The present disclosure provides rAAV vectors and rAAV virions that specifically express exogenous nucleic acid sequences in CD14.sup.+ cells. The rAAV vectors or virions are useful for specifically expressing exogenous nucleic acid sequences encoding, for example, cancer antigens, viral antigens, and/or bacterial antigens in monocytes and dendritic cells. The rAAV transduced CD14.sup.+ cells can be used as antigen presenting cells that induce antigen-specific T cell responses. The present disclosure further provides methods producing rAAV virions and methods of immunotherapy.

The present invention relates to nucleic acid regulatory elements that are able to enhance heart-and/or muscle-targeted expression of genes, in particular heart- and muscle-targeted gene expression, more particularly gene expression in cardiomyocytes, 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 heart- and/or muscle-directed gene therapy, e.g. for the treatment of cardiovascular diseases and disorders and muscle disorders, as well as other diseases and disorders that may benefit from high transgene expression in heart and/or muscle cells or tissue, and for vaccination purposes.

The present invention concerns a genetic tool comprising at least two distinct nucleic acids optimized to facilitate the transformation and modification by homologous recombination of a bacterium of the genus Clostridium, typically a solventogenic bacterium.

Methods of modulating expression of a target nucleic acid in a cell are provided including use of multiple orthogonal Cas9 proteins to simultaneously and independently regulate corresponding genes or simultaneously and independently edit corresponding genes.

The present invention concerns a genetic tool comprising at least two distinct nucleic acids optimized to facilitate the transformation and modification by homologous recombination of a bacterium of the genus Clostridium, typically a solventogenic bacterium.

Disclosed herein are recombinant baculoviruses suitable for introducing an exogenous gene into a pest insect, particularly, disease-transmitting mosquitos. The recombinant baculovirus is characterized in having a promotor that is any of a HzNV-1 viral early expressing gene pag1, a ceropin gene b1, a defensin gene a4, or hp70 gene; and an exogenous gene operably linked thereto the promoter. Also disclosed herein is a method of introducing an exogenous gene into a pest insect. The method includes transducing the pest insect with a recombinant baculovirus without suppressing the production of microRNAs (miRNAs) in the pest insect, wherein the recombinant baculovirus comprises a promoter of pag1, cecropin b1, defensin gene a, or hp70.

The present disclosure provides compositions and methods for the treatment of ocular diseases associated with angiogenesis, particularly wet age-related macular degeneration.

Disclosed are replication-enhanced oncolytic adenoviruses. These oncolytic adenoviruses have tumor-specific replication capable of enhanced tumor oncolysis and enhanced therapeutic transgene expression. Also disclosed are methods comprising administering a replication-enhanced oncolytic adenovirus for patients suffering from a cancer.

The present disclosure relates to: an adipocyte-targeting non-viral gene delivery complex comprising a sh(FABP4+FABP5) dual plasmid vector; and treatment for obesity and obesity-induced metabolic syndromes by using the same and, more particularly, to a gene delivery complex comprising: an adipocyte-targeting sequence; a nine-arginine (R9) peptide; and a dual plasmid vector comprising a gene for treatment of obesity and obesity-induced metabolic syndromes, wherein the gene for treatment of obesity and obesity-induced metabolic syndromes is a base sequence inhibiting the expression of a FABP4 gene and a FABP5 gene. According to the present disclosure, in order to treat obesity-related diseases, a dual plasmid vector capable of simultaneously inhibiting the FABP4 and FABP5 genes is produced, and this vector is bound to a predetermined delivery system that specifically delivers the vector into adipocytes so as to provide a gene delivery complex. In this way, it is possible to achieve an excellent therapeutic effect on obesity which targets only adipocytes without cytotoxicity.

Expression of a transgene is driven in a host cell using a pol I promoter which is not endogenous to an organism from the same taxonomic order from which the host cell is derived.