A composition for use in formulations for controlling insect populations, including populations of mosquito and flies. The composition comprises one or more double-stranded constructs inhibitory to RNA transcription of ribosomal proteins. The invention also relates to method of using the compositions in formulations to inhibit insect populations.

Delivery devices, systems, and methods related thereto may be used in humans for spinal delivery of cells, drugs or vectors. Thus, the system enables subpial delivery, which leads to a near complete spinal parenchymal AAV9-mediated gene expression or ASO distribution in both white and grey matter.

Compositions and methods are provided for the inhibition, treatment and/or prevention of Huntington's disease and related disorders.

The invention provides for recombinant AAV vectors comprising a polynucleotide sequence comprising the guide strand of miR-29c and methods of using the recombinant vectors to reduce or prevent fibrosis in subjects suffering from muscular dystrophy.

In one aspect, the embodiments disclosed herein relate to the production of fully-deleted adenovirus-based gen delivery vectors packaged without the use of an adenoviral helper virus, and more particularly in their use in the transfer of genes and the expression of proteins, vaccine development, and cell engineering. In another aspect, the production of adenoviral vectors deleted of all adenoviral genes is described that carry genes of interest with detrimental or toxic activities to eukaryotic cells.

Embodiments of the disclosure encompass systems, methods, and compositions related to selective advantages to somatic cells that harbor one or more particular genetic modifications. In particular embodiments, there is selective expansion of gene-targeted cells wherein the strategy involves deletion of an essential gene product that is replaced with targeted integration that also includes integration of a therapeutic transgene. The cells that harbor the replaced essential gene product, and thereby the therapeutic transgene, are selected for using pharmaceutical or nutritional agents that are linked to the function of the essential gene product.

Nucleic acids and viral vectors, particularly adeno-associated virus (AAV) vectors are provided that encode Cas9 and paired guide RNAs. The nucleic acids and vectors, and compositions that comprise them, can be used in methods to treat subjects, to alter cells in subjects who may suffer from an inherited retinal dystrophy such as CEP290 associated disease or who may be in need of alteration of a cell or a cellular nucleic acid sequence associated with an inherited retinal dystrophy such as the CEP290 gene, and/or to treat inherited retinal dystrophies including CEP290 associated disease.

The invention includes materials and methods to generate numerous small RNAs from one polynucleotide construct (synthetic gene) to facilitate RNA-guided multiplex genome editing, modification, inhibition of expression and other RNA-based technologies. The synthetic gene/polynucleotide construct encodes polycistronic RNA components separated by tRNAs, and preferably also includes regulatory components such as a promoter or terminator to form an expression cassette. Once transcribed in a cell, the transcript is processed by the cell to multiple RNA molecules by the endogenous tRNA processing system. The system can be sued for any RNA based gene manipulation method including RNA-mediated genome editing, artificial microRNA mediated gene silencing, small RNA mediated genetic manipulation, double-stranded RNA mediated gene silencing, antisense mechanisms and the like.

Disclosed are compositions, methods, and kits for performing cell-free protein synthesis (CFPS) and for expressing proteins in cells. Particularly disclosed are vectors comprising Golden Gate sites for cloning, methods for preparing such vectors, and the use thereof for performing CFPS and for expressing proteins in cells such as in naturally occurring or recombinant species of Clostridia, including Clostridium autoethanogenum.

The invention provides compositions and methods for reducing the immunogenicity of cells for transplant including cell-based immunotherapies. Vectors encoding beta 2 microglobulin (B2M) modifying RNAs along with targeting moieties and other signaling and/or suicide genes allow for efficient production of engineered CAR T-regulatory or other therapeutic cells from any source.