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Antisense oligonucleotide therapy for calmodulinopathy

ABSTRACT: Antisense oligonucleotide therapy for calmodulinopathy

PROVIDER: PRJNA1030420 | ENA |

REPOSITORIES: ENA

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			Action	DRS
	SRR26454170_1.fastq.gz	Fastqsanger.gz
	SRR26454170_2.fastq.gz	Fastqsanger.gz
	SRR26454171_1.fastq.gz	Fastqsanger.gz
	SRR26454171_2.fastq.gz	Fastqsanger.gz
	SRR26454172_1.fastq.gz	Fastqsanger.gz

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Antisense oligonucleotide therapy for calmodulinopathy

Project description:Calmodulinopathies are rare inherited arrhythmia syndromes caused by dominant gain of function variants in one of three genes, CALM1, CALM2, and CALM3, which each encode the identical calmodulin (CaM) protein. We hypothesized that antisense oligonucleotide (ASO)-mediated depletion of an affected calmodulin gene would ameliorate disease manifestations, while the other two calmodulin genes would preserve CaM level and function. Here we tested this hypothesis using human induced pluripotent stem cell-derived cardiomyocyte (iPSC-CM) and mouse models of CALM1 pathogenic variants. Human CALM1F142L/+ iPSC-CMs exhibited prolonged action potentials, modeling congenital long QT syndrome. CALM1-depleting ASOs did not alter CaM protein level and normalized repolarization of CALM1F142L/+ iPSC-CMs. Similarly, an ASO targeting murine Calm1 depleted Calm1 transcript without affecting CaM protein level. This ASO alleviated drug-induced arrhythmia in CalmN98S/+ mice without causing observable toxicity. These results provide proof-of-concept that ASOs targeting individual calmodulin genes are potentially effective and safe therapies for calmodulinopathies.

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