cr11, Ndufc2, AW112010, Scarb2 and Zc3hav1, which markedly improves infection with human viruses in none-human hosts. Furthermore provided are methods for the generation of the animal of the invention, methods for increasing or reducing the susceptibility of a cell to viral infection, methods for screening novel modulators of viral infection as well as new therapy options for the treatment of viral diseases, in particular hepatitis C.

The subject invention provides materials and method for making a recessive gene dominant. This is accomplished by interfering with the natural mechanisms that inhibit expression of the recessive gene and/or by interfering with the expression of the naturally dominant gene. In a preferred embodiment, the method of the subject invention comprises both reducing inhibition of expression of the recessive gene and increasing inhibition of the dominant gene.

The present invention relates to animal models that expresses SR-BICT knockin. The present invention further includes animal models that express SR-BICT and also have reduced expression or activity of ApoE and/or LDLR, wherein the latter can be accomplished by use of a compound or genetic manipulation of the gene. The present invention relates to mouse models crossed with SR-BICT knockin mice. Specifically, the present invention relates to SR-BICT knockin mice crossed with apolipoprotein E (ApoE) knockout mice (SR-BICT/apoE KO), a hypoE mouse (also referred to as ApoeR61.sup.h/h which expresses an impaired ApoE protein (SR-BICT/ApoeR61.sup.h/h)), or a LDLR knockout mouse (SR-BICT/LDLR KO). Screening methods and compounds using these mouse models are also encompassed.

The invention in some aspects relates to isolated nucleic acids, compositions, and kits useful for identifying adeno-associated viruses in cells. In some aspects, the invention provides kits and methods for producing somatic transgenic animal models using recombinant AAV (rAAV) to an animal having at least one transgene that expresses a small interfering nucleic acid or at least one binding site for a miRNA.

The present invention relates to processes for transfecting cells. In particular, the present invention relates to processes for using CRISPR to incorporate a polynucleotide into the genome of an avian primordial germ cell (PGC).

Non-human animal genomes, non-human animal cells, and non-human animals comprising a humanized TTR locus and methods of using such non-human animal genomes, non-human animal cells, and non-human animals are provided. Non-human animal cells or non-human animals comprising a humanized TTR locus express a human transthyretin protein or a chimeric transthyretin protein, fragments of which are from human transthyretin. Methods are provided for using such non-human animals comprising a humanized TTR locus to assess in vivo efficacy of human-TTR-targeting reagents such as nuclease agents designed to target human TTR. Methods are also provided for making such non-human animals comprising a humanized TTR locus.

The invention in some aspects relates to isolated nucleic acids, compositions, and kits useful for identifying adeno-associated viruses in cells. In some aspects, the invention provides kits and methods for producing somatic transgenic animal models using recombinant AAV (rAAV) to an animal having at least one transgene that expresses a small interfering nucleic acid or at least one binding site for a miRNA.

Non-human animal genomes, non-human animal cells, and non-human animals comprising a humanized TTR locus and methods of using such non-human animal genomes, non-human animal cells, and non-human animals are provided. Non-human animal cells or non-human animals comprising a humanized TTR locus express a human transthyretin protein or a chimeric transthyretin protein, fragments of which are from human transthyretin. Methods are provided for using such non-human animals comprising a humanized TTR locus to assess in vivo efficacy of human-TTR-targeting reagents such as nuclease agents designed to target human TTR. Methods are also provided for making such non-human animals comprising a humanized TTR locus.