Directed Acetylation of Cytidine in Cellular mRNA through Engineered snoRNA Adapters for the Treatment of Haploinsufficiencies

Summary: 

The National Cancer Institute (NCI) seeks research co-development partners and/or licensees for engineered chimeric snoRNA guides that recruit NAT10 to a specific target and cause directed acetylation of the target. They could be used to treat haploinsufficiency-associated disorders or diseases.

Description of Technology: 

Topoisomerase III (TOP3) Inhibitors and Antiviral Compounds based on Cyanine Dyes

Topoisomerase 3B (TOP3B) is the only topoisomerase that can act on RNA as well as DNA, and thus has been a target of interest for the development of cancer therapies and RNA viral infection therapies. In the context of cancer, TOP3B is not an essential gene, but a subset of cancer cells with pre-existing genome instability are particularly vulnerable to the inactivation of TOP3B. While inhibitors for other topoisomerases are among the most potent and widely used anticancer agents, there are no known inhibitors of TOP3B.

Enhanced Immune Response With Stabilized Norovirus VLPs: A Next-Generation Vaccine Approach

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Vesicular Stomatitis virus (VSV)-based Vaccine against Sudan Virus

There are five known Ebolavirus species: Ebola virus (Zaire ebolavirus); Sudan virus (Sudan ebolavirus or SUDV); Taï Forest virus (Taï Forest ebolavirus, formerly Cote d'Ivoire ebolavirus); Bundibugyo virus (Bundibugyo ebolavirus); and Reston virus (Reston ebolavirus). Last year an ebolavirus outbreak resulted in 164 cases and 55 deaths. While there is an FDA-approved Ebola virus vaccine authorized for use against Ebola virus infections, ERVEBO, this vaccine is not effective against SUDV due to the significant variation between Ebola virus and SUDV.

Potentiating Antibody Therapy for the Treatment of Cancer

This technology includes a strategy to target tumor cells that lost antigen following reaction with a therapeutic antibody by targeting the complement component C3d that has been deposited on target cells by the primary antibody. We previously generated a C3d-specific mouse/human chimeric antibody called C8xi and obtained proof of principle for the approach in two preclinical models. Here we summarize the generation of a new set of C3d targeting antibodies.

Single Scan Bright-blood and Dark-blood Phase Sensitive Inversion Recovery (PSIR) Late Gadolinium Enhancement (LGE) for Cardiovascular Magnetic Resonance (CMR) Imaging

This technology includes a technique to improves detection of myocardial scar compared with conventional bright-blood late gadolinium enhancement (LGE) techniques. Dark-blood late gadolinium enhancement (DB-LGE) improves tissue delineation with signal suppression of the blood pool based on T2-preparation pulse that is relatively independent from the blood flow velocities and improves scar detection in patients with known or suspected coronary artery disease.

Vitamin C renal leak as a clinical diagnostic tool in the detection, monitoring, and management of acute and chronic diseases

This technology includes a clinical diagnostic tool for measuring vitamin C elimination by human kidneys that can be used for detecting, monitoring, and managing acute and chronic diseases. Findings revealed significant associations between vitamin C renal leak status and clinical variables affecting renal function and blood glucose. The technology uses vitamin C depletion-repletion kinetics and pharmacokinetic models to establish a physiological vitamin C renal threshold.

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.

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.

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