Abstract: Methods for preparing highly purified rLV vector formulations at the scale needed to meet anticipated demand for human gene therapy are provided.

Abstract: Methods, devices, and systems are provided for the delivery of agents (e.g., nucleic acids, proteins, organic molecules, organelles, antibodies or other ligands, 5 etc.) into live cells and/or the extraction of the same from said cells. In various embodiments the photothermal platforms and systems incorporating such photothermal platforms are provided that permit efficient, high-throughput cargo delivery into live cells.

Abstract: The disclosure in some aspects, relates to nucleic acids, compositions and kits useful for gene therapy with reduced immune response to transgene products.

Abstract: 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.

Abstract: The invention provides a novel compounds derived from CD154, but without the intracellular domain. Notably, the invention provides TMZ-CD154 and a method to treat diseases in which the novel molecule TMZ-CD154 is used. In particular, TMZ-CD154 is used to modify the immune responses in patients with immune related diseases such as cancer and infectious disease. The invention also relates to a method to activate cells with the TMZ-CD154 in vitro either prior to using the activated cells for therapy or for diagnostic purposes.

Abstract: In certain aspects the present invention provides engineered neural cells, neural stem cells, or neural progenitor cells that contain a nucleotide sequence that encodes an adenovirus E40RF1 polypeptide and/or that contain an adenovirus E40RF1 polypeptide. The present invention also provides methods of making and using such engineered cells and compositions comprising such engineered cells.

Abstract: The invention provides a method of treating ovarian cancer in a patient by administering to the patient an adeno-associated virus (AAV) vector encoding an anti-VEGF antibody or antigen binding fragment thereof.

Abstract: The present invention provides polynucleotide vectors for high expression of heterologous genes. Some vectors further comprise novel transposons and transposases that further improve expression. Further disclosed are vectors that can be used in a gene transfer system for stably introducing nucleic acids into the DNA of a cell. The gene transfer systems can be used in methods, for example, gene expression, bioprocessing, gene therapy, insertional mutagenesis, or gene discovery.

Abstract: Genetic tests, such as whole genome analysis (WGA), have been employed to identify genetically superior embryos. The disclosed methods extend in vitro culture time of embryos while awaiting results of genetic tests being performed on a portion of the same embryos. The disclosed methods also help expand the number of cells in each embryo before implantation in the recipient.

Abstract: An artificial AAV capsid comprising a heterologous conducting airway targeting sequence is provided. The artificial AAV is useful as a targeting moiety, for delivery of heterologous molecules which are associated therewith. The artificial AAV is also useful in the generation of AAV vectors having the artificial capsid. Also described are methods of modifying the native tropism and transduction efficiency of vectors by improving and/or ablating their ability to transduce conducting airway cells. Methods of targeting conducting airway cells and delivering therapeutic and other molecules thereto are also provided.

Abstract: The present invention provides compositions and methods of use pertaining to rAAV-mediated delivery of therapeutically effective molecules for treatment of diseases such as Pompe disease. These compositions in combination with various routes and methods of administration result in targeted expression of therapeutic molecules in specific organs, tissues and cells.

Abstract: The present invention provides a genetically recombinant vaccinia virus effective in preventing or treating cancer. Specifically, the present invention provides a vaccinia virus comprising two polynucleotides, a polynucleotide encoding IL-7 and a polynucleotide encoding IL-12; a combination kit of two vaccinia viruses, a vaccinia virus comprising a polynucleotide encoding IL-7 and a vaccinia virus comprising a polynucleotide encoding IL-12; and use of the two vaccinia viruses in combination.

Abstract: Provided are cells containing exogenous antigen and uses thereof.

Abstract: Disclosed is a new process for the production of recombinant proteins, by transient transfection of suspension-grown human embryonic kidney cells (293 cell line and its genetic variants) with an expression vector, using polyethylenimine (PEI) as a transfection reagent. In a preferred embodiment, the process uses 293E cells expressing the Epstein-Barr virus (EBV) EBNA 1 protein, in combination with an oriP-based episomal expression vector having an improved cytomegalovirus expression cassette comprising the CMV5 promoter. The process combines in a single step the cell growth, transfection and protein expression, is carried out without changing the culture medium, and allows to achieve high expression levels in a short period of time. The process may be carried out in a serum-free, low-protein culture medium, is easily scalable, compatible with continuous production processes, and fully adapted to high-throughput production of milligram quantities of recombinant proteins.

Abstract: The present invention provides a genetically recombinant vaccinia virus effective in preventing or treating cancer. Specifically, the present invention provides a vaccinia virus comprising two polynucleotides, a polynucleotide encoding IL-7 and a polynucleotide encoding IL-12; a combination kit of two vaccinia viruses, a vaccinia virus comprising a polynucleotide encoding IL-7 and a vaccinia virus comprising a polynucleotide encoding IL-12; and use of the two vaccinia viruses in combination.

Abstract: Chimeric antigen receptors containing CD123 antigen binding domains are disclosed. Nucleic acids, recombinant expression vectors, host cells, antigen binding fragments, and pharmaceutical compositions, relating to the chimeric antigen receptors are also disclosed. Methods of treating or preventing cancer in a subject, and methods of making chimeric antigen receptor T cells are also disclosed.

Abstract: The disclosure relates, in some aspects, to compositions and methods for treatment of diseases associated with aberrant lysosomal function, for example Parkinson's disease and Gaucher disease. In some embodiments, the disclosure provides expression constructs comprising a transgene encoding beta-Glucocerebrosidase (GBA) or a portion thereof, Lysosomal Membrane Protein 2 (LIMP2), Prosaposin, or any combination of the foregoing. In some embodiments, the disclosure provides methods of Parkinson's disease by administering such expression constructs to a subject in need thereof.

Abstract: Provided herein are methods for treating myocilin (MYOC) glaucoma using adeno-associated viral (AAV) vectors. In some aspects, the AAV vectors encode R-spondin 1 (RSPO1), R-spondin 2 (RSPO2), R-spondin 3 (RSPO3) or R-spondin 4 (RSPO4) and/or RNAi that targets myocilin (MYOC). In one aspect, viral particles are administered to the eye of a human subject. Viral particles encoding RSPO1, RSPO2, RSPO3 and/or RSPO4 and/or MYOC RNAi are contemplated. In some aspects, variant AAV2 particles that transduce the trabecular meshwork are provided.

Abstract: In one or more embodiments, the present invention provides novel artificial, non-naturally-occurring double stranded DNA segments (and related methods) capable of acting as decoy binding sites for oncogenic transcription factors and a general method for suppressing aberrant activity of oncogenic transcription factors that promote cancer progression. In various embodiments, the present invention involves the sequestration of targeted oncogenic transcription factors at these artificial, non-naturally occurring engineered transcription factor binding sites, which have been introduced into the cells using oncolytic or other viruses that can be engineered to selectively target cancer cells.

Abstract: Provided are a composition for ribonucleoprotein delivery, comprising a guide RNA free of 5?-terminal phosphates, and a method for ribonucleoprotein delivery, using the same.