Sequences of novel adeno-associated virus capsids and vectors and host cells containing these sequences are provided. Also described are methods of using such host cells and vectors in production of rAAV particles. AAV-mediated delivery of therapeutic and immunogenic genes using the vectors of the invention is also provided.

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.

Adeno-associated virus rh.10 sequences, vectors containing same, and methods of use are provided.

Sequences of novel adeno-associated virus capsids and vectors and host cells containing these sequences are provided. Also described are methods of using such host cells and vectors in production of rAAV particles. AAV-mediated delivery of therapeutic and immunogenic genes using the vectors of the invention is also provided.

Methods for treating a neurological disorder, such as a traumatic spinal cord injury or traumatic brain injury, or a disorder such as Parkinson's disease or multiple sclerosis are provided. Such methods include administering a therapeutically effective amount of Gsx1 protein (such as a Gsx1-cell penetrating peptide fusion protein), or a nucleic acid molecule encoding such a protein (for example as part of a viral vector), thereby treating the neurological disorder.

Sequences of novel adeno-associated virus capsids and vectors and host cells containing these sequences are provided. Also described are methods of using such host cells and vectors in production of rAAV particles. AAV-mediated delivery of therapeutic and immunogenic genes using the vectors of the invention is also provided.

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.

Disclosed herein are methods and compositions for radiosensitizing tumor in subjects receiving radiation therapy by administering a gene therapy construct that results in expression of a secretory radiosensitizing agent in tumor endothelium.

The invention provides bidirectional expression vectors comprising Chinese hamster ovary elongation factor 1-a (CHEF1) transcriptional regulatory DNA elements, a gene of interest (GO I), a minimal cytomegalovirus (minCMV) and a selectable marker (SM) and/or a human adenovirus tripartite leader (AdTPL) sequence. The invention also provides method for increasing heterologous protein expression in a host cell comprising culturing the host cell the bidirectional expression vector(s).

The present invention provides nucleic acid constructs, expression vectors, transgenic cell and methods of making and using the same, wherein the nucleic acid construct includes a synthetic promoter designed from the endogenous promoter of BIRC5 and LAMC2. In illustrative working embodiments of the invention, an exogenous nucleic acid fragment encoding thymidine kinase is operably linked to the synthetic promoter which is then shown to regulate the expression of this polypeptide.