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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 Codon-Optimized Gene Therapeutic for Methylmalonic Acidemia

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),...

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

Keywords(s): Acidemia, Class, GB2AXX, GBXXXX, GXXXXX, Methylmalonic, Methylmalonyl-CoA, MMA, MUT, Mutase, Synthetic, TRANSGENE, treatment

Category(s): Collaboration Sought > Collaboration, Application > Therapeutics

Salen-Manganese Compounds for Therapy of Viral Infections

Salen-manganese compounds are synthetic, stable, low toxicity, low cost agents that may provide protection from immune reaction-related oxidative cell damage associated with many illnesses. In particular, oxidative cell damage has been associated with many viral infections including influenza. This invention demonstrates that treating mice with salen-manganese...

Published: 7/25/2024 | Inventor(s): Jeffery Taubenberger, Rodney Levine, Susan Doctrow, John Kash

Keywords(s): Catalase/Superoxide, DB4BXX, DB4XXX, DBXXXX, DISMUTASE, DXXXXX, INFECTIONS, MIMETICS, Synthetic, viral

Category(s): Collaboration Sought > Collaboration, Application > Therapeutics, TherapeuticArea > Infectious Disease

Vaccines Against Malarial Diseases

The invention offered for licensing is in the field of use of vaccines for malaria. The invention provides gene sequences encoding an erythrocyte binding protein of a malaria pathogen for the expression of the erythrocyte binding protein. The codon composition of the synthetic gene sequences approximates the mammalian codon composition. The synthetic...

Published: 7/25/2024 | Inventor(s): David Narum

Keywords(s): DC2XXX, DCXXXX, DXXXXX, GENES, Malaria, Malaria (Plasmodium sp.), MALARIAL, Methods, proteins, SACGHS DNA Patent Initial Set, Synthetic

Category(s): Collaboration Sought > Collaboration, Collaboration Sought > Licensing, TherapeuticArea > Infectious Disease, Application > Vaccines