Antibodies that Selectively Detect the Human Nestin Protein
Cannula for Pressure Mediated Drug Delivery
Engineering Neural Stem Cells Using Homologous Recombination
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.
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.
A Device to Measure Force Continuously During Handgrip Contraction and Relaxation for Myotonic Dystrophies
Novel Codon-Optimized MUT Gene Therapeutic for Methylmalonic Acidemia (MMA)
Novel Methods for Generating Retinal Pigment Epithelium Cells from Induced Pluripotent Stem Cells
The retinal pigment epithelial cells (RPE) make up a polarized monolayer in the vertebrate eye that separates the neural retina from the choroid, and performs a crucial role in retinal physiology by forming a blood-retinal barrier and closely interacting with photoreceptors to maintain visual function. Many ophthalmic diseases, such as age-related macular degeneration, are associated with a degeneration or deterioration of the RPE.
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.