A method of preparing a chimeric virus comprising bocavirus capsid protein (VP) and a recombinant adeno-associated (AAV) viral genome, and isolated mutant bocavirus genomes, are provided.

Provided herein, in some aspects, are compositions of nucleic acids comprising an initial transcribed sequence and unique nucleic acid designs for high-yield and cost-effective production of ribonucleic acids.

The present invention describes a recombinant herpesvirus of turkeys (rHVT) that can be used as a vector vaccine for poultry against infection and disease from multiple poultry pathogens. Specifically the rHVT expresses an infectious bursal disease virus (IBDV) viral protein 2 (VP2) gene and a Newcastle disease virus (NDV) fusion (F) protein gene from a first and a second expression cassette inserted in the unique small (Us) region, and expresses an avian influenza vims (AIV) haemagglutinin (HA) gene from a third expression cassette inserted in the unique long (UL) region of the genome of said rHVT either between the UL40 and UL41 genes, or between the UL44 and UL45 genes. This rHVT can be used to vaccinate poultry against MDV, IBDV, NDV and AIV.

Provided are a human genome-derived polynucleotide having a chromatic regulator factor function, a recombinant vector and a recombinant cell, each comprising same, and a method for producing a polypeptide of interest using same.

The present invention relates to nucleic acid expression cassettes and vectors containing liver-specific regulatory elements and codon-optimized factor IX or factor VIII transgenes, methods employing these expression cassettes and vectors and uses thereof. The present invention is particularly useful for applications using liver-directed gene therapy, in particular for the treatment of hemophilia A and B.

A nucleic acid trans-splicing molecule is provided that can replace an exon in a targeted mammalian ocular gene carrying a defect or mutation causing an ocular disease with an exon having the naturally-occurring sequence without the defect or mutation. The trans-splicing molecule includes a 3′ transcription terminator domain which enhances the efficiency of trans-splicing. The 3′ TTD comprises a triple helix domain and a tRNA-like domain.

Provided herein are methods of delivering “split” Cas9 protein or nucleobase editors into a cell, e.g., via a recombinant adeno-associated virus (rAAV), to form a complete and functional Cas9 protein or nucleobase editor. The Cas9 protein or the nucleobase editor is split into two sections, each fused with one part of an intein system (e.g., intein-N and intein-C encoded by dnaEn and dnaEc, respectively). Upon co-expression, the two sections of the Cas9 protein or nucleobase editor are ligated together via intein-mediated protein splicing. Recombinant AAV vectors and particles for the delivery of the split Cas9 protein or nucleobase editor, and methods of using such AAV vectors and particles are also provided.

The present disclosure relates to genetically modified non-human animals that express a human or chimeric (e.g., humanized) IL6R and/or IL6, and methods of use thereof.

Provided herein are methods of delivering “split” Cas9 protein or nucleobase editors into a cell, e.g., via a recombinant adeno-associated virus (rAAV), to form a complete and functional Cas9 protein or nucleobase editor. The Cas9 protein or the nucleobase editor is split into two sections, each fused with one part of an intein system (e.g., intein-N and intein-C encoded by dnaEn and dnaEc, respectively). Upon co-expression, the two sections of the Cas9 protein or nucleobase editor are ligated together via intein-mediated protein splicing. Recombinant AAV vectors and particles for the delivery of the split Cas9 protein or nucleobase editor, and methods of using such AAV vectors and particles are also provided.

Method of generating a barcoded library, comprising delivering a polynucleotide into a cell, each polynucleotide comprising: (i) a sequence encoding a barcoding construct operably linked to a first promoter that is an antisense promoter, wherein the barcoding construct comprises a trans-splicing element and a barcode sequence; and a sequence encoding a perturbation element operably linked to a second promoter; generating RNA transcripts of the polynucleotide delivered into the cell, wherein the RNA transcripts comprise the barcoding construct and the perturbation element; and splicing the barcoding sequence onto endogenous RNA molecules in the cell, thereby generating a barcoded library, each member of the barcoded library comprising the barcode sequence and the endogenous RNA molecule attached with the barcode sequence.