Work

The longer, the better? Optimizing dystrophin constructs for cardiac gene therapy in Duchenne muscular dystrophy

Year: 2026

Type: article

Abstract: Gene therapy for Duchenne muscular dystrophy (DMD) has so far focused mainly on improving skeletal muscle function; however, effective treatment of the disease will also require protection of the heart. In a recently published study in Molecular Therapy Advances, Keegan et al.1 addressed this challenge using a patient-in-a-dish model based on induced pluripotent stem cell-derived cardiomyocytes (iPSC-CMs), as discussed in the following text.

Source: Molecular Therapy Advances

DOI: 10.1016/j.omta.2026.201870

Authors: Agnieszka Łoboda, Józef Dulak

Institution: Jagiellonian University

Language: English

Topic: Muscle Physiology and Disorders

Subfield: Molecular Biology

Field: Biochemistry, Genetics and Molecular Biology

Domain: Life Sciences

SDG: Good health and well-being

Open Access status: hybrid

Funder: Narodowe Centrum Nauki