The present invention relates to a nucleic acid sequence derived from the regulatory region of the human gamma-synuclein gene and having a promoter activity in retinal ganglion cells. The present invention also relates to expression cassettes or vectors comprising said promoter operably linked to a nucleic acid sequence encoding a polypeptide of interest as well as viral particles or host cells comprising said expression cassette or vector. The present invention also relates to the use of said expression cassettes, vectors, viral particles or cells in the treatment of ocular disease, in particular ocular disease associated with retinal ganglion cell or photoreceptor cell degeneration.

Provided herein are methods and compositions useful in the production of recombinant AAV (rAAV) in insect cells. In some embodiments, methods and compositions include the use of modified Kozak sequences to express AAV VP1 proteins in amounts that are useful for producing infective rAAV particles.

Provided herein are nucleic acids, recombinant adeno-associated viral particles, compositions and methods related to treating Friedreich's ataxia. In some examples, the nucleic acids, recombinant adeno-associated viral particles, compositions and methods involve us of a FXN coding sequence, a truncated FXN 3′ UTR, and a prompter.

Provided herein are nucleic acids, recombinant adeno-associated viral particles, compositions and methods related to treating Friedreich's ataxia. In some examples, the nucleic acids, recombinant adeno-associated viral particles, compositions and methods involve use of a FXN coding sequence, a truncated FXN 3′ UTR, and a promoter.

The present invention relates to the provision of immunogenic or vaccine compositions comprising at least one recombinant Zika virus antigen, wherein the at least one recombinant Zika virus antigen is encoded by at least one nucleic acid sequence encoding at least one E-protein of a Zika virus or a functional fragment thereof. Further provided are nucleic acid molecules and a recombinant chimeric virus encoding and/or comprising selected antigens from a Zika virus, which are suitable as vaccine compositions. Preferably, the sequences encoding at least one Zika virus antigens suitable for eliciting an immune response are operably linked to a non-flavivirus derived vector backbone. Further provided are methods for purifying the recombinant chimeric virus particles or the immunogenic composition. Finally, there is provided an immunogenic/vaccine composition for use in a method of preventing or treating a Zika virus disease.

Provided herein are methods and compositions useful in the production of recombinant AAV (rAAV) in insect cells. In some embodiments, methods and compositions include the use of modified Kozak sequences to express AAV VP1 proteins in amounts that are useful for producing infective rAAV particles.

Provided is a polynucleotide, the polynucleotide can be used as a WXRE transcriptional regulatory element used to increase the protein expression level of a protein expression system. A protein expression vector or a protein expression system comprising the above-mentioned WXRE transcriptional regulatory element as well as the use thereof are also provided. The use of the WXRE transcriptional regulatory element can increase the expression level of a heterologous protein greatly with its biological activity unchanged.

The present invention provides novel mRNA elements capable of forming hairpin, double-stranded RNA structures independent of other non-coding RNAs. These mRNAs are stably expressed, lack polyadenylation tails, and allow minimal protein translation except when in the presence of specific proteins. Also provided, are compositions and kits comprising the mRNA element, as well as methods for its use in the regulation of protein translation. Advantageously, the disclosed elements represent a novel tool useful in regulating the expression of a wide variety of proteins of interest.

A hybrid promoter for recombinant expression of proteins of interest is disclosed that combines a mCMV enhancer sequence with a rat EF-1alpha intron sequence. Also disclosed are an expression cassette containing the hybrid promoter and a recombinant expression vector containing the expression cassette. A mammalian host cell, which comprises the recombinant expression vector is also disclosed, as is a method of producing a protein of interest that employs the mammalian host cell, optionally involving tetracycline-inducible expression of the protein.

Provided herein are improved gene therapy vectors and methods of use, in some embodiments, comprising sequences for improved expression and cellular targeting of a therapeutic protein.