The Man Behind the Molecules: John Maraganore’s RNAi Legacy
The 2024 OTS lifetime achievement winner, John Maraganore, is best known for his pioneering role in making RNA interference (RNAi) a successful therapeutic modality while CEO at Alnylam Pharmaceuticals. However, before joining and after leaving Alnylam, he was and continues to be influential in ...
Mastering the Art of Reviewing Scientific Papers
Date: December 12, 2024 Time: 8am PST / 11am EST / 5pm CET Title: Mastering the Art of Reviewing Scientific Papers Description: Join our webinar on mastering the art of reviewing scientific papers, presented by Prof. Dr. Annemieke Aartsma-Rus, co-editor in ...
Bridge Editing: A Novel Technique for Large-Scale DNA Modifications
Link Source: Hiraizumi, M., Perry, N.T., Durrant, M.G. et al. Structural mechanism of bridge RNA-guided recombination. Nature 630, 994–1002 (2024). Gene editing has swiftly advanced since CRISPR was discovered, with CRISPR being developed not only to provide therapeutic gene editing treatments for humans but also for uses ...
Near Sequence Homology Does Not Guarantee siRNA Cross-Species Efficacy
Iris Valeria Rivera Flores, Kathryn Monopoli, Samuel Jackson, Dimas Echeverria, Daniel O’Reilly, Robert H. Brown, and Anastasia Khvorova Pages:234–244Published Online:27 August 2024 https://doi.org/10.1089/nat.2024.0030
Personalized ASO Provides Improvements for a Girl with KAND, an Ultra-rare Disease
Within the world of rare diseases exist conditions so sparse and infrequent they’re called nano-rare — or N of 1 — diseases and typically affect only one to thirty people worldwide (1). While less than 10% of rare diseases have approved treatments, nano-rare or ...
2024 OTS Lifetime Achievement Award Winner John Maraganore
John Maraganore, Ph.D.Founding CEO & Director, Alnylam Pharmaceuticals 2002-2021Biography:Dr. John Maraganore served as the founding CEO and a Director of Alnylam from 2002 to 2021, where he built and led the company from early platform research on RNA interference through global approval and commercialization of the ...
Angelman Syndrome Therapies Show Positive Results in Early Phase Clinical Trials
A clinical trial for a drug designed to treat the rare neurodevelopmental disorder known as Angelman syndrome (AS) has achieved a favorable safety profile and lessening of overall symptoms in trial participants. In a recent press release, Ionis announced the detailed HALOS study results ...
Encouraging Progress in the Hunt for a Huntington’s Treatment
Described as a combination of Alzheimer's disease, Amyotrophic lateral sclerosis, and Parkinson's disease all at once, Huntington's disease is a rare and debilitating neurological disorder passed down within affected families. With a 50% chance of a child inheriting it from a parent with the ...
mRNA Nuclear Clustering Leads to a Difference in Mutant Huntingtin mRNA and Protein Silencing by siRNAs In Vivo
Date: September 19, 2024 Time: 8am PDT / 11am EDT / 5pm CEST Title: mRNA Nuclear Clustering Leads to a Difference in Mutant Huntingtin mRNA and Protein Silencing by siRNAs In Vivo Description: Huntington’s disease (HD) is an autosomal dominant neurodegenerative ...
Sequence- and Structure-Dependent Cytotoxicity of Phosphorothioate and 2′-O-Methyl Modified Single-Stranded Oligonucleotides
Authors: Laura V. Croft Mark Fisher, Tabassum Khair Barbhuiya, Serene El-Kamand, Samuel Beard, Aleksandra Rajapakse, Roland Gamsjaeger, Liza Cubeddu, Emma Bolderson, Ken O’Byrne , Derek Richard derek.richard@qut.edu.au, and Neha S. Gandhi Published Online: 17 June 2024 Abstract Single-stranded oligonucleotides (SSOs) are a rapidly expanding class ...
