Novel Codon-Optimized MUT Gene Therapeutic for Methylmalonic Acidemia (MMA)

Methylmalonic Acidemia (MMA) is a metabolic disorder characterized by increased acidity in the blood and tissues due to toxic accumulation of protein and fat by-products resulting in seizures, strokes, and chronic kidney failure. A significant portion of MMA cases stem from a deficiency in a key mitochondrial enzyme, methylmalonyl-CoA mutase (MUT), required to break down amino acids and lipids. Currently, there are no treatments for MMA and the disease is managed primarily with dietary restriction of amino acid precursors and liver-kidney transplantation in severe cases.

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.

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.

Plasmid for the Study of Bam Complex and Screening of Therapeutic Molecules

This technology includes a plasmid (designated pJH114) that encodes all five subunits of the E. coli Bam (barrel assembly machine) complex under the control of an inducible promoter to be used in the study of the Bam and screen for therapeutic small molecules. The Bam (barrel assembly machine) complex is a highly conserved heterooligomer that catalyzes the integration of membrane proteins that have a beta barrel structure into the outer membrane of Gram-negative bacteria. Research suggests that this complex is essential for the viability of most, if not all bacteria in this class.

Human Monoclonal Antibodies That Recognize Influenza A Viruses for Vaccine, Therapeutic, and Diagnostic Development

Human influenza A is one of two influenza virus types that cause seasonal epidemics of disease (known as flu season) almost every winter in the United States. Influenza A viruses are the only influenza viruses known to cause flu pandemics (i.e., global epidemics of flu disease). (Source.)

Hybridomas Producing Antibodies to Neuraminidase for Influenza A (H3N2) Diagnostics, Vaccine, and Therapeutic Development

Influenza A and B viruses can cause seasonal flu epidemics ― commonly known as the “flu season” ― and infect the nose, throat, eyes, and lungs in humans. Typically, flu seasons that are dominated by influenza A (H3N2) virus activity have higher associated hospitalizations and deaths in at-risk groups, such as people ages 65 and older and young children. Influenza A (H3N2) virus can also cause respiratory disease in animals, such as canines and swine.

Method To Generate Chondrocytes from Human Induced Pluripotent Stem Cells (hIPSCs) and their use in Repairing Human Injury and Degenerative Diseases

This technology includes a method for differentiating human induced pluripotent stem cells (hiPSCs) into stable chondrocytes, capable of producing cartilage, and their use in cartilage repair in human injury and degenerative diseases. In suspension culture, hiPSC aggregates demonstrate gene and protein expression patterns similar to articular cartilage.

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