Disclosed herein are vector systems for expression of polypeptides in eukaryotic cells; and methods of obtaining high-level expression of polypeptides in a eukaryotic cell. Methods and compositions for obtaining stable, long-term expression of recombinant polypeptides are also provided.

MiniVectors and compositions containing MiniVectors that target genes implicated in IPF selected from CDH11, STAT3, STAT6, FoxM1, MDM2, TGF?, SMAD, PDGFA, or TLR4 and/or increase intracellular levels of reduced glutathione, relaxin, and p53, are provided, along with uses in the treatment of idiopathic pulmonary fibrosis.

The presently disclosed subject matter provides for expression cassettes that allow for expression of a globin gene or a functional portion thereof, vectors comprising thereof, and cells transduced with such expression cassettes and vectors. The presently disclosed subject matter further provides methods for treating a hemoglobinopathy in a subject comprising administering an effective amount of such transduced cells to the subject.

The present invention provides constructs for producing modified recombinant adeno-associated virus (rAAV) vectors. The constructs comprise one or more CCCTC-binding factor (CTCF) binding sites, which facilitate DNA looping and promote efficient transgene expression. Also provided are modified rAAV virus particles comprising these constructs, methods for producing the modified rAAV virus particles, and methods of using the modified rAAV virus particles to deliver a transgene to a subject.

Disclosed are constructs and methods for expressing DNAs of interest in particular in non-primate eukaryotic host cells that display advantages with regard quantity and quality of expression including high stability of expression and, if appropriate, transport of the expression product out of the cell.

Compositions can be used to stimulate growth of a hair shaft from a hair follicle. These compositions can include methylated polynucleotides useful in treatment of autoimmune diseases or conditions, including those, such as alopecia areata, that result in hair loss.

The current invention reports a promoter that has the nucleic acid sequence of SEQ ID NO: 02 or SEQ ID NO: 03 which is a human CMV major immediate-early (hCMV-MIE) promoter/enhancer with C to G point mutation at position 41 and/or 179 relative to the transcription start site. This new promoter is especially useful for the production of polypeptides at large scale as it shows reduced promoter silencing and improved polypeptide production.

A method for stably achieving high expression of a foreign gene in mammalian cells using a novel DNA element is disclosed. More specifically, the present application discloses a DNA element which enhances the activation of transcription by changing the chromatin structure around a gene locus into which a foreign gene expression unit has been introduced.

The present disclosure teaches the treatment of a blood pathology, such as a blood pathology associated with impaired hemoglobin synthesis including the treatment of -thalassemia or a related hemoglobinopathy.

Disclosed is a method for stably achieving high expression of a foreign gene in mammalian cells using a novel DNA element. More specifically disclosed is a DNA element which enhances the activation of transcription by changing the chromatin structure around a gene locus into which a foreign gene expression unit has been introduced.