ASO approaches Several ASOs are being investigated in HD model systems, but we begin by exploring in detail the first targeted huntingtin-lowering compound to enter a human trial. Safety of antisense oligonucleotide and siRNA-based therapeutics. Each uses a protein-coding sequence encapsulated in a viral vector, injected intracranially, that transduces cells, causing them to produce a functional, non-native therapeutic protein. For instance, some synthetic oligonucleotides are recognised by toll-like receptors on immune cells, raising the risk of immunogenicity that may be difficult to predict from animal studies. A safety trial of an ASO in superoxide-dismutase 1 SOD1 mutation-associated amyotrophic lateral sclerosis revealed no concerns 48 and another is underway with a more potent ASO. Six-month partial suppression of Huntingtin is well tolerated in the adult rhesus striatum.
Divergent () - Full Cast & Crew - IMDb
He met his father, Carl Autry Sr. Tap here to turn on desktop notifications to get the news sent straight to you. Song Writers Hall of Fame Members. Not sure who Richard Quest is? Contentious material about living persons that is unsourced or poorly sourced must be removed immediately , especially if potentially libelous or harmful.
Ruling: Too late to change California rules on ballot photos
Description: District Judge Christine Arguello said Wednesday the way the law is written may pose valid constitutional problems over the right to free speech, but she did not immediately issue a ruling. He made his film debut in the motion picture Remember My Name. Twenty recordings that were written by or performed by or produced by Pittsburghers are included in the Grammy Hall of Fame. Retrieved July 21, Share or comment on this article: