Article

Dissociation of disease phenotype and allele silencing in hypertrophic cardiomyopathy

2019-06-12

Abstract excerpt

Allele-specific RNA silencing has been shown to be an effective therapeutic treatment in a number of diseases, including neurodegenerative disorders. Studies of allele-specific silencing in hypertrophic cardiomyopathy to date have focused on mouse models of disease. Here, we investigate two methods of allele-specific silencing, short hairpin RNA (shRNA) and antisense oligonucleotide (ASO) silencing, using a human...

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Literature Corpus work: cf6a99bc-6582-52bc-9b37-b7e89bb2eafb
DOI: 10.1101/642421

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