This technology includes a novel concept and design for a lipoprotein targeting protease inhibitor for the treatment of Alpha-1-antitrypsin (A1AT) deficiency. A1AT deficiency occurs in about 1 in 2500 individuals in the United States and Europe, and people with this condition develop severe liver disease and emphysema/chronic obstructive pulmonary disease (COPD). Current treatment involves intravenous infusion of purified human A1AT protein, which is very expensive and only modestly effective.

This technology includes a novel capsid protein for recombinant adeno-associated virus (AAV)-mediated gene transfer evaluation. We have identified a "fossilized" endogenous AAV sequence element (referred to as mAAV-EVE) within the germline of an ancient lineage of Australian marsupials and have cloned and sequenced mAAV-EVE orthologs from at least fifteen lineage-specific taxa.

This technology includes a strategy for retrieving a pair of implanted magnets from a patient’s body. This method includes maneuvering a guide catheter with a guide magnet on the distal end. Current methods are geared toward magnetic compression occurring in the digestive system, while this method can be used as a less invasive method for cardiac magnetic compression.

This technology includes efficient lentiviral gene delivery system for both guide RNA and Cas9 endonuclease as a method to cure sickle cell disease. Gene correction is an ideal gene therapy strategy for hereditary disease, including sickle cell disease.

This technology includes in vitro-generated trophectoderm (TE) cells, which are ideal for modeling diseases of the placenta, drug screening, and cell-based therapies. The TE lineage which gives rise to placental cells during early human development. Derivation of definitive placental cells from human pluripotent stem cells in culture remains controversial and so far, placental cells can only be derived directly from primary placental tissue, which largely limits their access and study in the laboratory.

This technology includes a novel cardiac patch which was 3D printed to repair damaged cardiac tissue. Based on biological and anatomical understanding of myocardial tissue, a novel 3D bioprinting technique was developed to directly fabricate the cellularized and vascularized cardiac patch with anisotropic fiber and perfusable vessel architecture. The design will integrate biomimetic aligned myocardial fibers and perfusable blood vessels to create a thick, functional cardiac patch, suitable for the human heart implantation.

This technology includes a pair of subsystems for a novel transcatheter bicuspid valve (frame and leaflets) intended for implantation in the mitral position. It is simple, it overcomes key limitations to transcatheter bicuspid mitral valve implants, and it overcomes key limitations to transcatheter tricuspid mitral valve implants.

This technology includes the use of nanodiamonds to achieve background-free imaging. We present several techniques to reduce or eliminate background florescence by exploiting properties of the fluorescent nanodiamonds. In particular, magnetic field modulation of the fluorescence intensity offers a simple, robust, and easily adaptable method to obtain background free imaging in a variety of imaging modalities, i.e., fluorescence microscopy and wide field fluorescence animal imaging.

This technology includes a new class of nanoparticles in the carbon family, fluorescent nanodiamonds (FNDs), exhibiting superb physical and chemical properties for diagnostic imaging applications. We have developed a simple, fast, and robust method to encapsulate FNDs in polydopamine that can be further functionalized. By integrating anatomical and molecular based imaging capabilities, multimodal nanoparticle probes are becoming important in the paradigm shift from conventional to future imaging technologies.

This technology includes the use of LZK and DLK inhibitors to be used for the treatment of head and neck squamous cell carcinoma (HNSCC) or lung squamous cell carcinoma (LSCC). Specifically, we demonstrate that inhibitors that can be repurposed to target LZK suppresses LZK kinase-dependent stabilization of MYC and activation of the PI3K/AKT pathway. In vivo preclinical cell line xenograft mouse model demonstrates that targeting LZK will suppress tumor growth. We also demonstrate that several additional compounds potently inhibit LZK and could serve as new therapeutic modalities.