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Gene Editing And Therapy Equipment Supplied In Usa New York
7 equipment items found
Manufactured by:Taconic Biosciences, Inc. based inRensselaer, NEW YORK (USA)
The Abb mouse was developed in the laboratory of Michael J. Grusby et al. of the Harvard School of Public Health. The model was created by targeting the H2-Ab1 gene in ES-D3 cells and injecting the targeted ES cells into C57BL/6 blastocysts. Resultant chimeras were backcrossed to C57BL/6 for 4 generations (N4). Taconic received stock from Tufts University in December 1991, and the mice were ...
Manufactured by:Rocket Pharmaceuticals, Inc. based inCranbury, NEW JERSEY (USA)
FA is a rare, genetic disorder affecting DNA repair. Approximately two-thirds of FA cases are caused by genetic defects in the FANCA gene, which results in the FA subtype known as FA Complementation Group A (FA-A). FA patients may develop bone marrow failure (very low blood counts), cancers of the blood or other ...
Manufactured by:Rocket Pharmaceuticals, Inc. based inCranbury, NEW JERSEY (USA)
PKD is a rare, genetic blood disorder. PKD is caused by a defect in the PKLR gene which is responsible for energy production in red blood cells (RBCs). RBCs carry oxygen to the rest of the body. When the PKLR gene has a defect, RBCs are limited in their ability to produce energy and remain intact. As a result, patients with PKD frequently have anemia, chronic fatigue, yellowing of the skin and ...
Manufactured by:Rocket Pharmaceuticals, Inc. based inCranbury, NEW JERSEY (USA)
LAD-I is a rare genetic disorder affecting the immune system and is caused by a defect in the ITGB2 gene, leading to a deficiency in CD18. Due to this deficiency, white blood cells (primarily neutrophils) are unable to leave the bloodstream to go to the site of infection. As a result, patients with severe LAD-I can develop life-threatening infections which they are unable to fight. Without a ...
Manufactured by:Hemogenyx Pharmaceuticals plc based inLondon, UNITED KINGDOM
Hemogenyx Pharmaceuticals utilizes postnatal human hemogenic endothelial cells (Hu-PHEC cells), discovered by Hemogenyx Pharmaceuticals’ Co-Founder Dr Vladislav Sandler, to generate cancer-free hematopoietic stem cells (HSC) for use in transplants to treat blood cancers with superior results. A patent for Dr Sandler’s work was issued in the US in February ...
Manufactured by:Rocket Pharmaceuticals, Inc. based inCranbury, NEW JERSEY (USA)
Danon Disease (DD) is a rare genetic disorder that is characterized by severe and primarily hypertrophic cardiomyopathy. Skeletal muscle weakness and mild cognitive impairment are also common. The causative mutation has been identified in the gene encoding for lysosome-associated membrane protein (LAMP2), and in particular the LAMP2B version of the gene which is primarily expressed in heart, ...
Manufactured by:Scopus BioPharma based inNew York, NEW YORK (USA)
Our licensed gene therapy, CO-sTiRNA™, is a dual-action STAT3 inhibitor. STAT3 is a gene that drives tumor cell growth and anti-tumor immune suppression. CO-sTiRNA™ is a highly selective and targeted gene therapy that silences the activity of the STAT3 gene by way of RNA interference. CO-sTiRNA™ also stimulates TLR9 receptors to activate the body’s immune defense to ...