Oligonucleotide Treatment Advances Offer Relief for Patients with Familial Chylomicronemia Syndrome
At just three weeks old, Darlene was sent to the emergency room after her mom had brought her to a pediatrician. The baby wouldn't stop crying and wasn't eating. Concerningly, her triglyceride levels were in the 20,000's, far over the normal 150 level. Darlene ...
Trainee Spotlight Series: Poster Winners #OTS24 Session 3
Date: March 13, 2025 Time: 12pm EDT / 5pm CET Join us for this Trainee Spotlight webinar featuring trainees Jessica Bennett, and Chris Saab, all of whom won poster awards at the 2024 OTS Annual Meeting. Title: Reprogramming DNA with a ...
Trainee Spotlight Series: Poster Winners #OTS24 Session 2
Date: February 13, 2025 Time: 11-12pm EST Join us for this Trainee Spotlight webinar featuring trainees Edwin van Oosten, Josh Robinson, and Trishalina Das, all of whom won poster awards at the 2024 OTS Annual Meeting. Title: Exploring Nucleic ...
Trainee Spotlight Series: Poster Winners #OTS24 Session 1
Date: January 23, 2025 Time: 11-12pm EST Title: Antisense oligonucleotides targeting linked-SNPs provide allele-specific knockdown of a dominant-negative SPTAN1 pathogenic variant Description: Our first webinar of the year features trainees Christiana Wang, Elaine Kang, and Sinan Faiad, all of whom won poster ...
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 ...
