Society News

Antisense oligonucleotide (ASO) therapies, which bind to RNA and modify protein expression, are promising drugs for treating neurological conditions. However, their inability to cross the blood-brain barrier (BBB) sometimes makes injection directly into the cerebral spinal fluid (via intrathecal injection) necessary, which is not without its risks or drawbacks (1). However, a recent study by Denali Therapeutics, as well ...

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 was referred to a lipid specialist at John Hopkins University Hospital and, after being genetically ...

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 the biotechnology sphere, with his leadership skills, optimism, and willingness to take risks still changing ...

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 such as basic research, as a diagnostic tool, and for large-scale genetic screens, as well ...

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 ultra-rare diseases are often entirely excluded from drug development programs (1). However, the sphere of ...