Transmembrane Protease, Serine 6 (TMPRSS6) Antisense Oligonucleotide (IONIS-TMPRSS6-LRX) Reduces Plasma Iron Levels of Healthy Volunteers in a Phase 1 Clinical Study
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DOI: 10.1182/blood-2018-99-115339
Abstract: Iron overload is the major cause of morbidity and mortality in beta-thalassemia patients. The low levels of beta-globin and ineffective erythropoiesis in these patients result in the suppression of hepcidin. The inappropriately low levels of… read more here.
Keywords: ionis pharmaceuticals; tmprss6; pharmaceuticals inc; ionis ... See more keywords
Therapeutic Targeting of Interferon Regulatory Factor 4 with Next Generation Antisense Oligonucleotides Produces Robust In Vivo Antitumor Activity in Preclinical Models of Multiple Myeloma
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DOI: 10.1182/blood.v130.suppl_1.3078.3078
Abstract: Despite significant recent advances in therapeutic options, multiple myeloma (MM) remains an incurable disease, and novel agents targeting novel molecular pathways with activity in advanced patients are urgently needed. Interferon Regulatory Factor 4 (IRF4) is… read more here.
Keywords: interferon regulatory; ionis pharmaceuticals; regulatory factor; multiple myeloma ... See more keywords
Inhibition of Transcriptional Regulator Taz (Wwtr1) by Antisense Oligonucleotide (ASO) Treatment Improves Obesity and Hepatic Steatosis in a Mouse Model of NASH
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DOI: 10.2337/db18-2439-pub
Abstract: Nonalcoholic fatty liver disease (NAFLD) is a common disorder that affects millions of people around the world. People with fatty liver disease alone are usually asymptomatic until a second insult to the liver, such as… read more here.
Keywords: aso treatment; taz; ionis pharmaceuticals; pharmaceuticals inc ... See more keywords