DLX3-floxed mice (DLX3f/f) for Use in Drug Development and In Vivo Research Studies for Ectodermal Dysplasia Disorders

This technology includes the creation of DLX3-floxed mice, specifically designed for conditional deletion of the DLX3 gene via Cre-mediated recombination. This innovative approach aims to develop mouse models for studying ectodermal dysplasia disorders. Ectodermal dysplasias are a diverse group of genetic conditions affecting the development of ectodermal structures, including hair, teeth, and bones. The DLX3f/f mice are particularly valuable for modeling specific disorders such as Tricho-dento-osseous syndrome (TDO), Amelogenesis Imperfecta (AI), and Dentinogenesis Imperfecta (DI).

Identification of a Novel Parvovirus for Vaccine Development and Use as a Diagnostic Tool

This technology includes a procedure for novel virus identification in a variety of human specimens by solexa high-throughput sequencing, which allows for the screening a large number of clinical specimens for novel virus discovery in a highly efficient and relatively economical method. By using this technique, we have successfully identified a novel parvovirus from samples of seronegative hepatitis patients.

Generation of AAVS1 and C13 “Safe Harbor” Transcription Activator-life Effector Nucleases (TALENs) for Drug Screening or Gene Therapy Development

This technology includes AAVS1 and C13 “safe harbor” transcription activator-life effector nucleases (TALENs) for drug screening or gene therapy applications. TALENs are engineered sequence-specific DNA endonucleases that can significantly enhance genome-editing efficiency by >100-1000 folds. “Safe harbor” such as AAVS1 safe harbor and C13 safe harbor is genome locus that allows robust and persistent transgene expression with no or minimal interference of endogenous gene expression and cell properties.

Anti-sense Therapy Against ApoC-III as a Treatment for High Cholesterol

This technology includes a new class of synthetic peptides that activate Lipoprotein Lipase (LPL), a key plasma enzyme that lowers triglycerides, by displacing apoC-111, a potent inhibitor of LPL. ApoC-11 is a known activator of LPL, whereas ApoC-111 inhibits LPL and raises triglycerides either directly by blocking lipolysis and or by preventing hepatic uptake of lipoproteins. Both apoC-II and apoC-III have to bind to the surface of a lipoprotein particle to mediate their effects.

Novel ApoC-11 Mimetic Peptides That Activate LPL for the Treatment of ApoC-11 Deficiency and Hypertriglyceridemia

This technology includes a new class of synthetic peptides that activate Lipoprotein Lipase (LPL), a key plasma enzyme that lowers triglycerides. Mutations in apoC-II is a genetic cause of severe hypertriglyceridemia, which can lead to cardiovascular disease and pancreatitis.

Infectious Clone of Human Parvovirus B19 and Methods of Use

This technology described in this patent application relates the first reported infectious human parvovirus B19 clone, methods of cloning the parvovirus B19 genome as well as other viral genomes that have secondary DNA structures that are unstable in bacterial cells. The infectious clone and methods of producing the same would be useful in producing infectious virus, which can in turn be used, among other things, to identify and develop therapeutic agents for treatment and/or prevention of human parvovirus B19 infections. The infectious parvovirus B19 clone is also available for licensing.

ApoA-1 Mimetic Peptides Promoting Lipid Efflux from Cells for Treatment of Vascular Disorders

This invention involves ApoA-1 mimetic peptides with multiple amphipathic alpha-helical domains that promote lipid efflux from cells and are useful in the treatment and prevention of dyslipidemic, inflammatory and vascular disorders. IND-enabling studies for one of the peptides, named Fx-5A, are completed in preparation for an IND filing at the FDA, to be followed by a Phase I clinical trial planned for 2017.

Selections of Genes

The invention provides selections of genes expressed in a cancer cell that function to characterize such cancer, and methods of using the same for diagnosis and for targeting the therapy of selected cancers. In particular, methods are provided to classify cancers belonging to distinct diagnostic categories, which often present diagnostic dilemmas in clinical practice, such as the small round blue cell tumors (SRBCTs) of childhood, including neuroblastoma (NB), rhabdomyosarcoma RMS), Burkitt’s lymphoma (BL), and the Ewing family of tumors (EWS).