The present invention provides PiggyBac transposase proteins, nucleic acids encoding the same, compositions comprising the same, kits comprising the same, non-human transgenic animals comprising the same, and methods of using the same.

Described are nucleic acid regulatory elements that are able to enhance liver-specific expression of genes, methods employing these regulatory elements and uses of these elements. Expression cassettes and vectors containing these nucleic acid regulatory elements are also disclosed. These are particularly useful for applications using gene therapy.

The present invention relates to the field of therapeutic use of proteins, genes and cells. More specifically the invention relates to therapy based on the biological function of a secreted therapeutic protein, NsG33, in particular for the treatment of disorders of the nervous system. NsG33 is a nerve survival and growth factor with antiapoptotic effects on a cell line with neuronal potential and with neuroprotective and/or neurogenesis effects on a neural precursor cell line and on primary striatal cultures. The invention also relates to novel bioactive NsG33 polypeptide fragments and the corresponding encoding DNA sequences.

The invention features nucleic acid constructs encoding chimeric immune T-cell receptors (CIRs) that are useful for treating HIV in patients. In general, the CIRs contain an extracellular domain which targets HIV or HIV infected cells (e.g., the extracellular domain of CD4), a transmembrane domain, and a cytoplasmic domain for mediating T-cell activation (e.g., CD3 zeta and/or the partial extracellular domain of CD28). The invention also features the use of host cells expressing CIRs in the treatment of HIV.

The present invention generally relates to methods of identifying modulators of central nervous system diseases and the use of the modulators in treatment and diagnosis. The methods utilize a novel high throughput screen that includes injection of a library of barcoded viral vectors expressing shRNA's, CRISPR/Cas systems or cDNA's into animal models of disease and detecting synthetic lethality.

The present invention is transgenic chickens obtained from long-term cultures of avian PGCs and techniques to produce and transgenic birds derived from prolonged PGC cultures. In some embodiments, these PGCs can be transfected with genetic constructs to modify the DNA of the PGC, specifically to introduce a transgene encoding an exogenous protein. When combined with a host avian embryo by known procedures, those modified PGCs are transmitted through the germline to yield transgenic offspring. This invention includes compositions comprising long-term cultures of PGCs and offspring derived from them that are genetically modified. The genetic modifications introduced into PGCs to achieve the gene inactivation may also include, but are not restricted to, random integrations of transgenes into the genome, transgenes inserted into the promoter region of genes, transgenes inserted into repetitive elements in the genome, site specific changes to the genome that are introduced using integrase, site specific changes to the genome introduced by homologous recombination, and conditional mutations introduced into the genome by excising DNA that is flanked by lox sites or other sequences that are substrates for site specific recombination.

Provided herein are methods of obtaining induced pluripotent stem cells from cells of a hematopoietic lineage.

A method for increasing the cell surface expression and/or reducing mispairing of a TCR.

The present invention relates to compositions and methods for generating RNA Chimeric Antigen Receptor (CAR) transfected T cells. The RNA-engineered T cells can be used in adoptive therapy to treat cancer.

The invention provides an isolated nucleic acid encoding a receptor, other than an immunoglobulin, wherein the receptor binds to a MUC1 tumor antigen independently of an major histocompatibility complex (MHC). The invention provides a method of activating a signaling pathway and/or killing a cancer cell using a receptor that is similar to or is a T cell receptor.