Fibroblast Cell Lines (with L444P/RecNci1 Genotype) for the Screening of Small Molecules for Gaucher Disease Treatment

This technology includes two human fibroblast cell lines to be used to study the defects in GBA1 gene and protein and to screen small molecules for involvement in Gaucher disease. Glucocerebrosidase (GBA1 or GCase or beta-glucosidase) is a lysosomal enzyme, responsible for breakdown of a fatty material called glucocerebroside (or glucosyl ceramide). Deficiency or malfunction of GBA1 leads to the accumulation of insoluble glucocerebrosides in tissues, which is a major symptom of Gaucher disease.

Monoclonal Antibodies for the Recognition of Oncogene Fusions and Alveolar Rhabdomyosarcoma (ARMS) Diagnosis

This technology includes monoclonal antibody (mAb) that binds to the junction region of the PAX3-FOXO1 and PAX7-FOXO1 fusion protein for the diagnosis of Alveolar Rhabdomyosarcoma (ARMS). Specifically, two monoclonal antibodies (PFM.1 and PFM.2) have been isolated that recognize the 92kDa bands found uniquely to the pediatric striated muscle tumors of the type Alveolar Rhabdomyosarcoma (ARMS) carrying the characteristic t(2;13)(q35;q14) or t(1;13)(p36;q14) chromosomal translocations.

DNA Methylation Based Non-invasive Blood Diagnostic Assay for Precision Cancer Detection and Classification

This technology includes a panel of 46 genomic loci of DNA methylation (represented by CpG dinucleotides on different chromosomes) with application in blood-based cancer screening. The markers robustly distinguish tumor from normal samples using 8 loci and classify 13 different tumor types. Using 39 loci, inventors were able to discriminate between individual tumor types or peripheral blood. In 4052 tumor samples from 13 tumor types, the true positive rate of classification was 91.4%.

MLL3 (KMT2C), MLL4, PA1, UTX And PTIP Antibodies for the Treatment of Development Diseases and Cancers

This technology includes polyclonal antibodies against MLL3 (KMT2C), MLL4, PA1, UTX And PTIP for the development of treatments for development diseases and cancer. Enhancers play a central role in cell-type-specific gene expression and are marked by H3K4me1/2. Active enhancers are further marked by H3K27ac. However, the methyltransferases responsible for H3K4me1/2 on enhancers remain elusive. Furthermore, how these enzymes function on enhancers to regulate cell-type-specific gene expression is unclear.

A Novel Carbohydrate Antibody to GalNac1-3Gal and Its Application for Cancer Diagnostic and Prognosis

Cervical cancer is one of the most common cancers among women worldwide. Currently, physical descriptors such as tumor size and depth are the primary factors used for deciding the course of treatment. Despite significant efforts to identify prognostic biochemical markers or therapeutic targets to improve diagnosis and treatment, none have achieved routine clinical use. An example of one previously identified biomarker is the Tn antigen, a carbohydrate moiety composed of a GalNAc residue linked to serine or threonine.

Cancer Vaccines against POTE for Treating Solid Tumors

POTE is a novel tumor antigen expressed in a variety of cancers including breast, prostate, colon, lung, ovary, and pancreas cancers.  POTE has limited expression in normal tissues and therefore a specific target for cancer treatments, including immunotherapy.  The researchers seek statements of capability or interest from parties interested in collaborative research to further develop, evaluate, or commercialize immunogenic peptides. 

Mouse Lines with Fluorescently Labelled Membrane Proteins Regulating Cellular Motility and Membrane Trafficking

Cell motility and membrane trafficking play important roles in regulating cell division, cell migration, cell death and autophagy. Impairment of these processes can result in enhanced cell proliferation and survival and increased migration and invasion leading to cancer. Several proteins involved in cell motility and membrane trafficking have been shown to be dysregulated in various cancers. There is therefore a need for development of animal models for studying the roles of these proteins in cancer and their responses to drug treatment in vivo.

A Preclinical Model for Mutant Human EGFR-driven Lung Adenocarcinoma

Previously described epidermal growth factor receptor- (EGFR) driven tumor mouse models develop diffuse tumors, which are dissimilar to human lung tumor morphology and difficult to measure by CT and MRI scans. Scientists at the National Cancer Institute (NCI) have developed and characterized a genetically engineered mouse (GEM) model of human EGFR-driven tumor model (hEGFR-TL) that recapitulates the discrete lung tumor nodules similar to those found in human lung tumor morphology.