Use of Antisense Oligodeoxynucleotides for Inhibiting JC Virus

Progressive multifocal leukoencephalopathy (PML) is a rare, fatal demyelinating disease of the brain caused by the polyomavirus JC (JCV) under immunosuppressive conditions. It is pathologically characterized by progressive damage of white matter of the brain by destroying oligodendrocytes at multiple locations. Clinically, PML symptoms include weakness or paralysis, vision loss, impaired speech, and cognitive deterioration. The prognosis of PML is generally poor. No effective therapy for PML has been established.

Device for Vascular Dilation

The invention is an enhanced vascular dilator that eliminates the vascular injury caused by the size mismatch between vascular introducer sheaths and vascular dilators, as the two are advanced into a blood vessel. The invention provides a “shoulder” to match the diameter of the introducer sheath so that there is a smooth transition, without size mismatch, between the dilator and the introducer sheath. The invention allows the dilator to be withdrawn in segments from the introducer sheath.

Mouse Model for Methylmalonic Acidemia, an Inherited Metabolic Disorder

Methylmalonic Acidemia (MMA) is a metabolic disorder affecting 1 in 25,000 to 48,000 individuals globally. MMA is 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. About 60% of MMA cases stem from mutations in the methylmalonyl CoA mutase (MUT) gene encoding a key enzyme required to break down amino acids and lipids. Previous efforts to develop mice with null mutations in MUT have been unsuccessful, as such mutations result in neonatal death.

Deconvolution Software for Modern Fluorescence Microscopy

This software invention pertains to Joint Richardson-Lucy (RL) deconvolution methods used to combine multiple images of an object into a single image for improving resolution in modern fluorescence microscopy. RL deconvolution merges images with very different point spread functions, such as in multi-view light-sheet microscopes, while preserving the best resolution information present in each image.

Human iPSC-Derived Mesodermal Precursor Cells and Differentiated Cells

Cells, cell culture methods, and cell culture media compositions useful for producing and maintaining iPSC-derived cell lines that are of higher purity and maintain cell type integrity better than current iPSC-derived cell lines are disclosed. Human induced pluripotent stem cells (hiPSCs) can be generated by reprogramming somatic cells by the expression of four transcription factors. The hiPSCs exhibit similar properties to human embryonic stem cells, including the ability to self-renew and differentiate into all three embryonic germ layers: ectoderm, endoderm, or mesoderm.

Real Time Medical Image Processing Using Cloud Computing

The invention pertains to a system for reconstructing images acquired from MR and CT scanners in a robust Gadgetron based cloud computing system. A hardware interface connects clinical imaging instruments (e.g., MR or CT scanners) with a cloud computing environment that includes image data reconstruction and processing software not limited by the computational constraints typical of static hardware with finite processor power.

Novel Dopamine D2 Receptor Antagonists and Methods of Their Use

Investigators at the NIH have identified a series of novel, small molecule antagonists of the dopamine D2 receptor. Among the dopamine receptor (DAR) subtypes, D2 DAR is arguably one of the most validated drug targets in neurology and psychiatry. For instance, all receptor-based anti-Parkinsonian drugs work via stimulating the D2 DAR, whereas all FDA approved antipsychotic agents are antagonists of this receptor. Unfortunately, most agents that act as antagonists of D2 DAR are problematic, either they are less efficacious than desired or cause multiple adverse effects.

AMA1-RON2 Complex-Based Vaccine Against Malaria

This technology relates to a malaria vaccine composed of a protein complex of Apical Membrane Antigen (AMA1) and rhoptry neck protein 2 (RON2) with an adjuvant. AMA1 is a crucial component of the Plasmodium invasion machinery and is a leading candidate for antimalarial vaccine development. AMA1-based vaccines have shown ability to block red cell invasion in in vitro assays, but protection has so far not translated to in vivo human infections.

Treatment of Chronic Kidney Disease with Synthetic Amphipathic Peptides

The invention is directed to treatment of chronic kidney disease by administering a synthetic, amphipathic helical peptide known as 5A-37pA, and novel derivatives thereof. Scientists at NIDDK have demonstrated that invention peptides antagonize activity of a particular scavenger receptor known as CD36. Using an in vivo model, NIDDK scientists have shown that invention peptides slowed progression of chronic kidney disease and can potentially be utilized as a therapeutic treatment.