CNS Therapeutics That Target Neuronal Ceroid-Lipofuscinoses and Thioesterase Deficiency Disorders

Clinically known as Neuronal Ceroid-Lipofuscinoses (NCL), Batten disease, is a rare neuron killing disease and one of the lysosomal storage disorders (LSDs).  It is associated with a mutation or lack of palmitoyl-protein thioesterase-1 (PPT1) gene. It manifests very early in a child's life causing absence of brain activity as early as 4 years of age.

Characterization and Comparison of LAD2 and LADR Mast Cell Lines: Insights into Mastocytosis and HIV Infection

LAD2 and LADR cell lines are invaluable tools in mast cell research, offering insights into mastocytosis and immune responses. Derived from CD34+ cells, LAD2 cells have been extensively used for over 18 years, while LADR cells, a newer variant, exhibit enhanced characteristics such as larger size, increased granulation, and faster doubling time. Both cell lines release granular contents upon FceRI aggregation and can be infected with various strains of HIV. LADR cells, in particular, show greater expression of certain surface receptors and mRNA compared to LAD2 cells.

Development of Mutations Useful for Attenuating Dengue Viruses and Chimeric Dengue Viruses

Although flaviviruses cause a great deal of human suffering and economic loss, there is a shortage of effective vaccines. This invention relates to dengue virus mutations that may contribute to the development of improved dengue vaccines. Site directed and random mutagenesis techniques were used to introduce mutations into the dengue virus genome and to assemble a collection of useful mutations for incorporation in recombinant live attenuated dengue virus vaccines.

Major Neutralization Site of Hepatitis E Virus and Use of this Neutralization Site in Methods of Vaccination

Hepatitis E is endemic in many countries throughout the developing world, in particular on the continents of Africa and Asia. The disease generally affects young adults and has a very high mortality rate, up to 20%, in pregnant women. This invention relates to the identification of a neutralization site of hepatitis E virus (HEV) and neutralizing antibodies that react with it. The neutralization site is located on a polypeptide from the ORF2 gene (capsid gene) of HEV.

TMC1, a Deafness-Related Gene

Hearing loss is a common communication disorder affecting nearly 1 in 1,000 children in the United States alone, and nearly 50% of adults by the age of eighty. Hearing loss can be caused by environmental and disease-related factors; however, hearing loss due to genetic factors accounts for approximately 50% of cases.

Variable Curve Catheter

The invention provides a deflectable tip guiding device, such as a catheter, that enables the operator to vary the radius of curvature of the tip of the catheter. This is a novel variation on the classic "fixed fulcrum" tip deflectors used in minimally invasive procedures in open surgical treatments. The described device permits a more comprehensive ability to navigate complex geometric pathways in patient's body and enables better access to target structures (e.g., to all endomyocardial walls from a transaortic approach).

Peptide Mimotope Candidates for Otitis Media Vaccine

This technology describes peptide mimotopes of lipooligosaccharides (LOS) from nontypeable Haemophilus influenzae (NTHi) and Moraxella catarrhalis that are suitable for developing novel vaccines against the respective pathogens, for which there are currently no licensed vaccines. The mimotopes not only immunologically mimic LOSs from NTHi and M. catarrhalis but will also bind to antibodies specific for the respective LOS. NTHi and M. catarrhalis are common pathogens that cause otitis media in children and lower respiratory tract infections in adults.

GATA-3 Reporter Plasmids for Revealing Underlying Mechanisms in Breast Cancer

GATA-3 is a transcription factor that is highly expressed in normal cells of the mammary luminal epithelium. GATA-3 plays a regulatory role in determining the fate of cells in the mammary gland. Disruption of GATA-3 expression leads to defects in the development of mammary cells, including an inability to differentiate properly into the correct cell type. GATA-3 function is also disrupted in various breast cancer models indicating that GATA-3 has tumor suppressive properties in normal cells.

Oxynitidine Derivatives as Tyrosyl DNA Phosphodiesterase (TDP) Inhibitors and Radiosensitizers

Summary: 

The National Cancer Institute (NCI) is actively seeking potential licensees and/or co-development research collaboration partners interested in further developing this family of oxynitidine derivatives as tyrosyl-DNA phosphodiesterase 1 (TDP1) inhibitors and radiosensitizers for the treatment of cancer. 

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