Novel Codon-Optimized Gene Therapeutic for Methylmalonic Acidemia

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.

High-Titer, Fast-Growth Chimeric Dengue/West Nile Viruses for Vaccine and Diagnostics Development

Mosquito-transmitted dengue virus is one of the leading causes of illness in the tropics and subtropics. There is currently no vaccine available and a number of DENV diagnostic and research applications depend on the production of large amounts of these viruses. However, due to the slow growing nature of DENVs these protocols are very time-consuming.

Heterocyclic Compounds for the Treatment of Hepatitis C Virus

The vast majority of people infected with Hepatitis C Virus (HCV) will have chronic infection. Over decades, this can lead to liver disease and liver cancer. In fact, HCV infection is the leading cause of liver transplants in the U.S. Several new drugs have recently come into the market that have changed the HCV treatment paradigm. However, the effectiveness of these new drugs can vary depending on the HCV genotype. Furthermore, all oral, interferon free therapeutic regimens for HCV infection will need combinations of drugs that target different aspects of the HCV life cycle.

Mobile Instrumentation for the Detection and Sampling of Aerosol Particles

Hazardous airborne particles pose a risk for health and safety in a variety of environments and thus detection of these small particles is essential. Current particle magnification systems are bulky and require a lot of power for operation, making them unsuitable to easily detect and analyze small particles in mobile and personal settings.

Microscopy System for Distinguishing Stimulated Emissions as a Means of Increasing Signal

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.

Octopod (8-Pointed Star) Iron Oxide Nanoparticles Enhance MRI T2 Contrast

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.

Cannabinoid Receptor Meditating Compounds for Metabolic Disease

There is evidence that the metabolic effects of endocannabinoids are mediated by CB1 receptors in peripheral tissues. While prior attempts at generating CB1 receptor blockers have had serious neuropsychiatric side effects, inventors at NIH have discovered compounds that block CB1 receptors with reduced brain penetrance. In addition, some of these compounds also have a direct inhibitory effect on inducible nitric oxide synthase (iNOS), whereas another group of the compounds directly activates AMP kinas.

LRRK2 Inhibitors: Novel Treatment for Intestinal Bowel Disorders

Use of Leucine Rich Repeat Kinase 2 (LRRK2) inhibitors for the treatment of Intestinal Bowel Disorders (IBD) is disclosed. IBD is a broad term that describes conditions with chronic or recurring immune response and inflammation of the gastrointestinal tract. Crohn's disease and ulcerative colitis, two common forms of idiopathic IBD, are chronic, relapsing inflammatory disorders of the gastrointestinal tract.

Highly Sensitive Tethered-Bead Immune Sandwich Assay

This technology is a highly sensitive tethered-bead immune sandwich assay. Analyte molecules are captured between two antibodies, a capture antibody and a detection antibody. The capture antibody on a micron-size bead binds analyte from a sample fluid. The bead-captured analyte is then exposed to a “detection” antibody that binds to the bead-captured analyte, forming a “sandwich”. The sandwiched analyte-bead complex then connects to a flexible polymer (such as DNA) anchored on a solid surface to form tethered particles.