A fleeting glimpse of functional benefit of the complete DMD gene in a large animal model of Duchenne muscular dystrophy

Assessment of systemic AAV-microdystrophin gene therapy in the GRMD model of Duchenne muscular dystrophy

(2023) Sharla M. Birch et al.   Science Translational Medicine

Is it time for genetic modifiers to predict prognosis in Duchenne muscular dystrophy?

(2023) Luca Bello et al.   Nature Reviews Neurology

Systemic deletion of DMD exon 51 rescues clinically severe Duchenne muscular dystrophy in a pig model lacking DMD exon 52

(2023) Michael Stirm et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

A cellular and molecular spatial atlas of dystrophic muscle

(2023) Michael J. Stec et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Phenotypic features of dystrophin gene knockout pigs harboring a human artificial chromosome containing the entire dystrophin gene

(2023) Masahito Watanabe et al.   Molecular Therapy-Nucleic Acids

Single-cell and spatial transcriptomics identify a macrophage population associated with skeletal muscle fibrosis

(2023) Gerald Coulis et al.   Science Advances

A Dystrophin Exon-52 Deleted Miniature Pig Model of Duchenne Muscular Dystrophy and Evaluation of Exon Skipping

(2021) Yusuke Echigoya et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

Somatic gene editing ameliorates skeletal and cardiac muscle failure in pig and human models of Duchenne muscular dystrophy

(2020) A. Moretti et al.   NATURE MEDICINE

Dystrophin-deficient pigs provide new insights into the hierarchy of physiological derangements of dystrophic muscle

(2013) Nikolai Klymiuk et al.   HUMAN MOLECULAR GENETICS