Engineering Neural Stem Cells Using Homologous Recombination

Methods for modifying the genome of a Neural Stem Cell (NSC) are disclosed. Also, methods for differentiating NSCs into neurons and glia are described. NSCs are multipotent, self-renewing cells found in the central nervous system, capable of differentiating into neurons and glia. NSCs can be generated efficiently from pluripotent stem cells (PSCs) and have the capacity to differentiate into any neuronal or glial cell type of the central nervous system.

Multidimensional MRI Signature for Specific Detection of Traumatic Brain Injury In Vivo

Traumatic brain injury (TBI) represents a major medical, social and economic concern worldwide due to significant mortality – especially among younger populations – and long-term disabilities. Various pathological brain lesions (e.g., intracerebral bleedings, necrotic-ischemic lesions, tissue avulsion) are produced by impacting mechanical forces. Among these, diffuse axonal injury (DAI) is one of the most significant brain lesions typically associated with trauma. However, DAI is not necessarily linked with TBI exposure. Therefore, the term “traumatic axonal injury (TAI)” is commonly used.

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.

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.

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.

A Device to Measure Force Continuously During Handgrip Contraction and Relaxation for Myotonic Dystrophies

This invention relates to two devices that reliably, sensitively, and accurately measures force during handgrip contraction and subsequent relaxation. A delayed relaxation after a sustained and forceful handgrip is a cardinal symptom of myotonic dystrophies (DM). This delayed relaxation, handgrip myotonia, may be a therapeutic response biomarker in clinical trials.

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.

High Relaxivity Mulitivalent Gadolinium on a Peptide Scaffold for Targeted MRI Applications in Disease Diagnosis

This technology includes a peptide containing alternating Alanine and Lys(DOTA-Gd) residues can be used to increase the MRI relaxivity of a peptide. The low molecular weight construct can be appended to proteins, antibodies and peptides to increase MRI signals. This approach offers advantages over previous dendrimeric constructs.

Astrocyte Differentiation of Neural Stem Cells with StemPro Embryonic Stem Cell Serum Free Medium for Research and Potential Therapeutic Use

This technology includes an innovative method for differentiating astrocytes from neural stem cells (NSCs). The process involves using Life Technologies StemPro embryonic stem cell serum-free medium to initially guide NSCs towards a neuronal lineage. Over a period of 28-35 days, as the cells are continually passaged, neurons gradually die off, leading to the proliferation of astrocytes. By the end of this differentiation protocol, approximately 70% of the cells exhibit markers characteristic of mature astrocytes, specifically GFAP.

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