Derivation of a >25 million-year-old Adeno-associated Virus Coat Protein Sequence for Gene Transfer Studies

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.

High Density Lipoprotein Targeting Protease Inhibitor Peptide for the Treatment of Alpha-1-antitrypsin (A1AT) Deficiency

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.

Application of AAV44.9 Vector in Gene Therapy for the Inner Ear

This technology includes a novel AAV isolate (AAV44.9) to be used as gene therapy for the inner ear for the treatment of deafness. The ability of AAV vectors to transduce dividing and non-dividing cells, establish long-term transgene expression, and the lack of pathogenicity has made them attractive for use in gene therapy applications. Vectors based on new AAV isolates may have different host range and different immunological properties, thus allowing for more efficient transduction in certain cell types.

Identification of EGFR as A Receptor for AAV6 Transduction

AAV vectors offer unique advantages in gene therapy applications. Studies have shown that these replication deficient parvovirus vectors can deliver DNA to specific tissues and confer long-term transgene expression in a variety of systems. Although many studies have looked at the tissue-specific expression elicited by each of the AAV serotypes, a true understanding of how AAV transduces these tissues is still unclear. Of the large AAV family, only a few receptors or co-receptors have been identified.

mTOR Inhibition for the Prevention of Epithelial Stem Cell Loss and Mucositis

The integrity of the epidermis and mucosal epithelia is highly dependent on self-renewing stem cells and, therefore, is vulnerable to physical and chemical damage from common cancer treatments, such as radiation or chemotherapy. Consequently, many cancer patients undergoing these treatments develop mucositis, a debilitating condition involving painful and deep mucosal ulcerations. Since current prevention and treatment options for mucositis are limited, providing only minor relief and no protection to stem cells, novel therapies are needed.

Diagnostic Biomarker of Metastasis for Improved Clinical Management of Head and Neck Cancer

Squamous Cell Carcinoma of the Head and Neck (HNSCC) is associated with poor prognosis due to the advanced stage of disease (metastasis) typically found at the time of diagnosis. Investigators at the NIH have developed a sensitive method using a protein biomarker for detecting even just a few HNSCC tumor cells in lymph nodes with occult disease.

Vitamin C renal leak as a clinical diagnostic tool in the detection, monitoring, and management of acute and chronic diseases

This technology includes a clinical diagnostic tool for measuring vitamin C elimination by human kidneys that can be used for detecting, monitoring, and managing acute and chronic diseases. Findings revealed significant associations between vitamin C renal leak status and clinical variables affecting renal function and blood glucose. The technology uses vitamin C depletion-repletion kinetics and pharmacokinetic models to establish a physiological vitamin C renal threshold.

Locally Delivered Alkaline Phosphatase for Treatment of Periodontal Disease

This technology includes a product for local delivery of alkaline phosphatase for the treatment of periodontal disease. Our laboratory has discovered that factors regulating phosphate metabolism and specifically the appropriate balance between phosphate (Pi) and pyrophosphate (PPi) at local sites are needed for formation (development), maintenance and regeneration of the tooth root surface (cementum), periodontal ligament (PDL) and surrounding alveolar bone, i.e., the periodontal apparatus.

Apparatus for Cryogenic-Electron Microscopy Sample Preparation

Cryo-Electron Microscopy (cryo-EM) is used to obtain high-resolution structural images of macromolecular structures. Samples must be purified and loaded onto cryo-EM grids before imaging. The ideal cryo-EM grid consists of particles that are evenly and richly distributed in a broad distribution of orientations throughout the holes of the support film. Current techniques to prepare cryo-EM grids are performed manually and require trial and error, resulting in a bottleneck in cryo-EM workflows.

3D Bioprinting of Cardiac Patch with Anisotropic and Perfusable Architecture for the Repair of Damaged Cardiac Muscle

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.

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