Novel Codon-Optimized MUT Gene Therapeutic for Methylmalonic Acidemia (MMA)

Methylmalonic Acidemia (MMA) is a metabolic disorder characterized by increased acidity in the blood and tissues due to toxic accumulation of protein and fat by-products resulting in seizures, strokes, and chronic kidney failure. A significant portion of MMA cases stem from a deficiency in a key mitochondrial enzyme, methylmalonyl-CoA mutase (MUT), required to break down amino acids and lipids. Currently, there are no treatments for MMA and the disease is managed primarily with dietary restriction of amino acid precursors and liver-kidney transplantation in severe cases.

Fibroblast Cell Lines (with L444P/RecNci1 Genotype) for the Screening of Small Molecules for Gaucher Disease Treatment

This technology includes two human fibroblast cell lines to be used to study the defects in GBA1 gene and protein and to screen small molecules for involvement in Gaucher disease. Glucocerebrosidase (GBA1 or GCase or beta-glucosidase) is a lysosomal enzyme, responsible for breakdown of a fatty material called glucocerebroside (or glucosyl ceramide). Deficiency or malfunction of GBA1 leads to the accumulation of insoluble glucocerebrosides in tissues, which is a major symptom of Gaucher disease.

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.

Vascular Anchoring Introducer Sheath for Interventional Cardiac Procedures

This technology includes a device and method for maintaining access to a location in the body while reducing or eliminating the potential for pulling an access device (i.e., catheter) back through an opening, such as a cardiac procedure. An introducer sheath includes a distal indented portion and a balloon, so that once placed in a desired location through tissue, the balloon can be inflated to anchor the sheath against retraction.

Compositions and Methods for Reducing Serum Triglycerides

This technology includes a vaccine for lowering plasma triglycerides by inducing the formation of autoantibodies against either ANGPTL3 or ANGPTL4, which are inhibitors of Lipoprotein Lipase. This was done by conjugating synthetic peptides based on ANGPTL3 or ANGPTL4 to virus- like particles (VLPS). Injection of the vaccine in animal models was shown to induce the autoantibody against the target and to lower plasma triglycerides.

Systems and Methods for Applying Pressure to the Heart for the Treatment of Tricuspid Valve Regurgitation

This technology includes structures and methods for cinching a band around the heart for treating conditions including tricuspid valve regurgitation (TR). When positioned appropriately along the atrioventricular groove, the band is tightened around the heart which narrows the tricuspid annulus and relieves TR.

Devices and Methods for Cerclage of Luminal Systems

This technology includes a family of transcatheter endovenous intramyocardial tether (MIRTH) procedures to impose myocardial constraint on the LV (MIRTH), LV and RV (SCIMITAR), and cardiac resynchronization procedures. Included is a set of advanced cardiac treatment technologies that focus on minimally invasive procedures for heart patients. The main technology is the transcatheter endovenous intramyocardial tether (MIRTH) procedure, which is designed to apply physical constraint to the left ventricle (LV) of the heart.

Expanded Claims for Transcatheter Coronary Sinus Mitral Valve Annuloplasty Procedure and Coronary Artery and Myocardial Protection

This technology includes a novel transcatheter repair for functional mitral valve regurgitation, called mitral cerclage annuloplasty. This includes coronary artery protection for mitral cerclage annuloplasty against inside-out compression from subsequent transcatheter valve-in-ring mitral valve implantation, wherein the ring is created by the cerclage annuloplasty. Cerclage annuloplasty is to create a semi-rigid ring at the level of the mitral annulus.

Segmented Metallic MRI Guidewires Using Stiffness-matched Nonconductive Connectors for Catheterization Procedures

This technology includes a metallic guidewire that is suitable for MRI catheterization, because it is mechanically long but electrically consists of short conductive segments that cannot resonate during MRI. The invention consists of stiffness-matched non-conductive connectors or connections that are used along with short metallic segments. The embodiment reduced to practice has torquability and flexibility comparable to marketed metallic guidewires, yet is free from MRI heating.

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