Web9 jun. 2024 · RNA toxicity underlies the pathogenesis of disorders such as myotonic dystrophy type 1 (DM1). Muscular dystrophy is a key element of the pathology of DM1. The means by which RNA toxicity causes muscular dystrophy in DM1 is unclear. Here, we have used the DM200 mouse model of RNA toxicity due to the … Web12 aug. 2024 · For studies performed at Ionis Pharmaceuticals, total RNA was isolated from mouse tissues as previously described 46,47 using Taqman primer and probe sets.
Mapping the RNA therapeutics R&D landscape in 2024
Web25 jan. 2024 · 1 Ionis Pharmaceuticals Inc. Carlsbad, CA 92010, USA. PMID: 33367834 PMCID: PMC7826274 DOI: 10.1093/nar/gkaa1235 Abstract Antisense oligonucleotides … Web18 apr. 2024 · Ionis is developing an in-house and partnered pipeline of RNA-targeted drugs for severe and rare diseases. The company's first approved product, Spinraza™ … grand timeo hotel taormina sicily
Antibody–oligonucleotide conjugates enter the clinic - Nature
Web3 nov. 2024 · Alnylam and Sanofi have revealed full phase 3 data on their RNA interference (RNAi) drug patisiran, soundly beating a rival candidate from Ionis, inotersen. Both drugs target hereditary... Web12 apr. 2024 · About IONS. Ionis Pharmaceuticals, Inc. discovers and develops RNA-targeted therapeutics in the United States. The company offers SPINRAZA for spinal muscular atrophy (SMA) in pediatric and adult patients; TEGSEDI, an injection for the treatment of polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults; … Web10 nov. 2024 · More than 30 speakers from across industry and academia will present at the conference, which is sponsored by Ionis, University of California San Diego School of Medicine, Nature Biotechnology and Nature Structural & Molecular Biology.The conference was inspired by Ionis founder and executive chairman of the board Stanley T. Crooke, … grand tire and auto reviews