Antisense Oligonucleotide Therapy for Calmodulinopathy

Calmodulinopathies are rare inherited arrhythmia syndromes caused by dominant heterozygous variants in , , or , which each encode the identical CaM (calmodulin) protein. We hypothesized that antisense oligonucleotide (ASO)-mediated depletion of an affected calmodulin gene would ameliorate disease ma...

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Veröffentlicht in:	Circulation (New York, N.Y.) N.Y.), 2024-10, Vol.150 (15), p.1199-1210
Hauptverfasser:	Bortolin, Raul H, Nawar, Farina, Park, Chaehyoung, Trembley, Michael A, Prondzynski, Maksymilian, Sweat, Mason E, Wang, Peizhe, Chen, Jiehui, Lu, Fujian, Liou, Carter, Berkson, Paul, Keating, Erin M, Yoshinaga, Daisuke, Pavlaki, Nikoleta, Samenuk, Thomas, Cavazzoni, Cecilia B, Sage, Peter T, Ma, Qing, Whitehill, Robert D, Abrams, Dominic J, Carreon, Chrystalle Katte, Putra, Juan, Alexandrescu, Sanda, Guo, Shuai, Tsai, Wen-Chin, Rubart, Michael, Kubli, Dieter A, Mullick, Adam E, Bezzerides, Vassilios J, Pu, William T
Format:	Artikel
Sprache:	eng
Schlagworte:	Action Potentials - drug effects Animals Calmodulin - genetics Calmodulin - metabolism Disease Models, Animal Genetic Therapy - methods Humans Induced Pluripotent Stem Cells - metabolism Long QT Syndrome - drug therapy Long QT Syndrome - genetics Long QT Syndrome - physiopathology Long QT Syndrome - therapy Mice Mice, Knockout Myocytes, Cardiac - metabolism Oligonucleotides, Antisense - pharmacology Oligonucleotides, Antisense - therapeutic use
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