Ionis core antisense research
Web17 dec. 2024 · About Ionis Pharmaceuticals, Inc. As the leader in RNA-targeted drug discovery and development, Ionis has created an efficient, broadly applicable, drug … WebAntisense oligonucleotides modified with phosphorothioate linkages (PS-ASOs) can enter cells via endocytic pathways and must escape from membraned organelles to …
Ionis core antisense research
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WebThe Ionis antisense pipeline Our antisense technology platform has served as a springboard for drug discovery and realized hope for patients with unmet needs. Our broad, diverse pipeline has more than 40 potential first-in-class and/or best-in-class medicines designed to treat a broad range of diseases. WebAt Ionis, Elaine’s research is focused on understanding the activity of GLP-1 conjugated antisense oligonucleotides in the beta cells of the pancreas. The goal: identifying and advancing new targets for Type 1 and Type 2 diabetes. So, …
Web19 dec. 2024 · Ionis Pharmaceuticals, Inc. 1 year 9 months Associate Director Jul 2024 - Present10 months Carlsbad, California, United States … Web10 jul. 2024 · Angelman syndrome (AS) is a rare neurodevelopmental disorder caused by loss of function of the maternally inherited UBE3A allele. In neurons, the paternal allele of UBE3A is silenced in cis by the long noncoding RNA, UBE3A-ATS . Unsilencing paternal UBE3A by reducing UBE3A-ATS is a promising therapeutic approach for the treatment of …
Web11 mei 2024 · 1 Core Antisense Research Ionis Pharmaceuticals, Inc, Carlsbad, CA 92008, USA. PMID: 35664704 PMCID: PMC9136273 DOI: 10.1016/j.omtn.2024.05.024 … WebThis is the approach we have taken at Ionis. Obviously, to create antisense technology, we have had to address a wide array of strategic questions, for example, the medicinal …
WebABOUT IONIS PHARMACEUTICALS, INC. As the leader in RNA-targeted drug discovery and development, Ionis has created an efficient, broadly applicable, drug discovery …
Web2 nov. 2024 · CORE is a global, double-blind, randomized, placebo-controlled, registrational, Phase 3 study in patients with severe hypertriglyceridemia. It is designed to compare … small diamond shaped mirrorsWeb2 sep. 2024 · A phase 1, randomized, double-blind, placebo-controlled, dose-escalation trial was designed to assess the safety and side-effect profile, pharmacokinetics, and pharmacodynamics of monthly... small diamond sets designsWeb10 okt. 2024 · - Ionis scientists, including CEO Stanley Crooke, M.D., Ph.D., recognized with prestigious Paper of the Year Award for their work on advancing core antisense research Published Oct 10, 2024 7:05AM EDT sonder the circ parkingWebOur antisense therapies are disrupting diseases and, we hope, changing their course—from the rarest of conditions, to those that impact millions of people. And as we … sonderthemaWeb2 dec. 2024 · Antisense oligonucleotides (ASOs) regulate gene expression by binding to complementary target RNA, and ASOs can be designed to take advantage of a growing array of post RNA binding molecular mechanisms. Intracellular trafficking of ASOs influences their efficacy. We have identified a number of membrane-less structures in the … sonderthemen fazWeb19 sep. 2024 · We recently showed that translation can be enhanced by antisense oligonucleotides (ASOs) that target upstream open reading frames. Here we report the … sonder the dutch nycWeb2 sep. 2024 · A phase 1, randomized, double-blind, placebo-controlled, dose-escalation trial was designed to assess the safety and side-effect profile, pharmacokinetics, and … sonder the drayton