Article

Non-cell autonomous cardiomyocyte regulation complicates gene supplementation therapy for <i>LMNA</i> cardiomyopathy

2023-07-18

Abstract excerpt

<h4>Aims</h4> Recombinant adeno-associated viruses (rAAVs) are federally approved gene delivery vectors for in vivo gene supplementation therapy. Loss-of-function truncating variants of LMNA , the coding gene for Lamin-A/C, are one of the primary causes of inherited dilate cardiomyopathy (DCM). Here we aim to study whether AAV-based LMNA supplementation could treat LMNA deficiency-triggered cardiac defects. <...

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Literature Corpus work: b44d7110-43a4-5674-8d07-cbc2588b4788
DOI: 10.1101/2023.07.18.549413

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