Main Menu

Cell transplantation therapy typically involves transplanting primary cells or immortalized cells into patients. The promising but still inconsistent data stemming from those clinical trials using primary cells in Parkinson's disease are believed to be due to an insufficient number, function and uniformity of the transplanted cells. In an effort to overcome these problems an improved method for isolating, growing and differentiating precursor cells into dopaminergic neurons has been developed.

Vitamin D receptor (VDR) is a nuclear receptor that is activated by calcitriol, the active form of vitamin D. It is best known for regulating dietary calcium uptake necessary for bone growth, but it also affects cell proliferation and differentiation. Therefore, it was thought that treatment with calcitriol or its derivatives could be useful to treat the uncontrolled proliferation typical of cancer cells. However, this approach has been unsuccessful to date because it leads to toxic levels of calcium in the blood.

Double-stranded RNA (dsRNA) has been shown to trigger sequence-specific gene silencing in a wide variety of organisms, including plant, nematode and invertebrate species. Recent intense work in the field has shown that small dsRNAs mediate sequence specific RNA degradation in the process known as RNA interference (RNAi).

This invention provides for synthetic dsRNAs (20-25 nucleotides in length) and methods that can inhibit gene-specific expression in mammalian cells.

A unique method of potentiating the effect of anti-cancer immunotoxins has been developed, thus offering to significantly improve the treatment of a number of cancers as well as autoimmune diseases. Prolonged treatment of human cancers with classical methods such as radiation and chemotherapy, or a combination of both, may cause greater damage than the underlying disease because healthy tissue is often damaged along with diseased tissue.

This work describes a novel nucleolar mechanism that controls the cell-cycle progression in CNS stem cells and cancer cells. The inventors identified a novel peptide, nucleostemin, found in the nucleoli of CNS stem cells, embryonic stem cells, and several cancer cell lines and preferentially expressed by other stem cell-enriched populations. When stem cells differentiate, nucleostemin expression decreases rapidly prior to cell-cycle exit both in vitro and in vivo. Depletion or overexpression of nucleostemin reduces cell proliferation in CNS stem cells and transformed cells.

This invention discloses and covers polyphenolic compounds that will bind bacterial toxins, methods for the treatment of such infections, specifically Stx-1 toxins from STEC strains of E. coli.

Bacterial infections not only cause disease by their presence but also upon the release of toxins. The common enteric bacteria, E. coli O157:H7 releases such toxins (Stx-1) upon treatment with antibiotics. These toxins, when released into the lumen of the intestinal tract, will cause cellular damage thus increasing the severity of the infection.

Available for licensing are NIH patent pending contrast particles for use in MRI and flow cytometry to track cells migration in real time. Present cell-tracking studies rely on labeling cells with ultra-small dextran-coated iron particles that are endocytosed. The contrast agent of the present invention uses larger iron oxide particles, approximately 1 µm, situated in a tri-layer structure.

Gaucher disease, the most common lysosomal storage disease, is an inherited metabolic disorder in which harmful quantities of the lipid glucocerebroside accumulate in the spleen, liver, lungs, bone marrow and in rare cases in the brain, due to a deficiency of the enzyme glucocerebrosidase (Gba) that catalyses the first step in the biodegradation of glucocerebrosides. Type 1 Gaucher disease is the most common and is distinguished from the other forms of the disease, types 2 and 3, by the lack of neurologic involvement.

Diabetes affects over 120 million people worldwide (16 million in the US) and is a major health problem with associated health costs estimated at almost $100 billion dollars. Type 2 diabetes affects as many as 10% of the population of the Western World (with 15 million patients in the US alone) and arises from a heterogeneous etiology, with secondary effects from environmental influences. Risk factors for type 2 diabetes include obesity, high blood pressure, high triglycerides and age.

This invention relates to the discovery that tristetraprolin (TTP) can promote the poly(A)RNase (PARN) mediated deadenylation of polyadenylated substrates containing AU-rich elements (AREs). As one aspect of the invention, the inventors have developed a cell free system that may be used for the purposes of assessing the effects of the various system components or their derivatives (i.e. AREs, PARN, or TTP) on the deadenylation process or the effects of various test agents on the deadenylation process.