Alpha-galactosidase-A Knockout Mouse Model for Studying Fabry Disease

This technology includes an alpha-galactosidase-A knockout mouse model that can be used to study Fabry disease, an X-linked lysosomal storage disorder. Alpha-galactosidase-A is a crucial enzyme responsible for the breakdown of glycolipids, particularly globotriaosylceramide (Gb3), within lysosomes. In Fabry disease, a rare and inherited lysosomal storage disorder, mutations in the GLA gene lead to deficient or non-functional alpha-galactosidase-A enzyme activity.

General-purpose Deep Learning Image Denoising Based on Magnetic Resonance Imaging Physics

This technology includes a novel method to train deep learning convolution neural network model to improve the signal-noise-ratio for the magnetic resonance (MR) imaging. The novelty lies on the fact that actual MR imaging physics information is used in the deep learning training. The resulting model achieves significant signal-to-noise ratio (SNR) improved for different acceleration factors in MR imaging. The resulting model can be used for many body anatomies (e.g., brain, heart, liver, spine, etc.) to significantly improve the SNR.

Method for HLA LOH Detection in Liquid Biopsies

Human leukocyte antigen (HLA) LOH (LOH) is a known resistance mechanism by which cancers evade T cell receptor-(TCR-)based immunotherapies. This class of therapies includes immune checkpoint inhibition (ICI, e.g., Pembrolizumab), engineered TCR (T cell receptor)-T cell adoptive transfer, tumor infiltrating lymphocytes (TIL), T-cell engagers, and other modalities. Dozens of therapies in this category were developed with many in clinical trials. The resistance mechanism noted here, HLA LOH, causes these therapies to fail.

Fluorinated MU-Opioid Receptor Agonists

Summary: 
Investigators at the National Institute on Drug Abuse seek co-development partners and/or licensees for collection of mu opioid receptor (MOR) agonists as alternatives for existing compounds.

Description of Technology: 
Although existing opioids are excellent analgesics and useful as positron emission tomography (PET) radiotracers, they come with debilitating side effects. These include addiction, respiratory distress, hyperalgesia, and constipation. Therefore, there is a need for alternatives with lower adverse effects.

Selective A3 Adenosine Receptor Agonists for the Treatment of Chronic Neuropathic Pain and Other Conditions

This technology includes the creation and use of A3 adenosine receptor (A3AR)-selective agonists for treating chemotherapy-induced peripheral neuropathy, chronic neuropathic pain, rheumatoid arthritis, psoriasis, and other conditions. A3 receptors for adenosine are found in most cells and endogenous activation of the A3 receptors can result in apoptosis, thereby relieving the inflammation or targeting a tumor. A3AR agonists have been a promising strategy for the treatment of various diseases.

Novel Small Molecule Agonists of the Relaxin Receptor as Potential Therapy for Heart Failure and Fibrosis

The present invention is directed to novel small molecule agonists of the mammalian relaxin family receptor 1 (RXFP1), including human RXFP1. Activation of RXFP1 induces: 1) vasodilation due to up-regulation of the endothelin system; 2) extracellular matrix remodeling; 3) moderation of inflammation by reducing levels of inflammatory cytokines; and 4) angiogenesis. Small molecule agonists of RXFP1 may be useful in treating acute heart failure (AHF), scleroderma, fibrosis, other conditions associated with the biology of relaxin, and in improving reproductive health and wound healing.

Zip14-AAV Genetic MRI Reporter System for Non-Invasive Cell & Gene-Therapy Tracking

This technology includes a gene-based magnetic resonance imaging (MRI) reporter platform that harnesses adeno-associated virus (AAV) delivery of the metal transporter Zip14 to create image contrast wherever the gene is expressed. By driving Zip14 from cell-specific promoters, investigators obtain robust, long-lasting signal changes on standard clinical MRI sequences (e.g., MPRAGE and GRE), enabling real-time visualization of living cells and their gene-expression patterns.

Gene Signature for Predicting Solid Tumors Patient Prognosis

HCC is the most frequent malignant tumor in the liver and the third leading cause of cancer death worldwide.  A progressive sequence of somatic mutations and epigenetic changes of oncogenes or tumor suppressor genes are believed to cause tumor development. However, high genomic instability in tumors causes the accumulation of genomic aberrations that do not contribute to tumor progression. Therefore, it is important to distinguish between ''driver'' mutations that are functionally important and ''passenger'' mutations that do not provide a selective advantage to the tumor cells.