Treating Kidney Disorders and Diabetic Nephropathy with N-acetyl mannosamine (ManNAc)

N-acetylmannosamine (ManNAc) is a small uncharged physiological molecule that crosses membranes readily and is the natural precursor of intracellular sialic acid synthesis. NHGRI investigators discovered that ManNAc can be used for therapeutic purposes, including treating certain kidney diseases (e.g., those involving proteinuria and hematuria), resulting primarily or secondarily from hyposialylation (lack of sialic acid). Notably, ManNAc can also be used to treat diabetic nephropathy or diabetes.

Treatment of Periodontal Disease via ENPPI Inhibition

This technology focuses on enhancing cementum production, a key component in treating periodontal regression. The method involves inhibiting ectonucleotide pyrophosphatase phosphodiesterases (ENPP1), enzymes that play a significant role in mineralization processes. Pyrophosphate (PPi) is known to impede the growth of hydroxyapatite crystals, essential for mineralization. ENPP1 catalyzes the hydrolysis of ATP, generating PPi, which then hinders mineralization.

Deuterated alpha5 Subunit-selective Negative Allosteric Modulators of Gamma-Aminobutyric Acid Type A Receptors as Fast Acting Treatments for Depression and Mood Disorders

This technology includes GABAa a5 Negative Allosteric modulators (GABAa a5 NAMs) which have been recently discovered to act as fast-acting antidepressants in a variety of mouse models of depression. These NAMs are actively metabolized in vivo. This invention involves the conceptualization and synthesis of GABAa a5 NAM molecules with a deuterium in the active metabolic position. This significantly increased the metabolic stability, while still retaining the antidepressant activity.

Java Applet for Modeling Human Metabolism and Energy Expenditure for Adaptive Dieting and Exercise Regimens

Known methods for predicting weight loss fail to account for slowing of metabolism as weight is lost and therefore overestimate the degree of weight loss. While this limitation of the 3500 Calorie per pound rule has been known for some time, it was not clear how to dynamically account for the metabolic slowing. The invention provides a Java applet for modeling of human metabolism to improve the weight change predictions. The model has been validated using previously published human data and the model equations have been published.

Monoclonal Antibodies to HIV-1 Vpr

Available for licensing are monoclonal antibodies against HIV-1 viral protein R (Vpr) and the respective hybridoma cell lines expressing the same. The antibodies provide a means for detecting HIV-1 Vpr. Currently, the mechanism of HIV pathogenesis believed to involve viral replication inside immune cells and other cells. At present, there are no clinical assays for detecting HIV-1 Vpr. Vpr circulates at detectable levels in the blood and is likely derived from degraded virions or released from infected cells. Vpr facilitates viral replication and disrupt normal cell function.

Conditionally Immortalized Human Podocyte Cell Lines

Podocytes, cells of the visceral epithelium in the kidneys, are a key component of the glomerular filtration barrier. Podocyte damage and loss contribute to the initiation of glomerular diseases. NIH investigators recently established long-term urinary cell cultures from two patients with focal segmental glomerulosclerosis and two healthy volunteers, via transformation with the thermosensitive SV40 large T antigen (U19tsA58) together with human telomerase (hTERT).

Construct for Tetracycline Inducible Podocyte Specific Gene Expression in Mice

The National Institutes of Health announces the generation of a construct by ligating 2.5kb human podocin promoter sequence to gene encoding reverse tetracycline-controlled transcriptional activator which enables tetracycline-inducible podocyte specific gene of interest expression with another construct consisting of tetracycline responsive element, minimal CMV promoter and gene of interest.

Truncated Methanocarba Adenosine Derivatives as A3 Adenosine Receptor Antagonists

Novel A3 adenosine antagonists available for licensing. A3 receptors are particularly highly expressed in inflammatory cells, making it a potentially desirable target for inflammatory diseases. This technology relates to highly specific antagonists and partial agonists of A3 adenosine receptors, which are negatively coupled to adenylate cyclase and have been broadly implicated in inflammation, cardiovascular disease, endocrine conditions and cancer. Further, A3 adenosine receptors have been implicated in asthma and glaucoma.

Glucocerebrosidase Activators as a Treatment for Gaucher Disease

This technology is a collection of small molecule activators of a genetically defective version of the enzyme called glucocerebrosidase (GCase), which causes Gaucher disease. Gaucher disease is a rare disease affecting 1 in 40,000 babies born. Ashkenazi Jews of eastern European descent (about 1 in 800 live births) are at particular risk of carrying this genetic defect. It is caused by inherited genetic mutations in the gene that encodes GCase, which result in reduced activity of the enzyme.