Enhanced Delivery of Ligand-Conjugated Antisense Oligonucleotides (C16-HA-ASO) Targeting DMPK Transcripts for the Treatment of Myotonic Dystrophy Type 1.
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DOI: 10.1089/hum.2022.069
Abstract: Myotonic dystrophy type 1 (DM1) is a neuromuscular disorder that affects many organs. It is caused by the expansion of a cytosine-thymine-guanine (CTG) triplet repeat in the 3' untranslated region (UTR) of the human myotonic… read more here.
Keywords: dystrophy type; mutant hdmpk; ionis 877864; myotonic dystrophy ... See more keywords
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