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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: 10/28/2024 | 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

Wild-Type and DNA Polymerase beta Null Mouse Embryotic Fibroblast Cell Lines Harboring a lambda-LIZ Transgene

Of great utility in toxicology and DNA repair research are knockout mice with cell lines enabling one to evaluate generations of gene mutations as a direct function of base excision repair. Of particular importance are lambda-LIZ transgenes. Likewise, wild-type and beta-pol null cell lines are equally important. While there exist cell lines carrying...

Published: 10/28/2024 | Inventor(s): Robert Sobol

Keywords(s): 3-@hydroxyacyl-coa dehydrogenase deficiency, 47 XYY syndrome, AC5XXX, ACXXXX, AXXXXX, B, Cell, DNA, Double Y, Embryonic, Fibroblast, HAD deficiency, harboring, HIS deficiency, Histidinemia, KO, Lines, Mouse, NULL, polymerase, SV40T-Ag, TRANSFORMED, TRANSGENE, WILD-TYPE, Y-LIZ

Category(s): Collaboration Sought > Licensing