Back to search

Article

Gene Therapy Mediates Therapeutic Improvement in Cardiac Hypertrophy and Survival in a Murine Model of <i>MYBPC3</i> -Associated Cardiomyopathy

2024-02-23

Abstract excerpt

<h4>Background</h4> Hypertrophic cardiomyopathy (HCM) affects an estimated 600,000 people in the U.S. and is the leading cause of sudden cardiac arrest in those under 18. Loss-of-function mutations in Myosin Binding Protein C3 , MYBPC3 , are the most common genetic cause of HCM. The majority of MYBPC3 mutations causative for HCM result in truncations. The sarcomeric pathophysiology of the majority of HCM patie...

Topics

Open a Topic to create a Post that cites this publication.

Identifiers and source

Literature Corpus work
598e0c1b-76f0-5df4-928c-be1020b5b143
DOI
10.1101/2024.02.19.581102
Open publication

Related research

Semantic proximity does not establish scientific evidence.

Click a neighbor to travelStep 1 · 12 closest
Interactive article relationship graphSelect a related publication card to move it into the centre and load its closest explainable connections. Solid lines are source-backed structured connections. Dashed lines are semantic discovery signals and are not scientific evidence.
Gene Therapy Mediates Therapeutic Improvement in Cardiac Hypertrophy and Survival in a Murine Model of <i>MYBPC3</i> -Associated CardiomyopathyDOI 10.1101/2024.02.19.581102
AAV9-mediated MYBPC3 gene therapy with optimized expression cassette enhances cardiac function and survival in MYBPC3 cardiomyopathy modelsSemantic proximity
Targeting the population for gene therapy with MYBPC3.Semantic proximity
The impact of non-cardiomyocyte <i>MYBPC3</i> expression on the development of hypertrophic cardiomyopathySemantic proximity
Broad therapeutic benefit of myosin inhibition in hypertrophic cardiomyopathySemantic proximity
Mavacamten shows broad benefit in human and mouse models of MYBPC3-related hypertrophic cardiomyopathy.Semantic proximity
Repair of Mybpc3 mRNA by 5′-trans-splicing in a Mouse Model of Hypertrophic CardiomyopathySemantic proximity
Cardiac myosin-binding protein C (MYBPC3) in cardiac pathophysiology.Semantic proximity
<i>MYBPC3</i> D389V Variant Induces Hypercontractility in Cardiac OrganoidsSemantic proximity
Homology-Directed Repair of an MYBPC3 gene mutation in a rat model of hypertrophic cardiomyopathySemantic proximity
<i>MYBPC3</i> Mutations cause Hypertrophic Cardiomyopathy by Dysregulating Myosin: Implications for TherapySemantic proximity
Evaluation of MYBPC3 trans -Splicing and Gene Replacement as Therapeutic Options in Human iPSC-Derived CardiomyocytesSemantic proximity
The Role of Cardiac Myosin Binding Protein C3 in Hypertrophic Cardiomyopathy-Progress and Novel Therapeutic Opportunities.Semantic proximity
Select a neighboring publication to make it the new centre.