Long-term maintenance of dystrophin expression and resistance to injury of skeletal muscle in gene edited DMD mice

The nuclear envelope protein Net39 is essential for muscle nuclear integrity and chromatin organization

(2021) Andres Ramirez-Martinez et al.   Nature Communications

CRISPR technologies for the treatment of Duchenne muscular dystrophy

(2021) Eunyoung Choi et al.   MOLECULAR THERAPY

Precise correction of Duchenne muscular dystrophy exon deletion mutations by base and prime editing

(2021) F. Chemello et al.   Science Advances

Enhanced CRISPR-Cas9 correction of Duchenne muscular dystrophy in mice by a self-complementary AAV delivery system

(2020) Yu Zhang et al.   Science Advances

Correction of muscular dystrophies by CRISPR gene editing

(2020) Francesco Chemello et al.   JOURNAL OF CLINICAL INVESTIGATION

Correction of three prominent mutations in mouse and human models of Duchenne muscular dystrophy by single-cut genome editing

(2020) Yi-Li Min et al.   MOLECULAR THERAPY

p38 MAPK in Glucose Metabolism of Skeletal Muscle: Beneficial or Harmful?

(2020) Eyal Bengal et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

In Vivo Gene Editing of Muscle Stem Cells with Adeno-Associated Viral Vectors in a Mouse Model of Duchenne Muscular Dystrophy

(2020) Jennifer B. Kwon et al.   Molecular Therapy-Methods & Clinical Development

CRISPR-Cas9 corrects Duchenne muscular dystrophy exon 44 deletion mutations in mice and human cells

(2019) Yi-Li Min et al.   Science Advances

Long-term evaluation of AAV-CRISPR genome editing for Duchenne muscular dystrophy

(2019) Christopher E. Nelson et al.   NATURE MEDICINE

Development of Novel Micro-dystrophins with Enhanced Functionality

(2019) Julian N. Ramos et al.   MOLECULAR THERAPY

In Situ Modification of Tissue Stem and Progenitor Cell Genomes

(2019) Jill M. Goldstein et al.   Cell Reports

AAV9 Edits Muscle Stem Cells in Normal and Dystrophic Adult Mice

(2019) Michael E. Nance et al.   MOLECULAR THERAPY

Search-and-replace genome editing without double-strand breaks or donor DNA

(2019) Andrew V. Anzalone et al.   NATURE

Barium chloride injures myofibers through calcium-induced proteolysis with fragmentation of motor nerves and microvessels

(2019) Aaron B. Morton et al.   Skeletal Muscle

Correction of diverse muscular dystrophy mutations in human engineered heart muscle by single-site genome editing

(2018) Chengzu Long et al.   Science Advances

Gene editing restores dystrophin expression in a canine model of Duchenne muscular dystrophy

(2018) Leonela Amoasii et al.   SCIENCE

Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy

(2017) Jerry R. Mendell et al.   NEW ENGLAND JOURNAL OF MEDICINE

Single-cut genome editing restores dystrophin expression in a new mouse model of muscular dystrophy

(2017) Leonela Amoasii et al.   Science Translational Medicine

CRISPR-mediated Genome Editing Restores Dystrophin Expression and Function in mdx Mice

(2016) Li Xu et al.   MOLECULAR THERAPY

Absence of Dystrophin Disrupts Skeletal Muscle Signaling: Roles of Ca2+, Reactive Oxygen Species, and Nitric Oxide in the Development of Muscular Dystrophy

(2016) David G. Allen et al.   PHYSIOLOGICAL REVIEWS

How much dystrophin is enough: the physiological consequences of different levels of dystrophin in themdxmouse

(2015) Caroline Godfrey et al.   HUMAN MOLECULAR GENETICS

The TREAT-NMD DMD Global Database: Analysis of More than 7,000 Duchenne Muscular Dystrophy Mutations

(2015) Catherine L. Bladen et al.   HUMAN MUTATION

Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophy

(2015) C. Long et al.   SCIENCE

In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy

(2015) C. E. Nelson et al.   SCIENCE

Immune-mediated pathology in Duchenne muscular dystrophy

(2015) Amy S. Rosenberg et al.   Science Translational Medicine

Gene expression in mdx mouse muscle in relation to age and exercise: aberrant mechanical–metabolic coupling and implications for pre-clinical studies in Duchenne muscular dystrophy

(2014) Giulia Maria Camerino et al.   HUMAN MOLECULAR GENETICS

Screening of Duchenne Muscular Dystrophy (DMD) Mutations and Investigating Its Mutational Mechanism in Chinese Patients

(2014) Chen Chen et al.   PLoS One

Therapy for Duchenne muscular dystrophy: renewed optimism from genetic approaches

(2013) Rebecca J. Fairclough et al.   NATURE REVIEWS GENETICS

Humoral Immune Response to AAV

(2013) Roberto Calcedo et al.   Frontiers in Immunology

The Effects of Low Levels of Dystrophin on Mouse Muscle Function and Pathology

(2012) Maaike van Putten et al.   PLoS One

Genotype-phenotype analysis in 2,405 patients with a dystrophinopathy using the UMD-DMD database: a model of nationwide knowledgebase

(2009) Sylvie Tuffery-Giraud et al.   HUMAN MUTATION