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National Institutes of Health researchers have developed knockout mice that do not express Tristetraprolin (TTP). TTP is an AU-rich element (ARE) binding protein and the prototype of a family of CCCH zinc finger proteins. AREs were identified as conserved sequences found in the 3’ untranslated region (3’ UTR) of a variety of transiently expressed genes including early response genes, proto-oncogenes, and other growth regulatory genes. AREs function as instability sequences that target ARE-containing transcripts for rapid mRNA decay.

Adeno-associated viruses (AAV) are used in gene delivery, but with limited success due to toxicity. The novel AAVs described in this technology may be more effective and useful in gene therapy applications.

The invention described and claimed in this patent application relates 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 Adeno-Associated Virus of serotype 4 (AAV4). The particular target cells identified are the ependymal cells of the brain. The methods described herein may be useful in carrying out gene therapy for diseases of the brain or central nervous system.

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 technology relates to the use of thymic stromal lymphopoietin (TSLP) to induce CD4+ T cell proliferation. This proliferation could be of particular relevance for patients in whom this cell population has been significantly reduced by HIV/AIDS or other conditions resulting in immunodeficiency. The proliferation of isolated CD4+ T cells can be induced through direct contact with TSLP or a nucleic acid encoding TSLP.

The invention relates to compositions of matter and methods for treating arthritis by modulating Tumor Necrosis Factor Alpha (TNF-alpha) signaling. TNF-alpha plays a key role in the pathogenesis of numerous diseases including rheumatoid and septic arthritis, and other autoimmune and inflammatory diseases. TNF-alpha mediates its effects through receptors that contain a Pre-ligand Assembly Domain (PLAD). The inventors have discovered compounds that interfere with PLAD can block the effects of TNF-alpha in vitro.

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.

The invention described and claimed in this patent application provides for novel vectors and viral particles which comprise adeno-associated virus serotype 5 (AAV5). AAV5 is a single-stranded DNA virus of either plus or minus polarity which, like other AAV serotypes (e.g., AAV4, AAV2) requires a helper virus for replication. AAV type 2 has the interesting and potentially useful ability to integrate into human chromosome 19 q 13.3-q ter. This activity is dependent on the non-structural, Rep, proteins of AAV2.