T Cell-Based Adoptive Transfer Immunotherapy for Polyomavirus-Associated Pathologies

Available for licensing are methods to generate T cells responsive to multiple polyomaviruses. The resulting T cell populations could be useful in treating immunosuppressed individuals with polyomavirus infections or polyomavirus-associated pathologies such as Merkel cell carcinoma (MCC), polyomavirus-associated nephropathy (PVAN), hemorrhagic cystitis, progressive multifocal leukoencephalopathy (PML), and trichodysplasia spinulosa (TS). The methods could also be used to restore polyomavirus-specific immunity in immunocompromised individuals.

A Novel Therapeutic Vector for Hemoglobin Disorders

Investigators at the National Heart, Lung, and Blood Institute have designed a novel lentiviral vector as a potential gene therapy for sickle cell anemia and beta-thalassemia. The novel lentiviral vector encodes the beta-globin gene in a forward orientation and can produce 5-10 fold higher viral titer and 4-10 fold higher gene transfer efficiency to hematopoietic stem cells than reverse-oriented lentiviral vectors. In vivo studies conducted in rhesus macaques show beta-globin production after transplantation with this novel lentiviral vector.

Three-Dimensional Curved Catheter for Right Atrial Appendage Traversal

Available for licensing and commercial development is a three-dimensionally configured curved catheter for safe traversal of the right atrial appendage (RAA). The device is configured to optimize one-way access of the pericardial space through the right atrium and into the RAA reducing the risk of coronary lacerations. Specifically the curved catheter is best described in three segments: a proximal segment, a transitional segment and a distal segment; the transition segment having a clockwise spiral shaped curvature.

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.

Remotely Monitored Mouse Feeding Experimentation Device

How much does a mouse eat per day? If a researcher is conducting dietary studies, the answer is very important. For instance, obesity studies require accurate measures of feeding. Existing automated methods for taking feeding measurements are expensive and use specialized caging that is not compatible with typical vivarium colony racks. As a result, many researchers simply weigh food each day or two to determine how much food the mice ate. This is time-consuming, can be error prone, and provides a low temporal resolution view of feeding.

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.

Transgenic Mice Expressing CNO-sensitive Gq- or Gs-coupled Designer Receptors Selectively in Pancreatic Beta Cells

Impaired functioning of pancreatic beta cells is a key hallmark of type 2 diabetes. Beta cell function is modulated by the actions of different classes of heterotrimeric G proteins. The functional consequences of activating specific beta cell G protein signaling pathways in vivo are not well understood at present, primarily due to the fact that beta cell G protein-coupled receptors (GPCRs) are also expressed by many other tissues.

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.