Antisense oligonucleotide therapeutic approach for Timothy syndrome

Nature, Published online: 24 April 2024; doi:10.1038/s41586-024-07310-6Antisense oligonucleotides effectively decrease the inclusion of exon  8A of CACNA1C in human cells both in vitro and in rodents transplanted with human brain organoids, and a single intrathecal administration rescued both calcium changes and in vivo dendrite morphology of patient neurons.

Source: Nature AOP - April 24, 2024 Category: Research Authors: Xiaoyu Chen Fikri Birey Min-Yin Li Omer Revah Rebecca Levy Mayuri Vijay Thete Noah Reis Konstantin Kaganovsky Massimo Onesto Noriaki Sakai Zuzana Hudacova Jin Hao Xiangling Meng Seiji Nishino John Huguenard Sergiu P. Pa șca Source Type: research