Denoising of Dynamic Magnetic Resonance Spectroscopic Imaging Using Low Rank Approximations in the Kinetic Domain

Accurate measurement of low metabolite concentrations produced by medically important enzymes is commonly obscured by noise during magnetic resonance imaging (MRI). Measuring the turnover rate of low-level metabolites can directly quantify the activity of enzymes of interest, including possible drug targets in cancer and other diseases. Noise can cause the in vivo signal to fall below the limit of detection. A variety of denoising methods have been proposed to enhance spectroscopic peaks, but still fall short for the detection of low-intensity signals.

Establishment of Induced Pluripotent Stem Cells (iPSC) from the Thirteen-lined Ground Squirrel

The limited choice in cell types available for in vitro studies has become an obstacle in hibernation research. 

Researchers at the National Eye Institute for the first time have successfully established iPSC line(s) from a mammalian hibernator, which can be potentially used to generate various cell types and tissue models for in-depth mechanistic studies of hibernation and coldness tolerance in vitro. 

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.

Oral Iron-Chelator Therapy for Treating Developmental Stuttering

This technology discloses the use of small-molecule iron chelators—drugs that bind and remove excess iron—for the oral treatment of developmental stuttering in children and adults. Mouse models carrying human stuttering mutations show both elevated striatal iron and impaired vocalization; daily low-dose deferiprone reverses these speech-like deficits while normalizing brain-iron MRI signals.

Angubindin-1 Peptide for Transient Blood-Brain Barrier Opening to Boost Chemotherapy in Malignant Glioma

This technology includes a first-in-class synthetic peptide, angubindin-1, designed to temporarily relax the blood-brain barrier (BBB)—the tightly sealed network of brain blood vessel cells that normally blocks most drugs—from the inside. By binding the tricellular tight-junction protein angulin-1/LSR, the peptide creates a reversible “molecular doorway” that lets cancer medicines such as liposomal doxorubicin (Doxil®) reach tumors in the central nervous system (CNS).

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.

Zinbryta® for Treatment of Relapsing Multiple Sclerosis

The 2017 Deals of Distinction™ Award was presented to National Institutes of Health, (NIH) along with its corporate partners, AbbVie and Biogen, for a license agreement related to the development and launch of Zinbryta® for treatment of relapsing multiple sclerosis (MS)   The award, one of the most prestigious for technology transfer, was given to NIH and its partners at the Licensing Executives Society Annual Meeting in Chicago, Illinois.