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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.

The invention describes a method for improving the spatial resolution of optical microscopes that use line-scanning excitation, such as line-scanning confocal microscopes, line-scanning STED microscopes, or line-scanning light-sheet microscopes.

The present invention is related to a recombinant viral vector for vaccines.

Currently available poxvirus vectors for humans and other animals exhibit suboptimal expression of recombinant gene(s) and high expression of vector proteins which causes weak immunogenicity and high anti-vector immune response.

The invention pertains to a system and method for distinguishing stimulated emissions as a means of enhancing signal strength of fluorescent markers in fluorescence microscopy applications. The system is arranged such that an excitation beam (e.g., laser beam) illuminates a sample along some axis exciting the fluorescent markers used in the sample. A second light beam, a stimulation beam, illuminates the sample along another axis, possibly the same as that of the excitation beam.

This technology is an ultra-sensitive colorimetric assay, based on an enzyme-catalyzed gold nanoparticle growth process, for detection of disease-associated proteins (biomarkers) and disease diagnosis. Current detection methods, such as ELISA immunoassays, measure concentrations above 0.1 ng/mL in a sample. PCR, although more sensitive than ELISA, requires expensive and specialized equipment and reagents, skilled labor, and complex analysis techniques. This assay detects fg/mL to pg/mL concentrations, allowing detection and diagnosis in the earliest stage of disease or infection.

The octopod-shaped iron oxide nanoparticles of this technology significantly enhance contrast in MRI imaging compared to spherical superparamagnetic iron oxide nanoparticle T₂ contrast agents. These octopod iron oxide nanoparticles show a transverse relaxivity that is over five times greater than comparable spherical agents. Because the unique octopod shape creates a greater effective radius than spherical agents, but maintains similar magnetization properties, the relaxation rate is improved. The improved relaxation rate greatly enhances the contrast of images.

Available for licensing are methods to generate T cells responsive to multiple polyomaviruses. The resulting T cell populations could be useful in treating immunosuppressed individuals with polyomavirus infections or polyomavirus-associated pathologies such as Merkel cell carcinoma (MCC), polyomavirus-associated nephropathy (PVAN), hemorrhagic cystitis, progressive multifocal leukoencephalopathy (PML), and trichodysplasia spinulosa (TS). The methods could also be used to restore polyomavirus-specific immunity in immunocompromised individuals.

Investigators at the National Heart, Lung, and Blood Institute have designed a novel lentiviral vector as a potential gene therapy for sickle cell anemia and beta-thalassemia. The novel lentiviral vector encodes the beta-globin gene in a forward orientation and can produce 5-10 fold higher viral titer and 4-10 fold higher gene transfer efficiency to hematopoietic stem cells than reverse-oriented lentiviral vectors. In vivo studies conducted in rhesus macaques show beta-globin production after transplantation with this novel lentiviral vector.

Available for licensing and commercial development as imaging agents for positron emission tomography of cancer are boramino acid compounds. The inventors showed that mimetics created by substituting the carboxylate group (-COO^-) of an amino acid with trifluoroborate (-BF_3^-) are metabolically stable and allow for the use of fluorene-18 (¹⁸F) as the radiolabel.

Available for licensing and commercial development is a three-dimensionally configured curved catheter for safe traversal of the right atrial appendage (RAA). The device is configured to optimize one-way access of the pericardial space through the right atrium and into the RAA reducing the risk of coronary lacerations. Specifically the curved catheter is best described in three segments: a proximal segment, a transitional segment and a distal segment; the transition segment having a clockwise spiral shaped curvature.