Diagnostics and Therapeutics for Hydrocephalus

Congenital hydrocephalus is a significant public health problem, affecting approximately one in 500 live births in the United States. Congenital hydrocephalus has an adverse effect on developing brain and may persist as neurological defects in children and adults. Some of these defects may manifest as mental retardation, cerebral palsy, epilepsy and visual disabilities. Improved diagnostics are needed for assessing the risks of developing this debilitating disease.

Oral Treatment of Hemophilia

This invention portrays a simple method for treatment of antigen-deficiency diseases by orally administering to a subject a therapeutically effective amount of the deficient antigen, wherein the antigen is not present in a liposome. This method increases hemostasis in a subject having hemophilia A or B, by orally administering to the hemophiliac a therapeutically effective amount of the appropriate clotting factor, sufficient to induce oral tolerance and supply exogenous clotting factor to the subject.

Recombinant Plasmids for Soluble Immunoreceptors

Immunoreceptors initiate signals leading to the activation of immune system against invasion pathogens. A number of soluble receptors, representing the extracellular ligand binding domains of the immunoreceptors, have been expressed using a recombinant bacteria expression and reconstitution system. This set of 21 plasmids, which can be used as immunological research reagents or to develop diagnostic tools, comprise the following:

Peptides for Treatment of Tumor Necrosis Factor alpha Mediated Inflammatory Disease

Tumor Necrosis Factor alpha (TNF-alpha) is a multifunctional cytokine that mediates inflammation, immune regulation, and cellular proliferation. This cytokine is converted to its active form by TNF-alpha converting enzyme (TACE). Pathological increases in TNF-alpha activity have been associated with a wide variety of inflammatory diseases, including inflammatory bowel disease, rheumatoid arthritis, and cancer. Inhibiting the conversion of TNF-alpha to its active form by inhibiting TACE represents a potential treatment for these diseases.

A Tet-Regulated Mouse Model for Cataract

Cataract is the most common cause of blindness worldwide, with an estimated 25 million blind and 119 million visually impaired individuals worldwide. Over 20 million adults in the US alone are currently diagnosed with cataracts making this disease a major health concern. The incidence of cataract increases with age and a number of etiologic factors have been proposed in the pathogenesis of age-related cataract in humans including genetic factors, environmental factors and metabolic and biochemical changes in the crystalline lens.

Codon-optimization of HIV-1 Viral Infectivity Factor (VIF) Gene

Expression of the HIV-1 Vif protein in the absence of other viral factors such a Tat and Rev is extremely inefficient due to the presence of inhibitory sequences on its mRNA. This invention uses codon optimization to remove such inhibitory sequences without altering the amino acid sequence of the protein. The modified vif gene in the resulting pcDNA -hVIF vector is expressed under the control of the CMV promoter. In this, the protein functions as wild type and is more amendable to high-level expression in mammalian cells.

Cloning and Characterization of an Avian Adeno-Associated Virus and Uses Thereof

Currently, adeno-associated virus (AAV) represents the gene therapy vehicle of choice because it has many advantages over current strategies for therapeutic gene insertion. AAV is less pathogenic than other virus types; stably integrates into dividing and non-dividing cells; integrates at a consistent site in the host genome; and shows good specificity towards various cell types for targeted gene delivery.

Brother of the Regulator of Imprinted Sites (BORIS)

The subject application discloses an isolated or purified nucleic acid molecule consisting essentially of a nucleotide sequence encoding a human or a non-human BORIS, or a fragment of either of the foregoing; an isolated or purified nucleic acid molecule consisting essentially of a nucleotide sequence that is complementary to a nucleotide sequence encoding a human or a non-human BORIS, or a fragment of either of the following; a vector comprising such an isolated or purified polypeptide molecule consisting essentially of an amino acid sequence encoding a human or a non-human BORIS, or a fragme

Method for the Treatment of Multiple Sclerosis

The invention relates to the discovery that humanized antibodies to the interleukin-2 receptor (IL-2R) such as (daclizumab) are effective in treating multiple sclerosis (MS). In particular, it has been discovered that patients who have failed to respond to therapy with interferon-beta show dramatic improvement when treated with daclizumab, with patients showing both a reduction in the total number of lesions and cessation of appearance of new lesions during the treatment period. Daclizumab is effective both in combination with interferon-beta and alone.

Multipotent Postnatal Stem Cells From Human Periodontal Ligament and Uses Thereof

It is estimated that over 40 percent of the adult population in the United States has periodontal disease in one form or another. Periodontal Disease is a chronic infection of the periodontal ligament (PDL) and the adjacent bone and cementum. The effects of Periodontal Disease range from simple gum inflammation to, in extreme cases, tooth loss.