Search Results - transgene

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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
NOVEL UBIQUITOUS CHROMATIN-OPENING ELEMENT FOR STABLE LONG-TERM TRANSGENE EXPRESSION
NOVEL UBIQUITOUS CHROMATIN-OPENING ELEMENT FOR STABLE LONG-TERM TRANSGENE EXPRESSION Researchers at Stanford and the Chan Zuckerberg Biohub have identified a novel chromatin remodeling element for stable long-term transgene expression. Gene therapy applications and biomanufacturing of therapeutic proteins require stable long-term transgene expression....
Published: 3/17/2023   |   Inventor(s): Christina Smolke, Shireen Rudina
Keywords(s): Chromatin Remodeling, Genetic Dissection, Genome Integration, Transgene
Category(s): Technology Classifications > Biology