Antisense Therapy Attenuates Phospholamban p.(Arg14del) Cardiomyopathy in Mice and Reverses Protein Aggregation

Antisense Therapy Attenuates Phospholamban p.(Arg14del) Cardiomyopathy in Mice and Reverses Protein Aggregation

Inherited cardiomyopathy caused by the p.(Arg14del) pathogenic variant of the phospholamban (<i>PLN</i>) gene is characterized by intracardiomyocyte PLN aggregation and can lead to severe dilated cardiomyopathy. We recently reported that pre-emptive depletion of PLN attenuated heart fail...

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Bibliographic Details
Main Authors: Tim R. Eijgenraam, Nienke M. Stege, Vivian Oliveira Nunes Teixeira, Remco de Brouwer, Elisabeth M. Schouten, Niels Grote Beverborg, Liu Sun, Daniela Später, Ralph Knöll, Kenny M. Hansson, Carl Amilon, David Janzén, Steve T. Yeh, Adam E. Mullick, Peter van der Meer, Rudolf A. de Boer, Herman H. W. Silljé
Format: Article
Language:English
Published: MDPI AG 2022-02-01
Series:International Journal of Molecular Sciences
Subjects:
phospholamban
genetic mutation
protein aggregation
cardiomyopathy
heart failure
gene therapy
Online Access:https://www.mdpi.com/1422-0067/23/5/2427

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https://www.mdpi.com/1422-0067/23/5/2427

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