Antisense approaches for the treatment of Duchenne muscular dystrophy and recent advances to address DMD brain comorbidities
Date: May 23, 2024 Time: 8am PDT / 11am EDT / 5pm CEST Title: Antisense approaches for the treatment of Duchenne muscular dystrophy and recent advances to address DMD brain comorbidities Description: This presentation will cover the development of ASO-based approaches ...
2023 Annual Meeting Poster Awardee Spotlight: Stan and Moravčík
Date: May 16, 2024 Time: 8am PDT / 11am EDT / 5pm CEST Title: Validation lab: allowing standardized in vitro and in vivo experiments for candidate treatments for Duchenne muscular dystrophy Description: The Validation Lab was established in 2015 with support ...
In-silico siRNA Off-Target Predictions: What Should We Be Looking For?
Date: May 2, 2024 Time: 8am PDT / 11am EDT / 5pm CEST Title: In-silico siRNA Off-Target Predictions: What Should We Be Looking For? Description: Assessing sequence-dependent off-target risks is a critical step during siRNA development to ensure the safety and ...
In vivo Optical Control of Gene-Silencing in Japanese Medaka Fish Embryos
Time: 8am PDT / 11am EDT / 5pm CEST Date: April 25, 2024 Title: In vivo Optical Control of Gene-Silencing in Japanese Medaka Fish Embryos Description: The siRNA oligonucleotide duplex inherent to biology, functions by binding and cleaving mRNA, a process ...
RNA Targeting Technology – Chemistry and Delivery
Time: 8am PDT / 11am EDT / 4pm CET Date: March 21, 2024 Title: RNA Targeting Technology – Chemistry and Delivery Description: Chemistry coupled with pharmacology, both preclinical and clinical, forms the basis for the pipeline we manage at Ionis today. ...
2023 Annual Meeting Poster Awardee Spotlight
Time: 8AM PST | 11AM EST | 5PM CET Date: February 29, 2024 Title: Towards Allele-Selective Gapmer ASO Design Description: Allele selective gapmer antisense oligonucleotides (ASOs) are a promising strategy for the treatment of rare monogenic neurodevelopmental disorders caused by gain-of-function or ...
Meet OTS’ New BOD Trainee Reps
Time: 8AM PST | 11AM EST | 5PM CET Date: February 22, 2024 Title: Advancing Therapeutic Approaches for Erythropoietic Protoporphyria Description: Currently, no curative treatment is available for the rare disease erythropoietic protoporphyria (EPP). The genetic background of EPP is well ...
2023 Annual Meeting Poster Awardee Spotlight
Time: 8AM PST | 11AM EST | 5PM CET Date: January 25, 2024 Title: siRNA therapy for ex vivo treatment of stem cell grafts and/or donor lymphocytes Description: Allogeneic T cells represent the most potent treatment for hematopoietic malignancies such es ...
Non-viral DNA delivery: Opportunities and challenges
Time: 8AM PST | 11AM EST | 5PM CET Date: October 12, 2023 This presentation will cover defining the key hurdles for non-viral DNA delivery, innate immune stimulation, and delivery to post-mitotic nuclei, as well as strategies to address these challenges. ...
ASO activity and distribution after LNP delivery
Time: 8AM PST | 11AM EST | 5PM CET Date: Thursday, September 14, 2023 Webinar title: Assessing antisense oligonucleotide delivery to the brain What is your research about? Amy Byrnes, PhD, focuses her research on understanding how delivery vehicles impact antisense oligonucleotide (ASO) ...
