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Synthetic Genes for the Treatment of Propionic Acidemia (PA) Caused by Mutations in Propionyl-coA Carboxylase Alpha (PCCA)

Propionic acidemia (PA) is an autosomal recessive metabolic disorder caused by mutations in either PCCA or PCCB. The products of these genes form the alpha and beta subunits of the enzyme propionyl-Co A carboxylase (PCC), a critically important mitochondrial enzyme involved in the catabolism of branched chain amino acids. NHGRI scientist have developed...

Published: 7/25/2024 | Inventor(s): Randy Chandler, Charles Venditti

Keywords(s): Acidemia, ALPHA, Carbox, CAUSED, GENES, Lase, Mutations, PA, Pcca, Propionic, PropionylcoA, Synthetic, treatment, VPXXXX, WIXXXX, XEXXXX

Category(s): Application > Therapeutics, TherapeuticArea > Infectious Disease, TherapeuticArea > Cardiology, TherapeuticArea > Oncology, TherapeuticArea > Ophthalmology, TherapeuticArea > Dental, TherapeuticArea > Endocrinology, Application > Research Materials

Novel Adeno-associated Viral (AAV) Vectors to Treat Hereditary Methylmalonic Acidemia (MMA) Caused by Methylmalonyl-coA Mutase (MMUT) Deficiency

Deficiency of the enzyme in methylmalonyl-CoA mutase (MMUT) results is a life-threatening disease, methylmalonic acidemia (MMA), that carries high rates of morbidity and mortality. NHGRI scientists have developed novel AAV vectors that combine the proprietary codon-optimized synMMUT alleles with either a liver-specific promoter from the human alpha-1...

Published: 7/25/2024 | Inventor(s): Randy Chandler, Charles Venditti

Keywords(s): AAV, Acidemia, Adeno-associated, CAUSED, DEFICIENCY, Hereditary, Methylmalonic, Methylmalonyl-CoA, MMA, Mmut, Mutase, Novel, TREAT, vectors, viral, VPXXXX, WIXXXX, WJXXXX, XEXXXX

Category(s): TherapeuticArea > Cardiology, Application > Therapeutics, TherapeuticArea > Infectious Disease, TherapeuticArea > Ophthalmology, TherapeuticArea > Dental, TherapeuticArea > Oncology, TherapeuticArea > Endocrinology, Application > Research Materials

Methods for Amelioration and Treatment of Pathogen-associated Inflammatory Response

This CDC invention provides methods for preventing or treating inflammatory response-linked, infection induced pathologies, which are mediated by endogenous substance P. Substance P is a naturally-occurring and major pro-inflammatory neuromediator or neuromodulator, and elevated levels of substance P have been implicated in numerous inflammation-associated...

Published: 7/25/2024 | Inventor(s): Larry Anderson, Deborah Moore, Ralph Tripp

Keywords(s): bronchoilitis, CAUSED, CDC Docket Import, CDC Docket Import CDC Prosecuting, DB3XXX, DB4XXX, DBXXXX, DISEASES, DXXXXX, IB3XXX, Infection, Inflammation, INFLAMMATORY, Methods, nitric oxide, OID-NCIRD-DVD, P, Pain, pain control, Prevention, Pulmonary, RESPONSE, RSV, RSV virus, SUBSTANCE, SUBSTANCE p, treatment, treatment methods, vasodilation, VASODILATOR, VAXXXX, VBXXXX, VDXXXX, VEXXXX, VJXXXX, VKXXXX, VLXXXX, VMXXXX, VOXXXX, VPXXXX, WAXXXX, WIXXXX, WJXXXX, WKXXXX, WMXXXX, XAXXXX, XCXXXX, YBXXXX, YCXXXX

Category(s): Collaboration Sought > Licensing, TherapeuticArea > Ophthalmology, TherapeuticArea > Infectious Disease, TherapeuticArea > Cardiology, TherapeuticArea > Oncology, Application > Consumer Products, Application > Therapeutics, TherapeuticArea > Dental, TherapeuticArea > Endocrinology, Application > Vaccines, ResearchProducts > Antibodies, TherapeuticArea > Immunology, TherapeuticArea > Neurology, Application > Research Materials, Application > Diagnostics, TherapeuticArea > Geriatrics

Selective 12-Human Lipoxygenase Inhibitors for the Treatment of Diabetes and Clotting

This invention discloses small molecule inhibitors of human 12-lipoxygenase (12-hLO). 12-lipoxygenase expression, activation, and lipid metabolites have been implicated in type 1 and type 2 diabetes, cardiovascular disease, hypertension, Alzheimer’s, and Parkinson’s disease. The development of 12-hLO inhibitors may be a potent intracellular approach...

Published: 7/25/2024 | Inventor(s): Jerry Nadler, Michael Holinstat, Anton Simeonov, Ajit Jadhav, Theodore Holman, David Maloney

Keywords(s): 12-human, Cardiovascular, CAUSED, Clot, DIABETES, Discovery, Disease, Formation, IA1AXX, IA1XXX, IAXXXX, Inhibitors, IXXXXX, LIPOXYGENASE, Platelet-mediated, Prevention, SELECTIVE, treatment

Category(s): Collaboration Sought > Licensing, Application > Research Materials, TherapeuticArea > Cardiology, Application > Therapeutics, Application > Diagnostics

Antigenic Chimeric Tick-Borne Encephalitis Virus/Dengue Virus Type 4 Recombinant Viruses

The tick-borne encephalitis virus (TBEV) complex is a group of viruses that can cause severe neutrotropic disease and up to thirty percent (30%) mortality. While these viruses can be found in many parts of the world, the largest impact of the disease occurs in Europe and Russia, where approximately fourteen thousand (14,000) hospitalized TBEV cases...

Published: 7/25/2024 | Inventor(s): Brian Murphy, Amber Engel, Alexander Pletnev

Keywords(s): 4, ANTIGENIC, CANDIDATES, CAUSED, chimeric, Chromosome 7, monosomy, DA4BXX, DA4XXX, DAXXXX, DC5BXX, DC5XXX, DCXXXX, DDXXXX, Deletion 7, Dengue (Flaviviridae), Development, Disease, DXXXXX, ENCEPHALITIS, Patent Category - Biotechnology, Prevention, recombinant, TBEV, TBEV/DEN4, TICK-BORNE, Tick-borne encephalitis, TYPE, Vaccine, virus, Virus/dengue, Viruses

Category(s): Collaboration Sought > Collaboration, Application > Research Materials, Application > Diagnostics, Application > Vaccines, TherapeuticArea > Infectious Disease