A method for treating an HIV disease in a subject in need of said treatment, comprising administering to the subject a therapeutically effective amount of a DNA vaccine comprising an expression vector and a pharmaceutically acceptable excipient, where the expression vector comprises: (a) a heterologous promoter operatively linked to a DNA sequence encoding a nuclear-anchoring protein, where the nuclear-anchoring protein comprises: (i) a DNA binding domain which binds to a specific DNA binding sequence, and (ii) a functional domain of the Bovine Papilloma Virus Type 1 E2 protein, where the functional domain binds to a nuclear component; (b) a multimerized DNA sequence that forms a binding site for the nuclear anchoring protein; and (c) at least one expression cassette comprising a DNA sequence encoding a protein or peptide that stimulates an immune response specific to the protein or peptide; where the expression vector lacks an origin of replication functional in mammalian cells.

Described herein are constructs used for liver-specific expression of a transgene.

The present disclosure provides polynucleotide cassettes, expression vectors and methods for the expression of a gene in cone cells.

Four zebrafish gene promoters, which are skin specific, muscle specific, skeletal muscle specific and ubiquitously expressed respectively, were isolated and ligated to the 5′ end of the EGFP gene. When the resulting chimeric gene constructs were introduced into zebrafish, the transgenic zebrafish emit green fluorescence under a blue light or ultraviolet light according to the specificity of the promoters used. Thus, new varieties of ornamental fish of different fluorescence patterns, e.g., skin fluorescence, muscle fluorescence, skeletal muscle-specific and/or ubiquitous fluorescence, are developed.

The present invention relates to a recombinant expression cassette comprising a polynucleotide encoding a SMN protein. This cassette can be included in a gene therapy vector and used in a method for the treatment of spinal muscular atrophy (SMA).

Disclosed are a recombinant expression vector and a host cell that contains the vector.

Regimens useful treating a human patient having familial hypercholesterolemia are described. Such regimens comprise co-administration of corticosteroids with a suspension of replication deficient recombinant adeno-associated virus (rAAV) comprising LDLR.

Methods and constructs for inserting an intron into a reporter protein coding sequence in a eukaryotic cell and their application of monitoring and reporting genomic modifications are provided. Various related compositions, cells and kits are also provided.

The present invention relates to nucleic acid regulatory elements that are able to enhance liver-specific expression of genes, methods employing these regulatory elements and uses of these elements. Expression cassettes and vectors containing these nucleic acid regulatory elements are also disclosed. The present invention is particularly useful for applications using gene therapy, more particularly liver-directed gene therapy, and for vaccination purposes.

The present invention relates to a polynucleotide comprising at least one promoter, at least one expressible construct, and an S/MAR element, wherein said polynucleotide is an integration construct or a non-integrative vector construct, wherein said S/MAR element is located downstream of said promoter and of said expressible construct, and wherein said S/MAR element is flanked by a splice donor and a splice acceptor. The present invention also relates to a composition and a host cell comprising said polynucleotide, as well as to uses and methods related thereto.