Multiplexing Homocysteine in Primary Newborn Screening Assays Using Maleimides as Select Derivatization Agents

Homocystinuria (HCU), a group of inherited disorders, causes symptoms ranging from failure to thrive and developmental delays in infants or young children to abnormal blood clots with onset in adults.1 Approximately 1 in 200,000 to 335,000 people have HCU globally.2

Minibody for Conditioning prior to Hematopoietic Stem Cell and Progenitor Cell Transplantation

Patient conditioning is a critical initial step in hematopoietic stem and progenitor cell (HSPC) transplantation procedures to enable marrow engraftment of infused cells. Conditioning regimens have traditionally been achieved by delivering cytotoxic doses of chemotherapeutic agents and radiation. However, these regimens are associated with significant morbidity and mortality, and cannot be used safely in elderly or subjects with comorbidities.

Compatible 3-D Intracardiac Echography Catheter and System for Interventional Cardiac Procedures

This technology includes a versatile intravascular 3D intracardiac echocardiography (ICE) catheter that can operate under conventional X-ray and MRI for use during interventional cardiac procedures. The 3D MRICE and custom, GPU-based, real-time imaging system are also included. Structural heart disease affects more than 2.9% of the US population, and common interventional procedures can be difficult because of limitations in catheter devices and inadequate image guidance.

Trans-auricular Left Atrial Appendage Ligation to Prevent Thrombosis

This technology includes an interventional device to occlude the left atrial appendage to prevent thrombus formation. Atrial fibrillation is the most common cardiac arrhythmia and is associated with formation of thrombus in the left atrial appendage. Standard preventative treatment involves anticoagulation, which is not tolerated by all patients. Existing devices necessitate improvement because they need trans-septal puncture and anticoagulation to prevent thrombus or are prone to life-threatening complications.

Single Scan Bright-blood and Dark-blood Phase Sensitive Inversion Recovery (PSIR) Late Gadolinium Enhancement (LGE) for Cardiovascular Magnetic Resonance (CMR) Imaging

This technology includes a technique to improves detection of myocardial scar compared with conventional bright-blood late gadolinium enhancement (LGE) techniques. Dark-blood late gadolinium enhancement (DB-LGE) improves tissue delineation with signal suppression of the blood pool based on T2-preparation pulse that is relatively independent from the blood flow velocities and improves scar detection in patients with known or suspected coronary artery disease.

Oral Treatment of Hemophilia

This invention portrays a simple method for treatment of antigen-deficiency diseases by orally administering to a subject a therapeutically effective amount of the deficient antigen, wherein the antigen is not present in a liposome. This method increases hemostasis in a subject having hemophilia A or B, by orally administering to the hemophiliac a therapeutically effective amount of the appropriate clotting factor, sufficient to induce oral tolerance and supply exogenous clotting factor to the subject.

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.

In-vivo System to Interrogate the Functions of Mucous Membranes and Identify Mucin/Glycan Mimetics and JAK/STAT Inhibitors for the Treatment of Diseases of the Oral Cavity and Digestive Tract

This technology includes a Drosophila mutant strain that can be used as an in vivo model for diseases of the oral cavity and digestive tract (Sjogren's syndrome, colitis, colon cancer, inflammatory bowel disease), where the mucous membrane is disrupted or non-functional. This mutant lacks a mucous membrane and displays epithelial cell damage, uncontrolled cell proliferation and the up-regulation of conserved signaling pathways (JAK/STAT).

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.

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