Galactose Kinase (GALK) Inhibitors for the Treatment of Galactosemia and Other Disorders of Galactose Metabolism

This technology includes selective inhibitors of the human enzyme galactokinase (EC 2.7.1.6), which may be useful for the treatment of Galactosemia and other diseases caused by aberrant galactose metabolism, including cancer. These compounds inhibit the first step in galactose metabolism, thereby eliminating the build-up of toxic metabolites during the aberrant metabolism of galactose, as well as inhibitor the entry of galactose into glycolysis and other downstream assays.

Identification and Use of Heterocyclic Alcohol Compounds for the Treatment of SULT1A1-expressing Cancers

This technology includes the identification and use of heterocyclic alcohol compounds, including RITA and N-BIC, for the treatment of SULT1A1-expression cancers. A high-throughput screen (qHTS) was performed using >1,000 caner cell lines identified a compound called YC-1 (also called Lificiguat) that is effective across cancer cell types that express the phase 2 detoxifying enzyme SULT1A1.

Attenuated Host-Range Restricted Dengue Viruses Derived by Site-Directed Mutagenesis of the Conserved 3-Stem and Loop Structure in Genomic RNA for Use as Vaccines

Although flaviviruses cause a great deal of human suffering and economic loss, there is a shortage of effective vaccines. The present invention is directed toward vector stage replication-defective flaviviruses that are replication-defective in mosquito vectors that transmit them to humans. The replication-defective flaviviruses of the present invention demonstrate a limited ability to replicate in the vector organisms that transmit flaviviruses from one host to another.

Activation of Recombinant Diphtheria Toxin Fusion Proteins by Specific Proteases Highly Expressed on the Surface of Tumor Cells

This invention relates to diphtheria toxin fusion proteins comprising a diphtheria toxin (DT) cell-killing component and a cell-binding component such as granulocyte macrophage colony-stimulating factor (GM-CSF), interleukin 2 (IL-2), or epidermal growth factor (EGF). Receptors for the latter three materials are present on many types of cancer cells; therefore, these fusion proteins bind preferentially to these cancer cells. A key feature is that these toxins are altered so as to require activation by a cell-surface protease that is overexpressed on many types of cancers.

Transgenic Mouse Models for Studying HLA-B57:01 and HLA-B15:02 Linked Immune Responses and Hypersensitivity Reactions

Transgenic mouse models expressing human HLA-B57:01 and HLA-B15:02 molecules have emerged as invaluable tools for unraveling the intricacies of immune responses and hypersensitivity reactions. The major histocompatibility complex (MHC) encoded proteins play a pivotal role in the immune system by presenting peptide fragments to T lymphocytes, and HLA-B57:01 has been associated with severe hypersensitivity reactions triggered by abacavir, a widely used anti-retroviral drug.

New Cholera Vaccine and Method for Conjugating Bacterial Polysaccharides to Proteins

A new conjugate vaccine for cholera has been developed. The invention includes a new method to conjugate the O-specific polysaccharide-core part of the bacterial lipopolysaccharide and protein subcomponents. Conventional technology has entailed chemical treatment of both components to introduce linkers, which made them amenable for covalent linking. The new method simplifies production by utilizing squaric acid chemistry for conjugating the free amine-containing species (e.g. polysaccharides) directly to amine-containing species (e.g.

Cancer Therapeutic Based on Hypoxia Inducible Factor 1 (HIF-1) Inhibitors

Hypoxia is a characteristic of many solid tumors resulting from accelerated cellular proliferation and inadequate vascularization. HIF-1 is a transcription factor critical for maintaining cellular homeostasis in, and adaptively responding to, low oxygen environments. HIF-1 becomes activated through binding to the transcriptional co-activator protein p300. Disruption of the HIF-1/p300 interaction could potentially modulate HIF-1 activity.

Photoactivatable Lipid-based Nanoparticles as a Vehicle for Dual Agent Delivery

The invention relates to novel lipid-based nanoparticles (liposomes) for use in targeted, on demand and on site drug delivery. The particles include a wall surrounding a cavity, wherein the wall is comprised of:

  1. A lipid bilayer comprising 1,2-bis(tricosa-10,12-diynoyl)-sn-glycero-3-phosphocholine (DC8,9PC), dipalmitoylphosphatidylcholine (DPPC), and 1,2-distearoyl-sn-glycero-3-

phosphoethanolamine-N-[methoxy(polyethylene glycol)-2000] (DSPE-PEG2000), and

Methods for Selection of Cancer Patients and Predicting Efficacy of Combination Therapy

Available for licensing from the Laboratory of Cancer Biology and Genetics of the National Cancer Institute (NCI) is a novel gene signature of thirty-seven drug-responsive genes that links changes in gene expression to the clinically desirable outcome of improved overall survival. Expression of these genes has been linked to prognosis in several cancers, including, but not limited to: multiple myeloma, melanoma, and lung and breast cancers.

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