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The disclosed invention provides materials and methods to treat granulocytopenia (low white cell count in the blood) which is characterized by a reduced number of granulocytes (relative) or an absence of granulocytes (absolute). This condition is commonly associated with cancer chemotherapy, but is seen less frequently in a number of conditions including the use of propylthiouracil, radiotherapy for marrow ablation for bone marrow transplantation, aplastic anemia, systemic lupus erythematosus, AIDS and a variety of other situations.

The invention described and claimed in this patent application is related to the delivery of heterologous nucleic acids or genes to particular target cells. In particular, the application relates to methods of delivering a heterologous nucleic acid or gene of interest to particular target cells using an Adeno-Associated Virus of serotype 5 (AAV5). The particular target cells identified include the alveolar cells of the lung and cerebellar and ependymal cells of the brain.

This invention describes methods and compositions of peptides that inhibit the binding of Plasmodium falciparum (P. falciparum) to erythrocytes. Malarial parasites enter the red blood cell through several erythrocyte receptors, each being specific for a given species of Plasmodia. For P. falciparum, the erythrocyte binding antigen (EBA-175) is the ligand of the plasmodia merozoites that interacts with the receptor glycophorin A on the surface of red blood cells.

This invention relates to materials and methods associated with polymorphic variants in two enzymes involved in folate-dependent and one-carbon metabolic pathways important in pregnancy-related complications and neural tube birth defects: MTHFD1 (5,10-methylenetrahydrofolate dehydrogenase, 5,10-methenyltetrahydrofolate cyclohydrolase, 10-formyltetrahydrofolate synthase) and methylenetetrahydrofolate dehydrogenase (NADP+ dependent) 1-like (MTHFD1L). These enzymes are extremely important in the promotion of DNA synthesis, a process that is critical for normal placental and fetal development.

This application describes a composition and method for treating inflammatory bowel disease or other autoimmune diseases. The composition utilizes a vector which contains a first promoter which controls the expression of a regulatory transcription factor and a second inducible promoter which controls the expression of the gene of interest. The preferred gene of interest encodes an isoform of TGF-beta such as TGF-beta₁ or TGF-beta₃. The isoform of TGF-beta does not have to be hTGF-beta and can be a latent or active isoform of TGF-beta.

The invention provides a method of diagnosing X-linked severe combined immunodeficiency (XSCID) in males or determining whether females are carriers. This method is based upon the presence of either a mutated or truncated IL-2Rgamma gene. The invention also discloses a method of treating XSCID as well as a method for monitoring the therapy.

Transformation-Associated Recombination (TAR) cloning in yeast is a unique method for selective isolation of large chromosomal fragments or entire genes from complex genomes without the time-consuming step of library construction.¹ The technique involves homologous recombination during yeast spheroplast transformation between genomic DNA and a TAR vector that has short (approximately 60bp) 5’ and 3’ gene targeting sequences (hooks).

A primary goal of diabetes therapy is to improve control of blood glucose levels (known as glycemic control) in patients. Prospective studies of both Type 1 and Type 2 diabetes indicate that careful glycemic control significantly reduces the risk of microvascular, neurological, and cardiovascular complications of diabetes. The current method of monitoring glycemic control involves measuring levels of the intracellular hemoglobin (HbA1C).

Approximately 30,000 patients are diagnosed with renal cell carcinoma (RCC) each year in the United States, and an estimated 12,000 patients die of this disease. Most patients are diagnosed with advanced local disease or metastatic disease. Metastatic RCC carries a poor prognosis with median survivals in the range of 10-12 months. Drugs that inhibit VEGF receptor tyrosine kinases such as Sorafenib and Sunitinib have recently been approved by the FDA to treat metastatic RCC.

Two chimpanzee mAbs specifically reacted with light chain of the botulinum neurotoxin A and neutralize the toxin in the mouse model. They can be used for emergency prophylaxis and treatment of either naturally acquired or terrorist associated botulism. Since the sequence of chimpanzee immune globulin is virtually identical to that of humans, the MAbs are not expected to have problems in repeated administration as equine antibodies. They can also be used for rapid diagnosis of botulinum neurotoxin A.