Antisense Oligonucleotide Therapy for Calmodulinopathy.

Raul H BortolinFarina NawarChaehyoung ParkMichael A TrembleyMaksymilian ProndzynskiMason E Sweat Peizhe Wang Jiehui Chen Fujian Lu Carter LiouPaul BerksonErin M KeatingDaisuke Yoshinaga Nikoleta Pavlaki Thomas Samenuk Cecilia B Cavazzoni Peter T Sage Qing Ma Robert D Whitehill Dominic J R Abrams Chrystalle Katte CarreonJuan PutraSanda Alexandrescu Shuai GuoWen-Chin TsaiMichael Rubart Dieter A Kubli Adam E Mullick Vassilios J BezzeridesWilliam T Pu

Published in: Circulation (2024)

These results provide proof of concept that ASOs targeting individual calmodulin genes are potentially effective and safe therapies for calmodulinopathies.

Keyphrases

protein kinase
genome wide
cancer therapy
bioinformatics analysis
genome wide identification
nucleic acid
dna methylation
gene expression
drug delivery