S21-376 Treatment of Timothy syndrome using an antisense oligonucleotide Scientists in Sergiu Pasca's group at Stanford University have used patient-derived organoids, assembloids and in vivo transplantation to discover and validate an antisense oligonucleotide drug for the treatment of Timothy syndrome. Sergiu Pasca Fikri Birey Xiaoyu Chen
S22-415 A Method for Suppressing Innate Immune Responses to RNA Therapy Stanford researchers have developed a method to suppress immune responses to RNA therapy by chemically treating the RNA with an acylating reagent. Eric Kool Linglan Fang