Clinically known as Neuronal Ceroid-Lipofuscinoses (NCL), Batten disease, is a rare neuron killing disease and one of the lysosomal storage disorders (LSDs). It is associated with a mutation or lack of palmitoyl-protein thioesterase-1 (PPT1) gene. It manifests very early in a child's life causing absence of brain activity as early as 4 years of age.
Researchers at the Vaccine Research Center (“VRC”) of the National Institute of Allergy and Infectious Diseases (“NAID”) continue to pursue a safe and effective HIV-1 vaccine to combat the HIV-1/AIDS pandemic.
This technology includes a vaccine for lowering plasma triglycerides by inducing the formation of autoantibodies against either ANGPTL3 or ANGPTL4, which are inhibitors of Lipoprotein Lipase. This was done by conjugating synthetic peptides based on ANGPTL3 or ANGPTL4 to virus- like particles (VLPS). Injection of the vaccine in animal models was shown to induce the autoantibody against the target and to lower plasma triglycerides.
This technology includes 1-13C-ketoglutarate which can be used for imaging the conversion to hydroxyglutarate (HG) or Gln in cancer cells with an IDH1 mutations by hyperpolarized MRI. The ability to detect the status of IDH1 mutations is clinically prognostic for multiple cancers. These exciting observations are limited by two factors, the major one being that the natural abundance of 13C at position C5 overlaps with 1-13C-2-hydroxyglutarate peak, which limits the sensitivity of analysis and prevents simultaneous observations of HG and Gln formation.
This technology includes structures and methods for cinching a band around the heart for treating conditions including tricuspid valve regurgitation (TR). When positioned appropriately along the atrioventricular groove, the band is tightened around the heart which narrows the tricuspid annulus and relieves TR.
This technology includes a new method to prepare very long chain fatty acids (VLCFA), which does not use the previously reported toxic mercury amalgam, for use as nutritional supplements, and as therapeutics for various diseases. The key coupling step involves an organocopper mediated coupling of the Grignard regent derived from the bromo alkyl tetraene with a bromoalkyl containing a protected alcohol. After the coupling the alcohol Is deprotected and oxidized to prepare the very long fatty acid. The synthetic approach is flexible and can be used to prepare the other VLCFA compounds.
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.
This technology includes efficient lentiviral gene delivery system for both guide RNA and Cas9 endonuclease as a method to cure sickle cell disease. Gene correction is an ideal gene therapy strategy for hereditary disease, including sickle cell disease.
This technology includes a novel transcatheter repair for functional mitral valve regurgitation, called mitral cerclage annuloplasty. This includes coronary artery protection for mitral cerclage annuloplasty against inside-out compression from subsequent transcatheter valve-in-ring mitral valve implantation, wherein the ring is created by the cerclage annuloplasty. Cerclage annuloplasty is to create a semi-rigid ring at the level of the mitral annulus.
This technology includes a metallic guidewire that is suitable for MRI catheterization, because it is mechanically long but electrically consists of short conductive segments that cannot resonate during MRI. The invention consists of stiffness-matched non-conductive connectors or connections that are used along with short metallic segments. The embodiment reduced to practice has torquability and flexibility comparable to marketed metallic guidewires, yet is free from MRI heating.