TRPC Knockout (KO) Mice and Mice with a Floxed Allele of TRPC Ion Channel Genes

TRPCs (Canonical Transient Receptor Potential Channels) are a group of non-selective cation channels that allow sodium and calcium into cells. There are seven different genes in mice that code TRPCs. The in vivo roles played by TRPCs as a whole are poorly understood and very little is known about the in vivo roles played by individual TRPCs nor the role of these channels in specific tissues or cells.

Metallic Nanoparticles for Photothermal Therapy

The invention relates to the preparation and application of 20-150nm metallic nanoparticulate vesicles for photothermal anti-cancer therapy. The vesicles comprise metallic nanoparticles covalently bound to a hydrophilic and hydrophobic polymer. The preparation method generally entails dispersing a polymer-bound metallic nanoparticle in an organic solvent, adding an aqueous solution with a dispersing aid, sonicating the mixture, and finally removing the organic solvent until the vesicle forms.

Gene Therapy for Niemann-Pick Disease Type C

Investigators at the National Human Genome Research Institute (NHGRI) of the National Institutes of Health (NIH) are seeking collaborators to further develop gene therapy to treat Niemann-Pick Disease Type C (NPC). NPC is a rare, autosomal recessive, neurodegenerative disease. Approximately 95% of patients with NPC have mutations in NPC1, a gene implicated in intracellular cholesterol trafficking. Mutations of NPC1 cause intracellular accumulation of unesterified cholesterol in late endosomal/lysosomal structures and marked accumulation of glycosphingolipids, especially in neuronal tissue.

Long Acting Therapeutic Conjugates with Evans Blue

This invention is a platform technology that pertains to the advantages of conjugating therapeutics to Evans Blue thus providing long lasting pharmacokinetic profiles by complexing with albumin. Notably, albumin bound therapeutic- or prodrug-Evans Blue conjugates provide a complex with a total molecular size above 60 kDa thus eliminating the risk for renal clearance. Interestingly, since albumin also crosses the blood-brain barrier and since all circulating Evans Blue is bound to albumin, Evans Blue bound therapeutics or prodrugs can also cross the blood-brain barrier.

Triazole Derivatives of 4,7-disubstituted 2 naphthoic acid (PPTN) as P2Y14 Receptor Antagonists

The Molecular Recognition Section of NIDDK announces the availability of a novel triazole-based probes, structures which act as antagonists at human P2Y14 receptors. Although the physiologic functions of this receptor remain undefined, recently it has been strongly implicated in immune and inflammatory responses. Prior work with a 4,7-disubstituted 2 naphthoic acid derivative (PPTN) established the ability to inhibit chemotaxis of human neutrophils in the lung and kidney.

Capsid-Free AAV Vectors for Gene Delivery and Their Use for Gene Therapy

The invention concerns novel capsid-free AAV vectors that can be used for gene delivery and gene therapy applications. The invention provides for a linear nucleic acid molecule comprising in this order: a first adeno-associated virus (AAV) inverted terminal repeat (ITR), a nucleotide sequence of interest, and a second AAV ITR, wherein said nucleic acid molecule is devoid of AAV capsid protein coding sequences. The said nucleic acid molecule can be applied to a host at repetition without eliciting an immune response.

Polyvalent Influenza Virus-Like Particles (VLPs) and Use as Vaccines

Influenza virus is a major public health concern, causing up to 500,000 deaths annually. The current strategy of reformulating vaccines annually against dominant circulating strains leads to variable protective efficacy and is unlikely to protect against novel influenza viruses with pandemic potential. Thus, there is a great need for a vaccine that provides “universal” protection against influenza viruses.

Enhanced Functionalization of Carbon Nanoparticles for Biomedical Applications

The invention pertains to methods of increasing the density of carboxylic acids on the surface of a carbon nanoparticle that can be functionalized with biologically relevant molecules, such as antibodies or peptides, for biomedical applications. Advantageously, the method could increase functionalization of a nanoparticle by at least about 1x107 functional groups/g of nanoparticle.

Non-invasive Pan-Cancer Detection Method

One of four deaths in the United States is due to cancer despite an emphasis on prevention, early detection, and treatment that has lowered cancer death rates by 20% in the past two decades. Further improvements in survival rates are likely to come from improving the limits of detection sensitivity at earlier stages of cancer. New approaches that rely heavily on genomic information, however, may change future testing strategies.

A Full-Length Infectious cDNA Clone of Zika Virus from the 2015 Epidemic in Brazil as a Genetic Platform for Studies of Virus-Host Interactions and Vaccine Development

An arthropod-borne virus, Zika virus (ZIKV), has recently emerged as a major human pathogen. Associated with complications during perinatal development and Guillain-Barré syndrome in adults, ZIKV raises new challenges for understanding the molecular determinants of flavivirus pathogenesis. This underscores the necessity for the development of a reverse genetic system based on an epidemic ZIKV strain. This technology relates to the generation and characterization in cell cultures of an infectious cDNA clone of ZIKV isolated from the 2015 epidemic in Brazil.