Development of Emerin mRNA Lipid Nanoparticles to Rescue Myogenic Differentiation

Abstract excerpt

Emery-Dreifuss muscular dystrophy 1 (EDMD1) arises from mutations in EMD . Most EDMD1 patients lack detectable emerin expression. They experience symptoms such as skeletal muscle wasting, joint contractures, and cardiac conduction defects. Currently, physicians rely on treating patient symptoms, without addressing the underlying cause – lack of functional emerin protein. Thus, there is a need for therapeutic appr...

Topics

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

• beta Catenin
• CAR-T cell therapy research
• CRISPR and Genetic Engineering
• Genetic Neurodegenerative Diseases
• Intracellular Signaling Peptides and Proteins
• Lamin Type A
• Muscle Physiology and Disorders
• Viral Infections and Immunology Research

Identifiers and source

Literature Corpus work

1d309c14-046e-569a-a94f-f0091e88d49a

DOI

10.1101/2025.06.11.659076